Navigation Links
Clinical trial for muscular dystrophy demonstrates safety of customized gene therapy
Date:11/30/2011

CHAPEL HILL, N.C. Researchers at the University of North Carolina at Chapel Hill have shown that it is safe to cut and paste together different viruses in an effort to create the ultimate vehicle for gene therapy. In a phase I clinical trial, the investigators found no side effects from using a "chimeric" virus to deliver replacement genes for an essential muscle protein in patients with muscular dystrophy.

"This trial demonstrates that gene therapy is no longer limited by the viruses we find in nature, and should usher in the next generation of viral delivery systems for human gene transfer," said senior study author R. Jude Samulski, PhD, professor of pharmacology and director of the Gene Therapy Center at UNC. The study appears online in the Nov. 8, 2011 issue of the journal Molecular Therapy.

Through gene therapy, scientists treat diseases by correcting a patient's faulty genes. Most of the time, this approach involves commandeering a natural system for infecting and introducing new genes into cells; thus, the virus. But even though there are lots of relatively innocuous viruses available for this purpose, none of them are perfectly suited for gene therapy.

Rather than rely on nature, Samulski and his colleagues decided to engineer their dream gene therapy virus in the laboratory. First they chose the adeno-associated virus or AAV, a small nonpathogenic virus that most humans are exposed to at some point in life. They then took their favorite attributes from different forms of AAV such as AAV type 1's ability to sneak into muscle, and AAV type 2's safe track record and combined them into one "chimeric" virus. In the first trial of this form of gene therapy, the investigators gave six boys with Duchenne muscular dystrophy (DMD) this new virus. An x-linked inherited disorder, DMD affects one in 4,000 newborn boys.

The virus was engineered to contain the dystrophin gene, which is missing in patients with muscular dystrophy and is the ultimate cause of the disease's progressive muscle weakness. The replacement genes were injected into the bicep in one arm and a placebo was injected into the other arm of each of the patients. The researchers were able to detect the new genes in all of the patients treated with the gene therapy, but no immunological response.

As they move on to the next phase of clinical trials, Samulski says they are carefully considering how best to administer the gene therapy vectors to patients. Delivering enough replacement genes to a therapeutic effect could require larger doses of virus, which in turn could elicit an unwanted immune response. So the researchers are exploring a number of different options, including using a new high pressure technique developed by William J. Powers, MD, professor and chair of neurology at UNC, reported last July in the same journal, to get the virus into muscle at lower doses.


'/>"/>

Contact: Les Lang
llang@med.unc.edu
919-966-9366
University of North Carolina School of Medicine
Source:Eurekalert  

Related biology news :

1. A new journal where molecular biology meets clinical research
2. Self-assembling nano-fiber gel delivers high concentrations of clinically approved drugs
3. Biochemistry of human physiology in health and disease is focus of updated clinical text
4. New book helps medical students master clinical skills
5. In just 5 years, gene discovery to clinical trial of potential treatment
6. Far-reaching genetics topics to be addressed: 2009 Annual Clinical Genetics Meeting, March 25-29
7. Genome Medicine: Bridging the gap between research and clinical practice
8. 3 new informatics pilot projects to aid clinical and translational scientists nationwide
9. TGen Clinical Research Services at Scottsdale Healthcare and Mayo Clinic study new cancer drug
10. ABC publishes monograph on scientific and clinical research of Sinupret
11. Researchers win award for best clinical paper in orthopedic physical therapy
Post Your Comments:
*Name:
*Comment:
*Email:
Related Image:
Clinical trial for muscular dystrophy demonstrates safety of customized gene therapy
(Date:2/3/2016)... -- Vigilant Solutions announces today that the ... solved two recent hit-and-run cases with the ... Solutions. Brian Wenberg explains, "I was ... out of a convenience store and witnessed an elderly male back out ... his vehicle and leaving the scene.  In his statement ...
(Date:2/2/2016)... NEW YORK , Feb. 2, 2016 ... healthcare facilities are primarily focused on medical ... that measure point-of-care parameters. Wearable devices that ... a user,s freedom of movement are being ... sensors for human biomedical signal acquisition coupled ...
(Date:2/2/2016)... 2, 2016 Checkpoint Inhibitors for Cancer ... Are you interested in the future of ... checkpoint inhibitors. Visiongain,s report gives those predictions to ... national level. Avoid falling behind in data ... and revenues those emerging cancer therapies can achieve. ...
Breaking Biology News(10 mins):
(Date:2/10/2016)... , Feb. 10, 2016  Matchbook, Inc., ... for fast growing biotech companies, announced today the ... Procurement Strategic Advisor. Jim brings nearly 25 years ... and procurement, having spent nearly two decades in ... Chain/Logistics and Procurement at Genzyme and, most recently ...
(Date:2/10/2016)... ... February 10, 2016 , ... ... registry built on the secure online PatientCrossroads platform, has exceeded both its one-year ... have joined the PROMPT study, which seeks to advance understanding of the hereditary ...
(Date:2/9/2016)... ... ... With a presidential election in November and the future of U.S. healthcare ... 500 top healthcare leaders for a night and day of debates and discussions about ... of the University of Pennsylvania’s Wharton School, will be held February 18 – 19, ...
(Date:2/9/2016)... ... February 09, 2016 , ... Tunnell Consulting, Inc. announced that Frédéric Lefebvre has ... focus on acquiring new accounts and work closely with existing Tunnell clients throughout Europe ... to our European clients more than 15 years of experience in the pharmaceutical industry, ...
Breaking Biology Technology: