By Cathy Yarbrough, Contributing Editor

Gene therapy pioneer Katherine High, M.D., was looking forward to her first meeting in 2011 with Jeffrey Marrazzo, then a consultant to the CEO of Childrens Hospital of Philadelphia (CHOP). A veteran of three life sciences companies, Marrazzo was meeting with Dr. High and other CHOP leaders to identify potential new revenue streams for the hospital.

Dr. High, an international leader in gene therapy research and clinical application, had considered postponing the meeting because she was so busy with her work as director of the hospitals Center for Cellular and Molecular Therapeutics (CCMT). However, she did not reschedule because she wanted to ask Marrazzo for a favor: Could he speak with the VCs who were calling her and inquiring about investing in CCMTs work on RPE65?

I hadnt spoken to them yet, because at the time I was busier than usual with my patient care, research, and teaching responsibilities. In addition, VCs are not a constituency that I normally deal with, said Dr. High, professor of pediatrics at the University of Pennsylvania as well as a Howard Hughes medical investigator.

Scheduled to last just 60 minutes, Dr. Highs first meeting with Marrazzo stretched to seven hours and was followed by many more meetings to determine the best approach for advancing CCMTs gene therapy discoveries. The result was a commitment of $50 million from CHOP to fund a new biotech company, Spark Therapeutics, to design, evaluate, and commercialize gene therapies for disorders that can lead to blindness, hemophilia, and neurodegenerative diseases. The company, like the hospital, is headquartered in Philadelphia.

CHOPs serving as the sole equity investor in Spark is definitely a novel financing model for early corporate activities to develop novel therapeutics, said Marrazzo, now president, CEO, and cofounder of Spark. Every situation is unique, and the situation should dictate the model.

Sparks situation was unusual because long before the companys official launch in late 2013, many assets were already in place, said Marrazzo, who uncovered them during his seven-hour conversation with Dr. High. It was like peeling back the layers of an onion, with each layer representing another asset, he said.

The assets included two clinical trials, a Phase 3 trial to treat a rare form of hereditary blindness, and a Phase 1/2 trial targeting hemophilia B, as well as staff members with gene therapy expertise in regulatory affairs, clinical research, and the manufacture of clinical grade vectors to transport genetic material into targeted cells.

Assembled at the center were world experts in gene therapy, said Marrazzo. CHOP had been incubating a biotech company within its four walls.

GENE THERAPY ASSETS UNDERVALUED Before investing $50 million to launch and operate Spark Therapeutics, CHOP officials considered but ruled out a licensing deal with an existing biopharm company or a start-up with VC funding. While we did have licensing deals on the table, that route would not have recognized the value of the asset in part because of the broad retrenchment that had occurred in the industry after the tragic 1999 death of Jesse Gelsinger in a gene therapy clinical trial, said Dr. High. Gelsinger died while participating in a clinical trial conducted by a University of Pennsylvania lab not connected to CCMT or CHOP.

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Ethics of Gene therapy

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Australian researchers are trying a novel way to boost the power of cochlear implants: They used the technology to beam gene therapy into the ears of deaf animals and found it improved hearing.

The approach isn't ready for human testing, but it's part of growing research into ways to let users of cochlear implants experience richer, more normal sound.

Normally, microscopic hair cells in a part of the inner ear called the cochlea detect vibrations and convert them to electrical impulses that the brain recognizes as sound. Hearing loss typically occurs as those hair cells are lost, whether from aging, exposure to loud noises or other factors.

Cochlear implants substitute for the missing hair cells, sending electrical impulses to directly activate auditory nerves in the brain. They've been implanted in more than 300,000 people. While highly successful, they don't restore hearing to normal, missing out on musical tone, for instance.

The idea behind the project: Perhaps a closer connection between the implant and the auditory nerves would improve hearing. Those nerves' bushlike endings can regrow if exposed to nerve-nourishing proteins called neurotrophins. Usually, the hair cells would provide those.

Researchers at Australia's University of New South Wales figured out a new way to deliver one of those growth factors.

They injected a growth factor-producing gene into the ears of deafened guinea pigs, animals commonly used as a model for human hearing. Then they adapted an electrode from a cochlear implant to beam in a few stronger-than-normal electrical pulses.

That made the membranes of nearby cells temporarily permeable, so the gene could slip inside. Those cells began producing the growth factor, which in turn stimulated regrowth of the nerve.

-- from wire reports

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Gene therapy may help hearing

According to the World Health Organization, more than 360 million people worldwide live with disabling hearing loss, and for many, devices such as hearing aids and cochlear implants allow them to maintain a normal life style. But what if a cochlear device could offer a biological solution that would enhance a patients experience?

For the first time, researchers at the University of New South Wales in Australia have used cochlear implants to regenerate auditory nerves through gene therapy, a process where therapeutic DNA is inserted into cells to treat a disease.

Cochlear implants work by converting sounds into electrical signals that are sent directly to the auditory nerve, bypassing the outer and middle ear. The process allows for significantly improved hearing, including the ability to maintain a phone conversation, but the sounds they provide for patients are monotone and robotic.

Ultimately, we hope that after further research, people who depend on cochlear implant devices will be able to enjoy a broader dynamic and tonal range of sound, which is particularly important for our sense of the auditory world around us and for music appreciation, says Professor Gary Housley, Director of the Translational Neuroscience Facility at UNSW Medicine.

In 1993 multiple labs discovered that mammals ears would have the ability to regenerate cells if triggered according to the National Organization for Hearing Research Foundation, but until now there hadnt been a safe or efficient way to deliver the necessary proteins to the cochlear area.

We think its possible that in the future this gene delivery would only add a few minutes to the implant procedure, says the papers first author, Jeremy Pinyon, whose PhD is based on this work. The surgeon who installs the device would inject the DNA solution into the cochlea and then fire electrical impulses to trigger the DNA transfer once the implant is inserted.

Gene therapy research has provided hope for a number of genetic disorders and diseases, including cancer, HIV and multiple sclerosis.

"Our work has implications far beyond hearing disorders, says co-author Associate Professor Matthias Klugmann, from the UNSW Translational Neuroscience Facility research team. Gene therapy has been suggested as a treatment concept even for devastating neurological conditions and our technology provides a novel platform for safe and efficient gene transfer into tissues as delicate as the brain.

The research was recently published in the journal Science Translational Medicine.

Professor Housley discusses the new gene delivery technique in the UNSW video below.

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Cochlear implant enhances patient experience through gene therapy

April 25, 2014

Alzheimer's, caused by toxic proteins that destroy brain cells, is the most common form of dementia. AFP/Relaxnews pic, April 25, 2014.Spanish scientists have for the first time used gene therapy to reverse memory loss in mice with Alzheimer's, an advance that could lead to new drugs to treat the disease, they said Wednesday.

The Autonomous University of Barcelona team injected a gene which causes the production of a protein that is blocked in patients with Alzheimer's into the hippocampus a region of the brian essential to memory processing in mice that were in the initial stages of the disease.

"The protein that was reinstated by the gene therapy triggers the signals needed to activate the genes involved in long-term memory consolidation," the university said in a statement.

Gene therapy involves transplanting genes into a patient's cells to correct an otherwise incurable disease caused by a failure of one or another gene.

The finding was published in The Journal of Neuroscience and it follows four years of research.

"The hope is that this study could lead to the development of pharmaceutical drugs that can activate these genes in humans and allow for the recovery of memory," the head of the research team, Carlos Saura, told AFP.

Alzheimer's, caused by toxic proteins that destroy brain cells, is the most common form of dementia.

Worldwide, 35.6 million people suffer from the fatal degenerative disease, which is currently incurable, and there are 7.7 million new cases every year, according to a 2012 report from the World Health Organisation.

In 2010 the total global societal cost of dementia was estimated to be US$604 billion, according to Alzheimer's Disease International, a federation of Alzheimer associations around the world. AFP/Relaxnews, April 25, 2014.

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Uniqure Gene Therapy Info.

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By Steven Reinberg HealthDay Reporter

WEDNESDAY, April 23, 2014 (HealthDay News) -- Australian researchers say that gene therapy may one day improve the hearing of people with cochlear implants, allowing them to appreciate music and hear in noisy environments.

In experiments with deaf guinea pigs, senior study author Gary Housley and colleagues found that inserting genes in the area of the cochlear implant and passing an electric charge through the implant stimulated the growth of cochlear cells.

"Our study found a [new] way to provide safe localized delivery of a gene to the cochlea, using the cochlear implant device itself. The gene acts as a nerve growth factor, which stimulates repair of the cochlear nerve," said Housley, a professor and director of the Translational Neuroscience Facility at the University of New South Wales, in Sydney.

The cochlear implant is surgically placed in the cochlea, in the inner ear. The implant works by using a line of small electrodes within the cochlea to selectively stimulate cochlear nerve fibers at different positions and enhancing different sounds, or frequencies, Housley explained.

"In the cochlea of a person with good hearing, sound vibrations are encoded by sensory cells, called 'hair cells,' which stimulate the cochlear nerve fibers," he said. "With hearing loss, the hair cells are lost, and without them the cochlear nerve fibers die and retract into the bone within the core of the cochlea."

This makes the job of the cochlear implant difficult as the amount of electrical current needed to stimulate the nerves is quite high, Housley added.

The gene therapy, which makes the cells close to the electrode produce the nerve growth factor, causes the nerve fibers to grow out to those cells -- and therefore to the electrodes, he explained. This means that much less current is needed, so more selective groups of nerve fibers can be stimulated.

"In the future, people with cochlear implants may get this gene therapy at the time of their implant, and the computer system -- which is part of the cochlea implant that converts sound to electrical pulses along the array of electrodes -- should be able to provide a better sound perception," Housley said.

Scientists note, however, that research with animals often fails to provide similar results in humans.

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Gene Therapy May Enhance Cochlear Implants, Animal Study Finds

The electrodes in a cochlear implant can be used to direct gene therapy and regrow neurons.

Growth factor: The cochlear nerve regenerates after gene therapy (top) versus the untreated cochlea from the same animal (bottom).

Researchers have demonstrated a new way to restore lost hearing: with a cochlear implant that helps the auditory nerve regenerate by delivering gene therapy.

The researchers behind the work are investigating whether electrode-triggered gene therapy could improve other machine-body connectionsfor example, the deep-brain stimulation probes that are used to treat Parkinsons disease, or retinal prosthetics.

More than 300,000 people worldwide have cochlear implants. The devices are implanted in patients who are profoundly deaf, having lost most or all of the ears hair cells, which detect sound waves through mechanical vibrations, and convert those vibrations into electrical signals that are picked up by neurons in the auditory nerve and passed along to the brain. Cochlear implants use up to 22 platinum electrodes to stimulate the auditory nerve; the devices make a tremendous difference for people but they restore only a fraction of normal hearing.

Cochlear implants are very effective for picking up speech, but they struggle to reproduce pitch, spectral range, and dynamics, says Gary Housley, a neuroscientist at the University of New South Wales in Sydney, Australia, who led development of the new implant.

Cyborg cavy: An Xray image shows the cochlear implant in the left ear of a guinea pig.

When the ears hair cells degrade and die, the associated neurons also degrade and shrink back into the cochlea. So theres a physical gap between these atrophied neurons and the electrodes in the cochlear implant. Improving the interface between nerves and electrodes should make it possible to use weaker electrical stimulation, opening up the possibility of stimulating multiple parts of the auditory nerve at once, using more electrodes, and improving the overall quality of sound.

Peptides called neurotrophins can encourage regeneration of the neurons in the auditory nerve. Housley used a common process, called electroporation, to cause pores to open up in cells, allowing DNA to get inside. It usually requires high voltages, and it hasnt found much clinical use, but Housley wanted to see whether the small, distributed electrodes of the cochlear implant could be used to achieve the effect.

