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:3/29/2017)... March 29, 2017  higi, the health IT company ... North America , today announced a Series ... acquisition of EveryMove. The new investment and acquisition accelerates ... tools to transform population health activities through the collection ... higi collects and secures data today on ...
(Date:3/23/2017)... The report "Gesture Recognition and Touchless Sensing Market by Technology (Touch-based and ... 2022", published by MarketsandMarkets, the market is expected to be worth USD 18.98 ... Continue Reading ... ...      ...
(Date:3/20/2017)... At this year,s CeBIT Chancellor Dr. Angela Merkel visited the ... the DERMALOG stand together with the Japanese Prime Minster Shinzo Abe. ... largest German biometrics company the two government leaders could see the three ... as DERMALOG´s multi-biometrics system.   Continue Reading ... ...
Breaking Biology News(10 mins):
(Date:5/23/2017)... ... May 23, 2017 , ... ... cells for research and the development of cardiac regeneration therapies. The development ... numbers of cardiomyocytes (hPSC-CMs). Due to varying differentiation efficiencies, further enrichment of ...
(Date:5/23/2017)... ... May 22, 2017 , ... NetDimensions has been ranked as ... Globe™ for Corporate Learning, 2017. , Aragon Research defines Leaders as organizations who ... perform against those strategies. NetDimensions’ ranking as a Leader due to its strengths ...
(Date:5/23/2017)... ... 2017 , ... Vortex Biosciences , provider of circulating tumor cell (CTC) ... cells using Vortex microfluidic technology ” in Nature Precision Oncology on May 8th. ... Carlo and Dr. Matthew Rettig at the University of California, Los Angeles. The publication ...
(Date:5/23/2017)... (PRWEB) , ... May 23, 2017 , ... ... Works as Vice President of Clinical Operations. She brings years of expertise ... Yaupon Therapeutics. From her professional foundation as a licensed occupational therapist, through a ...
Breaking Biology Technology: