Dr. Jean Bennett on Gene Therapy
http://www.fightblindness.org | Jean Bennett M.D., Ph.D., of the University of Pennsylvania School Of Medicine, discusses how gene therapy is making waves as a prom...
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Category Archives: Gene Therapy
Dr. Bill Hauswirth on Gene Therapy – Video
Dr. Bill Hauswirth on Gene Therapy
http://www.fightblindness.org | William Hauswirth, Ph.D., of the University of Florida College of Medicine, discusses how gene therapy is making waves as a promisin...
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Dr. Jeff Chulay on Gene Therapy – Video
Dr. Jeff Chulay on Gene Therapy
http://www.fightblindness.org | Jeff Chulay, M.D., of the Applied Genetics Technologies Corporation, discusses how gene therapy is making waves as a promising area ...
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Alliance for Cancer Gene Therapy to Host Inaugural Foundation Symposium at the 16th Annual American Society of Gene …
STAMFORD, Conn.--(BUSINESS WIRE)--
Alliance for Cancer Gene Therapy, Inc. (ACGT), http://www.acgtfoundation.org, https://twitter.com/acgtfoundation, and https://www.facebook.com/acgtfoundation, today announced that it will be hosting an inaugural Foundation Symposium Gene and Cell Therapy For Cancer on Thursday, May 16, from 6:00 p.m. to 8:00 p.m., Ballroom B, at the 16th Annual American Society of Gene and Cell Therapy Meeting, May 15-18, Salt Palace Convention Center, in Salt Lake City, Utah. The Symposium will be co-chaired by Xandra O. Breakefield, PhD, Massachusetts General Hospital, and Savio L.C. Woo, PhD, Mount Sinai School of Medicine.
This ACGT-sponsored Symposium will present state-of-the-art lectures by leading investigators in four exceptionally promising areas of scientific research and translational development in gene and cell therapies for cancer. Over the past decade, the power of gene and cell therapies has been harnessed into novel bio-therapeutics for the treatment of many different cancers that are both effective and safe.
We are honored to partner with ASGCT and to feature some of the worlds most renowned scientists, physicians and researchers in the field of cell and gene therapy, said Barbara Netter, ACGT President and Co-Founder.
Speakers and Topics Include:
ACGT is the only not-for-profit in the U.S. dedicated solely to cancer cell and gene therapies for all types of cancer. 100% of contributions go directly to research. ACGT has funded 41 grants from public donations since its founding in 2001 totaling almost $25 million for both basic research and clinical translation. Seventeen ACGT funded research projects have been approved for human clinical trials, 11 of which are underway. To donate, please visit http://www.acgtfoundation.org or call 203.358.8000.
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AAV-Mediated Inclusion Formation as a Novel Gene Therapy Strategy for ALS – Video
AAV-Mediated Inclusion Formation as a Novel Gene Therapy Strategy for ALS
Ole Isacson, Ph.D., McLean Hospital. Dr. Isacson is a CDMRP-funded investigator supported by the DoD Amyotrophic Lateral Sclerosis Research Program (ALSRP).
By: CDMRP
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AAV-Mediated Inclusion Formation as a Novel Gene Therapy Strategy for ALS - Video
Gene Therapy Final – Video
Research and Markets: Gene Therapy – Technologies, Markets and Companies – Updated 2013 Report
DUBLIN--(BUSINESS WIRE)--
Research and Markets (http://www.researchandmarkets.com/research/lvclfm/gene_therapy) has announced the addition of Jain PharmaBiotech's new report "Gene Therapy - Technologies, Markets and Companies" to their offering.