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Cochlear Implant Also Uses Gene Therapy to Improve Hearing

A new study out of Australia has promising potential for patients across the globe who use cochlear implants. Photo by Flickr user ryanjpoole

A new study outlines how gene therapy could reverse hearing loss and deafness. This may be music to the ears of the roughly 300,000 patients across the globe that depend on cochlear implants to hear.

Australian researchers published their findings Wednesday in the journal Science Translational Medicine. By stimulating gene cells, which were injected into the ear canal with electrical impulses, chemically deafened guinea pigs were able to regrow auditory nerve cells.

The scientists used guinea pigs as test subjects because of the similarities between the ear canals of humans and guinea pigs. While the researchers noted just how effective cochlear implants have been to date in helping those with profound hearing loss, they also noted their limitations. They hope to overcome those limitations through their research.

People with cochlear implants do well with understanding speech, but their perception of pitch can be poor, so they often miss out on the joy of music, said the studys senior author Gary Housley, a professor of neuroscience at the University of South Wales.

The cochlea is a tiny seashell-shaped organ located in the inner ear. It is filled with groups of microscopic hair cells that move in response to vibrations, and then convert those vibrations into electrical impulses that are carried to the brain and interpreted as sound. In some peoples ears, either because of genetics, old age, poisoning or loud noises, those tiny hair cells are damaged or lost and scientists havent found a way found to regrow them yet. In certain patients who experience profound hearing loss, a cochlear implant with electrodes can help stimulate whatever nerve cells are left.

With this study, Housley and his colleagues encouraged the production of neurotrophins, small proteins that stimulate the growth and maintenance of the hair-like nerve cells. They injected small rings of DNA, called plasmids, into the inner ear of the guinea pigs. Then, they exposed the animals cochleas to electrical currents that mimicked the electrical impulses provided to human cochleas through cochlear implants. By doing so, the membranes of the guinea pigs cells became more permeable to the injected DNA. The result triggered the production of neurotrophins and thus, the regrowth of nerve cells. The researchers are hoping that, in human subjects, they can achieve similar results.

While the researchers were ecstatic over the results, some of their enthusiasm was tempered because in some guinea pigs, results began to taper after three to six weeks. They hope to continue studying the application of gene therapy going forward.

The development of electrode array gene delivery may not only improve the hearing of cochlear implant recipients but also find broader therapeutic applications, Housely said. [Gene therapy] could be used to treat a range of neurological disorders, from Parkinsons disease to psychiatric disorders.

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Gene therapy shows promise to help people regrow auditory nerve cells

A procedure that uses a series of electric jolts to inject lab-designed DNA molecules into cells of the inner ear may help to regrow auditory nerves in people with profound hearing loss, according to researchers.

In a paper published Wednesday in Science Translational Medicine, Australian researchers said they used tiny electrodes and gene therapy to regenerate nerve cells in chemically deafened guinea pigs.

The procedure, they said, may one day improve the functioning of human cochlear implants -- electronic devices that provide hearing sensations to the deaf.

"People with chochlear implants do well with understanding speech, but their perception of pitch can be poor, so they often miss out on the joy of music," said senior author Gary Housley, a professor of neuroscience at the University of South Wales.

"Ultimately we hope that after further research, people who depend on cochlear implant devices will be able to enjoy a broader dynamic and tonal range of sound," Housely said in a prepared statement.

Houseley and his colleagues studied the procedure on guinea pigs because the structure of their inner ear is similar to that of humans.

The cochlea is shaped like a snail's shell, and is filled with a multitude of tiny hair cells that move in response to sound vibrations. Those vibrations are then converted into electrical nerve impulses that are carried to the brain.

If the hair cells are lost or damaged due to age, genetics, chemical poisoning or loud noise, they will not grow back. In some people who are profoundly deaf, an electrode may be implanted within the cochlea that can stimulate some nerve cells.

