Ruben Hernandez: "Recent developments in the gene therapy of solid tumours"
Educational Day* at ESGCT Conference in Madrid. Rubn Hernndez, PI in Cancer Gene Therapy at Universita de Nvarra (University of Navarra, Spain) gives a le...
By: European Society for Gene and Cell Therapy
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Ruben Hernandez: "Recent developments in the gene therapy of solid tumours" - Video
Personalized Gene Therapy Reducing HIV In Some Patients
For the first-time ever, doctors at the University of Pennsylvania have successfully used personalized gene therapy in a dozen patients with HIV, knocking the virus down to the point where...
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Personalized Gene Therapy Reducing HIV In Some Patients - Video
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Newswise UCLA stem cell researchers have pioneered a stem cell gene therapy cure for children born with adenosine deaminase (ADA)-deficient severe combined immunodeficiency (SCID), often called Bubble Baby disease, a life-threatening condition that if left untreated can be fatal within the first year of life.
The groundbreaking treatment was developed by renowned stem cell researcher and UCLA Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research member Dr. Donald Kohn, whose breakthrough was developed over three decades of research to create a gene therapy that safely restores immune systems in children with ADA-deficient SCID using the patients own cells with no side effects.
To date, 18 children with SCID have been cured of the disease after receiving the stem cell gene therapy in clinical trials at UCLA and the National Institutes of Health.
All of the children with SCID that I have treated in these stem cell clinical trials would have died in a year or less without this gene therapy, instead they are all thriving with fully functioning immune systems said Kohn, a professor of pediatrics and of microbiology, immunology and molecular genetics in Life Sciences.
To protect children born with SCID they are kept in isolation, in controlled environments because without an immune system they are extremely vulnerable to illness and infection that could be lethal.
Other current options for treating ADA-deficient SCID are not always optimal or feasible for many children, said Kohn. We can now, for the first time, offer these children and their families a cure, and the chance to live a full healthy life.
Defeating ADA-Deficient SCID: A Game-Changing Approach
Children born with SCID, an inherited immunodeficiency, are generally diagnosed at about six months. They are extremely vulnerable to infectious diseases, and in a child with ADA-deficient SCID even the common cold can prove fatal. The disease causes cells to not create an enzyme called ADA, which is critical for production of the healthy white blood cells that drive a normal, fully-functioning immune system. About 15 percent of all SCID patients are ADA-deficient.
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UCLA Stem Cell Researcher Pioneers Gene Therapy Cure for Children with "Bubble Baby" Disease
The gene therapy that has seemingly cured a 2-year-old girl could be used to treat other diseases, like Sickle Cell Anemia.
UCLA DR. DONALD KHON has perfected the treatment that has now affected the lives of over a dozen families, including the parents of Evangelina, who was born with the deadly SCID. The immune system disease is known by its common name, Bubble Baby disease, because the children have to live in bubbles, since their immune systems do not function. Most die within the first year of life, unless they can get and survive a bone marrow transplant.
Evangelina's twin sister, Annabella, was not a bone marrow transplant. So Dr Kohn included the sick twin in his study, transfering the patient's own bone marrow to the child. At two, her immune system is working well.
It's a difficult and time consuming process, but it seems to work. The same procedure could work with other blood diseases that are curable with bone marrow transfusions. That includes sickle cell anemia, which will be the focus of the next trial.
The implications are huge, as there are 30 or 40 diseases where the treatment could work.
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Bubble Baby: Gene Therapy Cured 2-year-old Girl Of Deadly SCID At UCLA
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WESTWOOD (CBSLA.com) Doctors say a groundbreaking stem cell therapy treatment out of UCLA may have cured Bubble Baby syndrome once and for all.
KNX 1070s Brian Ping reports Dr. Donald Kohn has perfected a gene therapy that has now cured 18 children born without an immune system, known as ADA-deficient severe combined immunodeficiency (SCID).
Only weeks after giving birth to fraternal twins in 2012, Alysia and Christian Padilla-Vaccaro found out their daughter Evangelinas immune system was so deficient that she could have no exposure to the outside world.