Gene therapy can be broadly defined as the transfer of defined genetic material to specific target cells of a patient for the ultimate purpose of preventing or altering a particular disease state. Genes and DNA are now being introduced without the use of vectors and various techniques are being used to modify the function of genes in vivo without gene transfer. If one adds to this the cell therapy particularly with use of genetically modified cells, the scope of gene therapy becomes much broader. Gene therapy can now combined with antisense techniques such as RNA interference (RNAi), further increasing the therapeutic applications. This report takes broad overview of gene therapy and is the most up-to-date presentation from the author on this topic built-up from a series of gene therapy report written by him during the past decade including a textbook of gene therapy and a book on gene therapy companies. This report describes the setbacks of gene therapy and renewed interest in the topic
Gene therapy technologies are described in detail including viral vectors, nonviral vectors and cell therapy with genetically modified vectors. Gene therapy is an excellent method of drug delivery and various routes of administration as well as targeted gene therapy are described. There is an introduction to technologies for gene suppression as well as molecular diagnostics to detect and monitor gene expression.
Clinical applications of gene therapy are extensive and cover most systems and their disorders. Full chapters are devoted to genetic syndromes, cancer, cardiovascular diseases, neurological disorders and viral infections with emphasis on AIDS. Applications of gene therapy in veterinary medicine, particularly for treating cats and dogs, are included.
Research and development is in progress in both the academic and the industrial sectors. The National Institutes of Health (NIH) of the US is playing an important part. As of 2012, over 2030 clinical trials have been completed, are ongoing or have been approved worldwide.A breakdown of these trials is shown according to the areas of application.
The voluminous literature on gene therapy was reviewed and selected 750 references are appended in the bibliography.The references are constantly updated. The text is supplemented with 73 tables and 15 figures.
Profiles of 181 companies involved in developing gene therapy are presented along with 204 collaborations. There were only 44 companies involved in this area in 1995. In spite of some failures and mergers, the number of companies has increased more than 4-fold within a decade. These companies have been followed up since they were the topic of a book on gene therapy companies by the author of this report. John Wiley & Sons published the book in 2000 and from 2001 to 2003, updated versions of these companies (approximately 160 at mid-2003) were available on Wiley's web site. Since that free service was discontinued and the rights reverted to the author, this report remains the only authorized continuously updated version on gene therapy companies.
Benefits of this report
- Up-to-date on-stop information on gene therapy with 73 tables and 15 figures
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Research and Markets: Gene Therapy - Technologies, Markets and Companies - Updated 2013 Report
Bharat Book Pressents : Gene Therapy Technologies, Markets and Companies – Video
Bharat Book Pressents : Gene Therapy Technologies, Markets and Companies
For more information kindly visit : http://www.bharatbook.com/healthcare-market-research-reports/gene-therapy-technologies-markets-and-companies.html Benefit...
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Bharat Book Pressents : Gene Therapy Technologies, Markets and Companies - Video
Bone Marrow Stem Cell Transplantation and Gene Therapy for Cystinosis – Video
Bone Marrow Stem Cell Transplantation and Gene Therapy for Cystinosis
A presentation by Stephanie Cherqui, PhD, University of California, San Diego at the 2013 Day of Hope Cystinosis Research Foundation Family Conference, Balbo...
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Bone Marrow Stem Cell Transplantation and Gene Therapy for Cystinosis - Video
Health: Gene therapy
London, United Kingdom (Reuters) - British scientists are stepping up clinical tests of gene therapy in a bid to help people with advanced heart failure pump blood more efficiently. Researchers said on Tuesday they planned to enroll patients into two new clinical trials using Mydicar, a gene therapy treatment made by privately held US biotech company Celladon. After more than 20 years of research, the ground-breaking method for fixing faulty genes is starting to deliver, with European authorities approving the first gene therapy for an rare metabolic disease last November.
In the case of heart failure, the aim is to insert a gene called SERCA2a directly into heart cells using a modified virus, delivered via a catheter infusion. Lack of SERCA2a leads to ever weaker pumping in people with heart failure.
Although drugs offer some relief, there is currently no way of restoring heart function and the prognosis for those with advanced disease is worse than for many cancers.
One of the studies, led by scientists at Imperial College London, is part of a wider mid-stage Phase II project sponsored by Celladon that involves 200 patients worldwide, some of whom have already been treated in the United States and Denmark.
The second trial, which is due to start in the summer, will test the same treatment in 24 British patients already fitted with mechanical heart pumps to see how the approach may help in this particular setting.
It promises to be a long haul, with extensive Phase III studies still needed once results of the current mid-stage tests are received, which Celladon expects in the first half of 2015. Gene therapy has experienced a series of advances and setbacks over the decades. The most notable blow came in 1999 when an Arizona teenager died in a gene therapy experiment. More recent results, however, have been promising in fields ranging from immune system diseases to blindness.
''It is a great example of the slow burn of good laboratory science translating into a potential clinical treatment,'' said Peter Weissberg, medical director of the British Heart Foundation, which is co-funding the second trial.
Because gene therapy replaces or boosts the activity of a faulty gene, it offers the possibility of a one-time ''fix'' - and that creates an economic challenge.
Any gene therapy is bound to be expensive, since a single dose could last a lifetime and the manufacturer will have just one shot at recouping its investment.
But Alexander Lyon of Imperial College, lead investigator on both studies, said it could be a cost-effective solution in heart failure if it avoided the need for interventions such as heart transplants at 200,000 pounds ($300,000) each.
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Gene therapy to repair failing hearts starts trial
More than 200 people with heart failure are to receive a pioneering form of gene therapy to try to get their hearts beating properly again. "This is the first ever gene therapy trial to target heart failure," says lead investigator Alexander Lyon of Imperial College London.
Heart failure results after damage to the heart muscle causes it to deteriorate, which in turn progressively weakens cells that govern heartbeat. The result is serious fatigue due to the heart's inability to pump blood efficiently. Each year in the UK alone it affects 120,000 people who have never had the condition before, killing a third of them within 12 months.
Doctors will inject participants with harmless viruses that ferry a gene called SERCA2a into their heart muscle. The gene codes for a protein that recycles calcium within heart muscle cells, vital for driving each heartbeat and priming the next one.
In damaged cells, this recycling is impaired. By loading new copies of the gene the aim is to compensate for this decline. "The gene therapy will reset the calcium control," says Lyon.
A preliminary trial of the same therapy three years ago in 39 people demonstrated that it is safe and delivers benefits. Those who got the highest dose of the virus, for example, spent only a tenth as long in hospital as those given a placebo (Circulation, doi.org/bzvxst). The impending follow-up trial will recruit 200 people split equally between the US and Europe.
In a separate trial of the same therapy, doctors will treat 24 people who already have temporary mechanical implants to aid heartbeat while they await heart transplants.
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Gene therapy to offer up to 1m heart patients new lease of life
The first attempt in Britain to treat heart failure patients with gene therapy is to begin within weeks, as part of study aimed at improving the lives of up to a million people in the UK who suffer the debilitating and potentially fatal condition.
Click HERE to view 'how to treat a failing heart' graphic
Two clinical trials are planned for a few dozen British patients who will be deliberately exposed to a virus carrying a synthetic copy of a human gene known to be involved in boosting heartbeat.
The first trial will be carried out at the Royal Brompton Hospital in London and the Golden Jubilee National Hospital in Glasgow. The patients will be part of a group of 200 from around the world who will have the virus injected via a cardiac catheter inserted through a vein in the leg. A second trial at the Harefield and Papworth hospitals will be based entirely within the UK and involve 24 patients with chronic heart failure who are already fitted with an "artificial heart" known as a left ventricular assist device, which helps to pump blood around the body.
The aim in both trials is to inject additional copies of a healthy gene, known to be responsible for a key protein involved in regulating the rhythmic contraction of the heart muscle. It is hoped that the extra genes will remain active within a patient's heart for many months or even years.
Scientists believe the approach could lead to a significant improvement in the efficiency of the diseased heart to pump blood around the body so improving the quality of life of thousands of patients with progressive heart failure who develop serious ailments as well as severe fatigue.
Scientists warned that it will still be several years before the technique can be made widely available. They do not want to raise hopes unduly as many previous gene therapy trials on patients with a range of other illnesses have failed to live up to expectations.
However, the heart researchers said they are optimistic that the gene technique will improve the quality of life in at least some of the patients, who would otherwise suffer deteriorating health and life expectancy a third of patients die within a year of diagnosis.
"Once heart failure starts, it progresses into a vicious cycle where the pumping becomes weaker and weaker, as each heart cell simply cannot respond to the increased demand," said Alexander Lyon, a consultant cardiologist at the Royal Brompton.
"Our goal is to fight back against heart failure by targeting and reversing some of the critical molecular changes arising in the heart when it fails."
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Gene therapy to offer up to 1m heart patients new lease of life
Gene therapy to offer heart patients new lease of life
The first attempt in Britain to treat heart failure patients with gene therapy is to begin within weeks, as part of study aimed at improving the lives of up to a million people in the UK who suffer the debilitating and potentially fatal condition.
Click HERE to view 'how to treat a failing heart' graphic
Two clinical trials are planned for a few dozen British patients who will be deliberately exposed to a virus carrying a synthetic copy of a human gene known to be involved in boosting heartbeat.
The first trial will be carried out at the Royal Brompton Hospital in London and the Golden Jubilee National Hospital in Glasgow. The patients will be part of a group of 200 from around the world who will have the virus injected via a cardiac catheter inserted through a vein in the leg. A second trial at the Harefield and Papworth hospitals will be based entirely within the UK and involve 24 patients with chronic heart failure who are already fitted with an "artificial heart" known as a left ventricular assist device, which helps to pump blood around the body.
The aim in both trials is to inject additional copies of a healthy gene, known to be responsible for a key protein involved in regulating the rhythmic contraction of the heart muscle. It is hoped that the extra genes will remain active within a patient's heart for many months or even years.
Scientists believe the approach could lead to a significant improvement in the efficiency of the diseased heart to pump blood around the body so improving the quality of life of thousands of patients with progressive heart failure who develop serious ailments as well as severe fatigue.
Scientists warned that it will still be several years before the technique can be made widely available. They do not want to raise hopes unduly as many previous gene therapy trials on patients with a range of other illnesses have failed to live up to expectations.
However, the heart researchers said they are optimistic that the gene technique will improve the quality of life in at least some of the patients, who would otherwise suffer deteriorating health and life expectancy a third of patients die within a year of diagnosis.
"Once heart failure starts, it progresses into a vicious cycle where the pumping becomes weaker and weaker, as each heart cell simply cannot respond to the increased demand," said Alexander Lyon, a consultant cardiologist at the Royal Brompton.
"Our goal is to fight back against heart failure by targeting and reversing some of the critical molecular changes arising in the heart when it fails."
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Gene therapy for heart failure
Doctors at the Royal Brompton Hospital in London will treat 200 patients next month as part of the first ever gene therapy trial for heart failure, which is taking place in 50 centres around the world.
A separate trial, which will test the same therapy in 24 patients who have been fitted with mechanical heart pumps, is expected to begin later this year.
Prof Sian Harding of Imperial College London, who developed the treatment, said: "It's been a painstaking, 20-year process to find the right gene and make a treatment that works, but we're thrilled to be working with cardiologists to set up human trials that could help people living with heart failure."
Prof Peter Weissberg, Medical Director of the British Heart Foundation, which is co-funding the research, said: "While drugs can offer some relief, there is currently no way of restoring function to the heart for those suffering with heart failure. Gene therapy is one of the new frontiers in heart science."
Excerpt from:
New gene therapy trials aim to mend broken hearts
British scientists are stepping up clinical tests of gene therapy in a bid to help people with advanced heart failure pump blood more efficiently.
Researchers said on Tuesday they planned to enroll patients into two new clinical trials using Mydicar, a gene therapy treatment made by privately held U.S. biotech company Celladon.
After more than 20 years of research, the ground-breaking method for fixing faulty genes is starting to deliver, with European authorities approving the first gene therapy for an rare metabolic disease last November.
In the case of heart failure, the aim is to insert a gene called SERCA2a directly into heart cells using a modified virus, delivered via a catheter infusion. Lack of SERCA2a leads to ever weaker pumping in people with heart failure.
Although drugs offer some relief, there is currently no way of restoring heart function and the prognosis for those with advanced disease is worse than for many cancers.
One of the studies, led by scientists at Imperial College London, is part of a wider mid-stage Phase II project sponsored by Celladon that involves 200 patients worldwide, some of whom have already been treated in the United States and Denmark.
The second trial, which is due to start in the summer, will test the same treatment in 24 British patients already fitted with mechanical heart pumps to see how the approach may help in this particular setting.
It promises to be a long haul, with extensive Phase III studies still needed once results of the current mid-stage tests are received, which Celladon expects in the first half of 2015.
Gene therapy has experienced a series of advances and setbacks over the decades. The most notable blow came in 1999 when an Arizona teenager died in a gene therapy experiment. More recent results, however, have been promising in fields ranging from immune system diseases to blindness.
"It is a great example of the slow burn of good laboratory science translating into a potential clinical treatment," said Peter Weissberg, medical director of the British Heart Foundation, which is co-funding the second trial.
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Rett’s Syndrome: Finding a Cure Through Gene Therapy – Video
Rett #39;s Syndrome: Finding a Cure Through Gene Therapy
A lab research project for the student bio expo 2013 by Mimansa Dogra I explore Rett #39;s Syndrome and the causes of this disorder, as well as treatments than c...
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Rett's Syndrome: Finding a Cure Through Gene Therapy - Video
MRI Interventions' ClearPoint(R) System Helps Advance Brain Cancer Clinical Trial of Investigational Gene Therapy Drug …
IRVINE, Calif., April 25, 2013 (GLOBE NEWSWIRE) -- MRI Interventions, Inc. (MRIC) today announced that its ClearPoint(R) Neuro Intervention System is advancing the minimally-invasive precision delivery of the investigational gene therapy drug Toca 511 into malignant brain tumors. The procedure is being performed under real-time visualization and direct magnetic resonance imaging (MRI) guidance at select clinical trial sites. Highlights of the ClearPoint System's application in the Toca 511 trial include:
Improved flow rates during drug delivery. The recently-introduced SmartFlow(R) large-bore cannula increases the drug delivery rate threefold over documented flow rates of the original SmartFlow cannula. Like the original SmartFlow cannula, the new cannula incorporates a stepped tip design to prevent reflux and leakage of the drug outside of the target area, but the new cannula's large bore allows for a clinically meaningful increased rate of delivery.
Precise delivery of the therapeutic agent into the brain tumor. Direct visualization of the procedure in real-time allows surgeons to monitor and confirm delivery to the tumor of Toca 511 mixed with MRI contrast agent at the time of infusion.
Multiple trajectories in a single case. The ClearPoint System includes a SmartFrame(R) targeting device that works with software to allow convenient repositioning of the cannula for multiple trajectories in a single case, and an adjustable head stabilization device that accommodates a wide range of possible entry points.
Tocagen Inc., the clinical trial sponsor, is developing the investigational drug Toca 511 (vocimagene amiretrorepvec) in combination with Toca FC (an investigational extended-release formulation of 5-FC) for the treatment of recurrent high grade glioma, including glioblastoma multiforme (GBM, Grade IV glioma), the most common and aggressive form of brain cancer. Toca 511 is a retroviral replicating vector (RRV) encoding the genetic instructions for the enzyme cytosine deaminase (CD). Toca 511 is designed to selectively infect dividing cancer cells and spread through the tumor after administration. Each patient then begins a course of Toca FC. Within infected cells the CD enzyme converts 5-FC to the anti-cancer drug 5-FU. By producing 5-FU locally, this technology has the potential to produce much higher concentrations of 5-FU in the tumor than can be safely attained with systemic administration.