While cochlear implants help roughly 300,000 patients throughout the world to detect and interpret speech, researchers believe they can be improved if nerve cells are encouraged to grow closer to the electrode. In this latest study, Housely and his colleagues set out to stimulate growth in spiral ganglion neurons in guinea pigs.

Study authors believed they could do this by causing inner ear cells to produce neurotrophins, proteins that control the development, maintenance and function of nerve cells.

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Hear me now? Gene therapy improves 'bionic ear' technology

Ricky Ashby Microbiology Podcast
This video is an assignment for Environmental Microbiology. It is a mock lecture on Gene Therapy and the utilization of viruses found in the environment as vectors. The supplemental reading...

By: Frederick Ashby

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Ricky Ashby Microbiology Podcast - Video

Jean Bennett on gene therapy as a treatment for blindness
Jean Bennett of the University of Pennsylvania on gene therapy as a treatment for blindness http://www.CharlieRose.com.

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Jean Bennett on gene therapy as a treatment for blindness - Video

Pronounce Medical Words Gene Therapy
This video shows you how to say Gene Therapy. How would you pronounce Gene Therapy?

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Pronounce Medical Words Gene Therapy - Video

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WASHINGTON Australian researchers are trying a novel way to boost the power of cochlear implants: They beamed gene therapy into the ears of deaf animals and found the combination improved hearing. The approach reported Wednesday isnt ready for human testing, but its part of growing research into ways to let users of cochlear implants experience richer, more normal sound.

Normally, microscopic hair cells in the cochlea detect vibrations and convert them to electrical impulses that the brain recognizes as sound. Hearing loss typically occurs as those hair cells are lost, whether from aging, exposure to loud noises or other factors.

Cochlear implants substitute for the missing hair cells, sending electrical impulses to directly activate auditory nerves in the brain. Theyve been implanted in more than 300,000 people but, while highly successful, they dont restore hearing to normal, missing out on musical tone, for instance.

The idea behind the project was a closer connection between the implant and the auditory nerves, whose bush-like endings can regrow if exposed to nerve-nourishing proteins called neurotrophins, usually provided by the hair cells.

Researchers at Australias University of New South Wales figured out how to deliver one of those growth factors. They injected a growth factor-producing gene into the ears of deaf guinea pigs, animals commonly used as a model for human hearing. Then they adapted an electrode from a cochlear implant to beam in stronger-than-normal electrical pulses. That made the membranes of nearby cells temporarily permeable, so the gene could slip inside. Those cells began producing the growth factor, which in turn stimulated regrowth of the nerve fibers closing some of the space between the nerves and the cochlear implant, the team reported in the journal Science Translational Medicine. The animals still needed a cochlear implant to detect sound but those given the gene therapy had twice the improvement, they concluded. Senior author Gary Housley estimated small studies in people could begin in two or three years.

Thats a really clever way of delivering the nerve booster, said Stanford University otolaryngology professor Stefan Heller, who wasnt involved with the Australian work. But Heller cautioned that its an early first step and its not clear how long the extra improvement would last or if it really would spur richer sound. He said other groups are exploring such approaches as drug coatings for implants; Hellers own research aims to regrow hair cells.

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Gene therapy may boost hearing, study finds

The performance of cochlear implants has been improved with the use of gene therapy, suggesting a new avenue for developing better hearing aids

A computer-tomography scan shows a deaf guinea pig's skull and cochlear implant. Credit:UNSW Australia Biological Resources Imaging Laboratory and National Imaging Facility of Australia

Gene therapy delivered to the inner ear can help shrivelled auditory nerves to regrow and in turn, improve bionic ear technology, researchers report today inScience Translational Medicine. The work, conducted in guinea pigs, suggests a possible avenue for developing a new generation of hearing prosthetics that more closely mimics the richness and acuity of natural hearing.

Sound travels from its source to ears, and eventually to the brain, through a chain of biological translations that convert air vibrations to nerve impulses. When hearing loss occurs, its usually because crucial links near the end of this chain between the ears cochlear cells and the auditory nerve are destroyed. Cochlear implants are designed to bridge this missing link in people with profound deafness by implanting an array of tiny electrodes that stimulate the auditory nerve.