After enrolling their daughter in Dr. Donald Kohns revolutionary stem cell gene therapy treatment which was nearly three decades in the making doctors extracted stem cells from the bone marrow in Evangelinas hip, then used a modified mouse virus to correct her faulty gene before replacing the marrow.
You hear the words mouse virus and you want to run the other way, said mom Alysia. But they modify it so that its teaching it to do something that they want it to do, which is put something in there that was missing.
Evangelinas new immune system developed without side effects and she is now living a healthy normal life.
Her mother Alysia said while the process was difficult for any mom to go through, it was all worth it.
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Gene Therapy- Channel 4 news
Anchor Josh Barnette interviews Sydney Brooks and Hunter White and talks about their groundbreaking research in the field of genetics.
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Dr Zwerling on Gene Therapy
Ian #39;s school project.
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Gene Therapy: Choroideremia
It #39;s a form of blindness that some call especially cruel. People with choroideremia are born with perfect vision and then begin to lose their sight, sometimes as kids or teens.
By: NewsChannel 5
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Gene therapy restores eyesight
Restoring sight to people suffering from a rare kind of eye disease - researchers at the University of Oxford have just published the results of a trial, whi...
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Cartilage Regeneration
Christopher H. Evans, Ph.D., Director of Rehabilitation Medicine Research Center, Mayo Clinic PM R, discusses his lecture at AAPM R 2014 on the damage done to cartilage from trauma and arthritis.
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KANSAS CITY,Kan. Restoring hearing with a drug is the goal of a metro researcher. Now hes begun the first study in humans of a gene therapy that could help people hear again.
Rob Gerk was ready to become a medical pioneer at the University of Kansas Hospital.
Just wanta get it over with. All the testing and stuff like that kinda makes it build up to the big moment, said Gerk.
The Denver man lost almost all of his hearing as toddler when he had meningitis. On October 28th, he became the first person in the world to get a drug infused in the inner ear to see if it can restore hearing. The drug is made up of a virus and a gene.
We take a virus that normally causes cold-like symptoms and we take all of the guts of the virus that make it infectious out. And we replace it with the gene of interest, said Dr. Hinrich Staecker.
Dr. Staecker has been working on the gene therapy for 17 years. In mice, it restored hearing.
Through the regeneration of little cells that detect sound vibrations in the inner ear, said the researcher.
The therapy triggered the growth of sensory receptors.
The initial human study with 21 patients will evaluate safety. Each patient will have one ear treated. Potential risks include more hearing loss or dizziness. Dr. Staecker says it should be known within six to eight weeks whether the therapy is restoring hearing, although results may not be made public for a year.
Study participants must have severe hearing loss resulting from noise, medications or some diseases.
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Cancer Gene Therapy by Deirdre Creegan group 9
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Linda Carroll TODAY contributor
6 hours ago
The parents of Eliza ONeill, the little girl with an incurable and deadly brain disorder, are making one final desperate push to raise funds for the clinical trial that will test a gene therapy for the diseaseand perhaps offer hope for Eliza.
Courtesy of Glenn ONeill
The ONeills have already raised $1.3 million through a viral video and hope that by Elizas fifth birthday on Sunday theyll hit their $1.8 million goal.
While there is no guarantee that Eliza will be among those chosen to participate in the trial, which could start sometime in the middle of 2015, its organizers say that at this point there is no reason that she wouldnt qualify.
Over the past year, the ONeills have tried to focus on aspects of life over which they have some control, such as helping to make the trial a reality and keeping their daughter as healthy as possible. Sometimes, they even allow themselves to think about what life might be like if Eliza was helped by the experimental medication.
Our hopes are for Eliza to have a life, and a good life, said her dad, Glenn ONeill. I sometimes let my mind go to places of future birthdays and her dating and getting married.
Stacey Quattlebaum
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A birthday wish: 'Saving Eliza' gets final push for medical trial
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13-Nov-2014
Contact: Service de presse AFM-Tlthon gmonfort@afm-telethon.fr AFM-Tlthon @AfmPresse
Duchenne muscular dystrophy is the most common neuromuscular disease of children (affecting 1 boy in 3500-5000 births). It is caused by a genetic defect in the DMD gene residing on the X chromosome, which results in the absence of the dystrophin protein essential to the proper functioning of muscles.