"So far we successfully delivered Toca 511 precisely to the brain cancer in three patients, all of whom went home the next day," said Manish Aghi, MD, a neurosurgeon and principal investigator for the Toca 511 trial at University of California, San Francisco. "This new ability to deliver large volumes of Toca 511 rapidly into the tumor at flow rates up to 1.8 ml/h (30 microliters a minute) under real-time visualization represents a major technological advance that will enable the neurosurgeon to accurately deliver large quantities of a therapeutic agent, while providing the patient the benefit and safety of a minimally-invasive procedure."
"Our collaboration with Tocagen underscores the advantages of real-time MRI-guided delivery of therapeutic agents to the brain, and we are pleased to be a key contributor to the rapid progress being achieved," said Kimble Jenkins, CEO of MRI Interventions.
Each year approximately 10,000 new cases of GBM are diagnosed in the US. In a recent population-based study, median survival in all diagnosed patients was 10 months.
Tocagen is presently enrolling patients in its investigational Phase I clinical trials. Currently, University of California, San Francisco, University of California, San Diego, Cleveland Clinic Foundation, and Henry Ford Hospital in Detroit are enrolling patients, and additional sites are in the process of joining this study. For more information about participating in this study, please submit an inquiry form to Tocagen.
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Gene Therapy Used in Dogs to Treat Type 1 Diabetes
ALEXANDRIA, VA--(Marketwired - Apr 23, 2013) - In a major advance in the use of gene therapy for treating type 1 diabetes, researchers have shown that this approach can provide beneficial therapeutic effects in dogs for up to four years, without causing hypoglycemia, according to a study published in the May issue of the journal Diabetes.
Researchers from the Universitat Autonoma de Barcelona, led by Dr. Fatima Bosch, had previously shown positive effects using gene therapy to regulate glycemic control in mice. This study was the first to show long-term beneficial effects in large animals (beagle dogs), a major step toward ultimately testing the procedure in humans.
"Moving from mice to large animals is a big step," said Bosch, who is the Director of the Center of Animal Biotechnology and Gene Therapy. "If something works well in large animals, we have reason to believe, based on the previous experience in the field of gene transfer, that it is likely that we will see a similar outcomes in humans. For example, gene therapy that worked well in large animals to treat hemophilia is now seeing positive results in clinical trials with humans. We hope that in a few years, we'll be able to test this therapy for type 1 diabetes in humans as well."
Bosch and her team injected five lab dogs with two genes -- insulin and glucokinase -- using an adenoassociated virus (AAV) vector. The vector, or vehicle for DNA transmission, comes from a virus that is non-pathogenic and is able to "infect" cells in the animal's skeletal muscle with the two genes. Because skeletal muscle cells do not divide, the genes remain there long-term. This means the animal only needs to be injected once and the genes can continue to do their work, acting as "glucose sensors", continuously releasing low levels of insulin and expressing glucokinase, which helps regulate glucose uptake in response to rising and falling circulating glucose levels, for years.
In this study, the treated dogs maintained normal blood glucose levels for more than four years after injection, without showing signs of hypoglycemia, a risk associated with other treatments that seek to achieve tight blood glucose control. This is also the first time a single therapeutic intervention has been used to achieve long-term maintenance of normoglycemia after development of diabetes, the authors said.
Treating the dogs with just one gene did not achieve the same results, the researchers said, noting that both were needed to keep blood glucose levels in the normal range. The next step is to test the procedure on "companion animals," different breeds of dogs that have type 1diabetes and live with families, rather than in the lab. In this study, researchers will test their ability to better adjust the insulin doses in different size and breeds of dogs living in "real life" situations. Once they are able to determine how to adjust the therapy in this context, the investigators will be ready to test the treatment on humans, Bosch said.