Although cochlear implants often work well in quiet situations, people who have them still struggle to understand music or follow conversations amid background noise. After long-term hearing loss, the ends of the auditory nerve bundles are often frayed and withered, so the electrode array implanted in the cochlea must blast a broad, strong signal to try to make a connection, instead of stimulating a more precise array of neurons corresponding to particular frequencies. The result is an aural smearing that obliterates fine resolution of sound, akin to forcing a piano player to wear snow mittens or a portrait artist to use finger paints.

To try to repair auditory nerve endings and help cochlear implants to send a sharper signal to the brain, researchers turned to gene therapy. Their method took advantage of the electrical impulses delivered by the cochlear-implant hardware, rather than viruses often used to carry genetic material, to temporarily turn inner-ear cells porous. This allowed DNA to slip in, says lead author Jeremy Pinyon, an auditory scientist at the University of New South Wales in Sydney, Australia.

Pinyon and his colleagues were able to deliver a gene encoding neurotrophin, a protein that stimulates nerve growth, to the inner-ear cells of deaf guinea pigs. After injecting the cells with a solution of DNA, they sent a handful of 20-volt pulses through the cochlear-implant electrode arrays. The cells started producing neurotrophin, and the auditory nerve began to regenerate and reach out for the cochlea once again. The researchers found that the treated animals could use their implants with a sharper, more refined signal, although they did not compare the deaf guinea pigs to those with normal hearing. The work was partially funded by Cochlear, a cochlear-implant maker based in Sydney.

Regenerating nerves and cells in the inner ear to boost cochlear implant performance has long been a goal of auditory scientists. This clever approach is the most promising to date, says Gerald Loeb, a neural prosthetics researcher at the University of Southern California in Los Angeles, who helped to develop the original cochlear implant. Although clinical applications are still far in the future, the ability to deliver genes to specific areas in the cochlea will probably reduce regulatory obstacles, he says. But it is unclear why cochlear implants help some patients much more than others, so whether this gene therapy translates into actual clinical benefit is still unclear.

Listening to sounds is an intricate process, and a cochlear implant cannot simulate such complexity, says Edward Overstreet, an engineer at Oticon, a hearing technology company in Somerset, New Jersey. So it is not clear that simply sharpening the electrodes signal will help a user to hear sounds in a more natural way. We would probably need a leap in cochlear-implant electrode array technology to make this meaningful in terms of patient outcomes, he says.

If the method works well in humans, the authors say, it might help profoundly deaf people enjoy music and follow conversations in restaurants. And it might also enhance a newer type of hearing technology: hybrid electro-acoustic implants, which are designed to help people who have only partial hearing loss. The gene therapy might work to keep residual hearing intact and allow the implants to replace only what is missing, creating a blend of natural and electric hearing.

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Bionic Ears Boosted by Gene Therapy and Regrown Nerves

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Washington, April 24 : Researchers have identified the protein which prevents memory consolidation when blocked.

Researchers from the Institute of Neuroscience at the Universitat Autonoma de Barcelona have discovered the cellular mechanism involved in memory consolidation and were able to develop a gene therapy which reverses the loss of memory in mice models with initial stages of Alzheimer's disease.

The therapy consists in injecting into the hippocampus - a region of the brain essential to memory processing - a gene which causes the production of a protein blocked in patients with Alzheimer's, the "Crtc1" (CREB regulated transcription coactivator-1). The protein restored through gene therapy gives way to the signals needed to activate the genes involved in long-term memory consolidation.

To identify this protein, researchers compared gene expression in the hippocampus of healthy control mice with that of transgenic mice which had developed the disease.

Through DNA microchips, they identified the genes ("transcriptome") and the proteins ("proteome") which expressed themselves in each of the mice in different phases of the disease.