The treatment being developed by researchers at Atlantic Gene Therapies, Gnthon and the Institute of Myology, is based on the use of an AAV vector (Adeno Associated Virus) carrying a transgene for the skipping of a specific exon which allows functional dystrophin production in the muscle of the patient.
Safety, efficacy and stability of the treatment in dogs
In GRMD (Golden Retriever Muscular Dystrophy) dogs the treatment aimed at skipping exons 6, 7 and 8 of the dystrophin gene. The product was given by loco-regional administration in the forelegs of 18 dogs who were followed for 3.5 months after injection. It was well tolerated by all treated dogs; no immune response against the synthesized dystrophin was observed. Exon skipping resulted in high levels of expression of dystrophin in the treated muscles. The results of this treatment also indicate that, once injected into the muscle tissue a prolonged and stable effect is produced over the observation time of the study and, unlike antisense oligonucleotides already used clinically for exon skipping, it does not need to be re- administered regularly. The synthesis of "new" dystrophin is dependent on the dose of vector injected: the higher the dose, the greater the exon skipping is effective. Muscle strength also increases with dose. 80% of muscle fibers expressed the "new" dystrophin at the highest dose. This is a very encouraging result because a minimum of 40% of dystrophin in muscle fibers is believed to be necessary for the muscle force to be significantly improved.
A phase I/II clinical trial phase
These results open the way for a phase I / II clinical trial by loco-regional administration in the upper limb of non-ambulatory Duchenne muscular dystrophy patients which are amenable to treatment by the specific skipping of exon 53. The regulatory toxicology and biodistribution studies have just ended and the filing of an application with regulatory authorities is planned for 2015.
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Effectiveness of innovative gene therapy treatment demonstrated in canine model of DMD
Gene Therapy for Inherited Disorders - Gerard Wagemaker
Gene therapy will be of great significance for patients with hereditary disorders and society at large, says Gerard Wagemaker EU project: HIGHLIGHT (http://www.youris.com/Health/Video_Interviews/G...
By: European health innovation transfer network
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Gene Therapy for Inherited Disorders - Gerard Wagemaker - Video
Gene Therapy for Treating Cancer
Gene Therapy for Treating Cancer.
By: WTNH News8
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[1.09] How to use medical items
1) Pick up pills/gene therapy/bandages/med kit 2) Open your inventory wheel by pressing 1, and click on the item 3) Once you see a pair of hands, tap your left mouse button You can still shove...
By: Riley3212
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When Glenn ONeill and his wife, Cara, learned about a gene therapy treatment that could save their daughters life, they started a foundation and set off to raise the $2.3 million it would take to fund the manufacturing and clinical trial costs for the drug.
Today, thanks to about 26,000 donors from 70 different countries, theyre just $530,000 away from their goal.
Their daughter, 4-year-old Eliza ONeill, suffers from Sanfilippo Syndrome-Type A, a disease that causes children to lack an enzyme necessary for normal cellular function. The disease eventually causes a lethal buildup of a toxic material called heparin sulfate, leading to learning disabilities and behavioral problems. The disease is seen in 1 in 70,000 births, and most children born with with Sanfillipo Syndrome-Type A die by the time they are teenagers.
While there is currently no cure or treatment for the disease, researchers at Nationwide Childrens Hospital in Columbus, Ohio have found a potential gene therapy that, in a study, successfully rid mice of heparin sulfate buildup.
When Cara spoke to lead researcher Dr. Haiyan Fu, principal investigator at the Center for Gene Therapy at Nationwide, and learned about the potential treatment, the family began raising money to fund it.
That was the first glimmer of hope that I got in all of this, Cara ONeill told FoxNews.com.
A couple of months after the diagnosis in July 2013, they began a slew of traditional fundraising efforts, from bake sales and 5Ks, to parties and a golf tournament. At the end of 2013, they started the Cure Sanfilippo Foundation and have launched SavingEliza.com through the fundraising site GoFundMe.com. In April 2014, a videographer made a free video for the family that went viral, and the family started a social media campaign called Sing Two Lines, similar to the ALS Ice Bucket Challenge, where people would challenge others to sing two lines of their favorite song. Actress Andie MacDowell and members of the band Gloriana participated.