"Overall the report by Bosch and colleagues is a substantial advance in the attempts to develop clinical gene therapy for type 1 DM," writes Timothy O'Brien, Director of the Regenerative Medicine Institute, at the National University of Ireland, Galway, in a commentary accompanying the study in Diabetes. He notes that "substantial challenges" remain in pursuing this type of treatment, but that "they are worthy of pursuit given the ultimate prize if the approach is successful."
Bosch notes that gene therapy does not represent a "cure" for type 1diabetes because it does not regenerate beta cells, but rather could lead to an effective long-term treatment. Maintaining blood glucose levels in the normal range is critical for managing diabetes and for preventing serious complications associated with the disease, such as nerve damage, heart and kidney disease.
This study also holds promise for treating dogs with diabetes, Bosch said. As it has in humans, diabetes has been increasing in recent years in dogs, she noted.
To reach lead researcher Fatima Bosch, Center of Animal Biotechnology and Gene Therapy, Barcelona, Spain: email Fatima.bosch@uab.es or phone: 34 93 581 4182.
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New gene therapy approach may stop Parkinson's in it tracks
Washington, April 22 (ANI): A gene therapy approach developed by researchers at Northeastern University in Boston may one day stop Parkinson's disease (PD) in it tracks, preventing disease progression and reversing its symptoms.
The novelty of the approach lies in the nasal route of administration and nanoparticles containing a gene capable of rescuing dying neurons in the brain.
Parkinson's is a devastating neurodegenerative disorder caused by the death of dopamine neurons in a key motor area of the brain, the substantia nigra (SN). Loss of these neurons leads to the characteristic tremor and slowed movements of PD, which get increasingly worse with time.
Currently, more than 1 percent of the population over age 60 has PD. The available drugs on the market for PD mimic or replace the lost dopamine but do not get to the heart of the problem, which is the progressive loss of the dopamine neurons.
The focus of Dr. Barbara Waszczak's lab at Northeastern University in Boston is to find a way to harvest the potential of glial cell line-derived neurotrophic factor (GDNF) as a treatment for PD. GDNF is a protein known to nourish dopamine neurons by activating survival and growth-promoting pathways inside the cells.
Not surprisingly, GDNF is able to protect dopamine neurons from injury and restore the function of damaged and dying neurons in many animal models of PD. However, the action of GDNF is limited by its inability to cross the blood-brain barrier (BBB), thus requiring direct surgical injection into the brain.
To circumvent this problem, Waszczak's lab is investigating intranasal delivery as a way to bypass the BBB. Their previous work showed that intranasal delivery of GDNF protects dopamine neurons from damage by the neurotoxin, 6-hydroxydopamine (6-OHDA), a standard rat model of PD.
Taking this work a step further, Brendan Harmon, working in Waszczak's lab, has adapted the intranasal approach so that cells in the brain can continuously produce GDNF.
His work utilized nanoparticles, developed by Copernicus Therapeutics, Inc., which are able to transfect brain cells with an expression plasmid carrying the gene for GDNF (pGDNF). When given intranasally to rats, these pGDNF nanoparticles increase GDNF production throughout the brain for long periods, avoiding the need for frequent re-dosing.
Now, in new research presented during Experimental Biology 2013 in Boston, MA, Harmon reported that intranasal administration of Copernicus' pGDNF nanoparticles results in GDNF expression sufficient to protect SN dopamine neurons in the 6-OHDA model of PD.
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Gene Therapy / Dr. Nael El Shal – Video
Gene Therapy / Dr. Nael El Shal
Interview with Dr. Nael El Shal about Gene Therapy with AL Aan TV.
By: AlGarhoudPvtHospital
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