Researchers observed that the set of genes involved in memory consolidation coincided with the genes regulating Crtc1, a protein which also controls genes related to the metabolism of glucose and to cancer. The alteration of this group of genes could cause memory loss in the initial stages of Alzheimer's disease.

In persons with the disease, the formation of amyloid plaque aggregates, a process known to cause the onset of Alzheimer's disease, prevents the Crtc1 protein from functioning correctly.

"When the Crtc1 protein is altered, the genes responsible for the synapsis or connections between neurons in the hippocampus cannot be activated and the individual cannot perform memory tasks correctly", explains Carlos Saura, researcher of the UAB Institute of Neuroscience and head of the research.

The research has been published in The Journal of Neuroscience.

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Gene therapy helps reverse loss of memory in mice suffering from Alzheimer's

Australian researchers are trying a novel way to boost the power of cochlear implants: They used the technology to beam gene therapy into the ears of deaf animals and found the combination improved hearing.

The approach reported Wednesday isn't ready for human testing, but it's part of growing research into ways to let users of cochlear implants experience richer, more normal sound.

Normally, microscopic hair cells in a part of the inner ear called the cochlea detect vibrations and convert them to electrical impulses that the brain recognizes as sound. Hearing loss typically occurs as those hair cells are lost, whether from aging, exposure to loud noises or other factors.

Cochlear implants substitute for the missing hair cells, sending electrical impulses to directly activate auditory nerves in the brain. They've been implanted in more than 300,000 people. While highly successful, they don't restore hearing to normal, missing out on musical tone, for instance.

The idea behind the project: Perhaps a closer connection between the implant and the auditory nerves would improve hearing. Those nerves' bush-like endings can regrow if exposed to nerve-nourishing proteins called neurotrophins. Usually, the hair cells would provide those.

Researchers at Australia's University of New South Wales figured out a new way to deliver one of those growth factors.

They injected a growth factor-producing gene into the ears of deafened guinea pigs, animals commonly used as a model for human hearing. Then they adapted an electrode from a cochlear implant to beam in a few stronger-than-normal electrical pulses.

That made the membranes of nearby cells temporarily permeable, so the gene could slip inside. Those cells began producing the growth factor, which in turn stimulated regrowth of the nerve fibers - closing some of the space between the nerves and the cochlear implant, the team reported in the journal Science Translational Medicine.

The animals still needed a cochlear implant to detect sound - but those given the gene therapy had twice the improvement, they concluded.

Senior author Gary Housley estimated small studies in people could begin in two or three years.

View post:

Gene therapy may boost power of cochlear implants, study says

Australian researchers are trying a novel way to boost the power of cochlear implants: They used the technology to beam gene therapy into the ears of deaf animals and found the combination improved hearing.

The approach reported Wednesday isn't ready for human testing, but it's part of growing research into ways to let users of cochlear implants experience richer, more normal sound.

Normally, microscopic hair cells in a part of the inner ear called the cochlea detect vibrations and convert them to electrical impulses that the brain recognizes as sound. Hearing loss typically occurs as those hair cells are lost, whether from aging, exposure to loud noises or other factors.

Cochlear implants substitute for the missing hair cells, sending electrical impulses to directly activate auditory nerves in the brain. They've been implanted in more than 300,000 people. While highly successful, they don't restore hearing to normal, missing out on musical tone, for instance.

The idea behind the project: Perhaps a closer connection between the implant and the auditory nerves would improve hearing. Those nerves' bush-like endings can regrow if exposed to nerve-nourishing proteins called neurotrophins. Usually, the hair cells would provide those.

Researchers at Australia's University of New South Wales figured out a new way to deliver one of those growth factors.

They injected a growth factor-producing gene into the ears of deafened guinea pigs, animals commonly used as a model for human hearing. Then they adapted an electrode from a cochlear implant to beam in a few stronger-than-normal electrical pulses.

That made the membranes of nearby cells temporarily permeable, so the gene could slip inside. Those cells began producing the growth factor, which in turn stimulated regrowth of the nerve fibers closing some of the space between the nerves and the cochlear implant, the team reported in the journal Science Translational Medicine.