The ONeills have raised $1.7 million since December and have been able to fund the manufacturing costs of the drug as well as some of the preclinical work. Their goal is to meet the $2.3 million mark by Elizas fifth birthday, Nov. 16.
Weve made it a point not to ask previous donors to donate again, Glenn ONeill told FoxNews.com, but at some point we knew it was going to come to crunch time. At this point, with [Elizas] birthday coming up, we really need to spend more time with Eliza, and focusing on her and keeping her learning at the top of the game.
Researchers at Nationwide have proposed what they feel is a sufficient toxicology plan to move forward with the clinical trial, and they are currently waiting for the Food and Drug Administrations (FDA) comments.
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Saving Eliza: Family raising money for Sanfilippo Syndrome drug $530K away from goal
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ELK GROVE VILLAGE, Ill., Nov. 5, 2014 /PRNewswire-USNewswire/ -- A manuscript from the laboratory of Dr. Brian Kaspar of Nationwide Childrens Hospital was recently published in the journal Molecular Therapy. The paper, Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA-a dose response study in mice and nonhuman primates is the first publication resulting from a groundbreaking collaboration between Cure SMA, the National Institute of Neurological Disorders and Stroke (NINDS), and Dr. Kaspar.
Beginning in 2010, Cure SMA made a series of grants to Dr. Kaspar to study gene therapy, also called gene transfer. Spinal muscular atrophy (SMA) is caused by a mutation in the survival motor neuron 1 gene (SMN1). Because of this mutation, the individual does not produce enough survival motor neuron (SMN) protein. Gene transfer may increase SMN levels by using a virus, called a vector, to deliver the SMN1 gene to affected cells. Kaspars laboratory discovered that Adeno-associated virus serotype 9 (AAV9) had the unique ability to cross the blood brain barrier and the Blood-Cerebrospinal Fluid Barrier (CSF).
Dr. Kaspar and his team have studied two approaches for SMA: an injection into a vein, a process known as systemic delivery which is currently in Phase I/2 clinical trials, and delivery directly into the cerebrospinal spinal fluid (CSF), a process known as CSF-delivered gene therapy.
Using the data generated with Cure SMA funding on the CSF-delivery of the drug, Dr. Kaspar and his team were able to secure a $4 million grant from NINDS in 2013, to develop this delivery approach for human clinical trials in SMA.
Development of therapies requires collaboration of academics, advocacy, industry, and government-no single party has the resources to do this alone. The collaboration between Dr. Brian Kaspar, Cure SMA, and the NIH is an exciting model in leveraging resources and expertise in the hope of accelerating therapy development for SMA, said Dr. John Porter, PhD, Program Director at the National Institute of Neurological Disorders and Stroke.
The results of this research collaboration are the subject of Dr. Kaspars latest article. Using a one-time delivery of the AAV9 carrying the human SMN gene, the researchers found SMA animals, which typically die at 15 days of age, surpassed 280 days median survival, with many animals surviving past 400 days. This is a remarkable extension in survival with normal motor function. Furthermore, the group tested this delivery approach in larger species and found significant targeting of motor neurons throughout the brain and spinal cord.
Dr. Kaspar stated, We are very pleased with the results of this study and are working diligently to advance a CSF route of delivery to human clinical trials for SMA. We are grateful for the support from Cure SMA and NINDS. We stand at an exciting juncture in SMA research and clinical translation with strong will to see effective therapies for all those with SMA.
We are excited to see expansion of the gene therapeutic program and the potential to advance this route of delivery to patients with SMA. The latest results supports further development of a CSF-delivered gene therapy treatment, said Jill Jarecki, PhD, Cure SMAs research director.
Current Clinical Trials for SMA Gene Therapy
The technology for both systemic and CSF-delivered gene therapy has been licensed to AveXis, a clinical stage biotechnology company. AveXis and Nationwide Childrens Hospital are currently collaborating on a Phase I/2 clinical trial testing the systemic delivery method in infants with SMA. The trial (NCT02122952) opened for enrollment in Columbus, Ohio in April 2014 and is currently recruiting in the dose-escalation phase of the trial.
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CSF-Delivered Gene Therapy Shows Promise for Spinal Muscular Atrophy