The animals still needed a cochlear implant to detect sound but those given the gene therapy had twice the improvement, they concluded.

Senior author Gary Housley estimated small studies in people could begin in two or three years.

Original post:

Study: Gene Therapy May Boost Cochlear Implants

The gene therapy study is hoped to lead to the development of new drugs to treat the incurable disease

GENE THERAPY. Spanish scientists injected a gene which causes the production of a protein that is blocked in patients with Alzheimers, into the hippocampus in mice that were in the initial stages of the disease.

MADRID, Spain Spanish scientists have for the first time used gene therapy to reverse memory loss in mice with Alzheimer's, an advance that could lead to new drugs to treat the disease, they said Wednesday, April 23.

The Autonomous University of Barcelona team injected a gene which causes the production of a protein that is blocked in patients with Alzheimer's into the hippocampus a region of the brian essential to memory processing in mice that were in the initial stages of the disease.

"The protein that was reinstated by the gene therapy triggers the signals needed to activate the genes involved in long-term memory consolidation," the university said in a statement.

Gene therapy involves transplanting genes into a patient's cells to correct an otherwise incurable disease caused by a failure of one or another gene.

The finding was published in The Journal of Neuroscience and it follows 4 years of research.

"The hope is that this study could lead to the development of pharmaceutical drugs that can activate these genes in humans and allow for the recovery of memory," the head of the research team, Carlos Saura, told Agence France-Presse.

Alzheimer's, caused by toxic proteins that destroy brain cells, is the most common form of dementia.

Worldwide, 35.6 million people suffer from the fatal degenerative disease, which is currently incurable, and there are 7.7 million new cases every year, according to a 2012 report from the World Health Organization.

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Scientists reverse memory loss in mice with Alzheimer's

Cochlear implants have restored hearing to many deaf people, but they havent advanced much since they were unveiled in the 1970s. That may be set to change with an exciting new advance, not in the technology of the device itself, but rather in using gene therapy to increase the devices effectiveness. Today researchers announced that theyve been able to restore tonal hearing in guinea pigs with the new method of gene delivery.

Cochlear implants, or bionic ears, work by stimulating the auditory nerve to restore a rudimentary kind of hearing. This works pretty well, butthe gap between the electrodes and the degenerating nerve is pretty big, which makes communication difficult. Andeven the state-of-the-art implants only have 22 electrodes, enabling them to hear 22 different tones. They cant, for example, distinguish between the soft buzz of a clarinet and the shrill sound of a flute.

Teams of researchers have tried to improve upon the implants over the last decade by trying to focus the electrical currents more narrowly, to stimulate a smaller, more pitch-specific area of the nerve, or to use drugs that improve the communication between the electrodes and the neurons. But this new method, reported today in Science Translational Medicine, has a distinct advantage: it actually encouraged the regrowth of the auditory nerve. This decreased the gap between the nerve and the cochlear implant, and improved communication between the two.

Image credit: Science Translational Medicine

The team implanted bionic ears indeaf guinea pigs, whose auditory systems are very similar to humans. With the device, then, they delivered DNA that coded for a protein called brain-derived neruotrophic factor (BDNF), which encourages nerves to grow. The DNA was taken up by cells in the cochlea and, after two weeks, the nerves had grown significantly toward the electrodes. When the guinea pigs hearing was tested they found that animals that were once completely deaf had their hearing restored to almost normal levels.

Its unclear, however, whether the treatment will work long-term: neuron production in the guinea pigs dropped off six weeks after the gene therapy. Researchers are also unsure whether tones heard after this treatment accurately reflect how they sound with normal hearing.

The technique is very close to being ready for human trials, where some of these questions should be answered. If it proves successful in clinical trials, the technique of combining gene therapy with device could also be used for other implants like retinal prosthesis and deep brain stimulation.

Top image credit:Elizabeth Hoffmann/Shutterstock

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Cochlear Implant Plus Gene Therapy Could Restore Hearing to the Deaf