HOME >> BIOLOGY >> NEWS
Penn Researchers Develop Gene Therapy Technique That Reverses Muscle Membrane Weakness In Muscular Dystrophy Variant

(Philadelphia, PA) - Previous gene therapy approaches to treat muscular dystrophy have been hampered by an inability to successfully place the therapeutic genetic material into deficient muscle cells. Now, for the first time, using a naturally-occurring hamster model of limb girdle muscular dystrophy (LGMD), researchers at the University of Pennsylvania Health System have developed a technique that successfully produces widespread transfer of corrective genetic material into muscle cells throughout an entire limb. In so doing, the research team also found evidence for a cascade of events that eventually results in cellular destabilization - a process which manifests as muscle weakness in patients with this and other forms of muscular dystrophy.

It is hoped that the Penn-developed technique would be ultimately transferable to human clinical trials involving gene therapy treatments of many forms of muscular dystrophy, as well as other types of debilitating or fatal disorders - such as metabolic myopathy and cardiomyopathy (heart failure). "We've demonstrated proof of concept that the methodology can work for both skeletal and cardiac muscle," says principal author Hansell Stedman, MD, assistant professor of surgery at Penn's Institute for Human Gene Therapy, "so the next step will be to establish its safety and effectiveness in patients with this form of muscular dystrophy." Stedman cautions that clinical trials will have to address safety issues one step at a time before significant therapeutic benefit can be approached.

The research is being published as the cover article in the April 1999 issue of Nature Medicine.

Induced Vessel Leakage Nets Massive Cell Saturation
In limb girdle muscular dystrophy, the instability of muscle tissue is linked directly to the level of genetic disruption that occurs within the sarcoglycan complex - a critical muscle structure composed of four membrane-spanning proteins. Depending on the nature of
'"/>

Contact: Rebecca Harmon
harmonr@mail.med.upenn.edu
215-349-5660
University of Pennsylvania School of Medicine
30-Mar-1999


Page: 1 2 3 4

Related biology news :

1. Researchers determine genetic cause of Timothy syndrome
2. Researchers find color sensitive atomic switch in bacteria
3. Researchers identify protein promoting vascular tumor growth
4. Researchers devise potent new tools to curb ivory poaching
5. Researchers create nanotubes that change colors, form nanocarpet and kill bacteria
6. Researchers ID chlorophyll-regulating gene
7. Researchers develop fast track way to discover how cells are regulated
8. Researchers identify distinctive signature for metastatic prostate cancer
9. Researchers report new gene test for isolated cleft lip and palate
10. Researchers discover why mutant gene causes colon cancer
11. Researchers identify the genomes controlling elements

Post Your Comments:
*Name:
*Comment:
*Email:


(Date:11/7/2018)... (PRWEB) , ... November 07, 2018 , ... ... research and advisory firm serving the life science industry. The event was attended ... of science” that advance biological research and drug discovery. The winners were determined ...
(Date:11/3/2018)... ... November 01, 2018 , ... ... New Challenges and New Opportunities, **Presented by FDAnews**, Dec. 6, 2018 – Kleinfeld, ... , Early bird pricing for Emerging Trends in Medical Device Regulation and Quality ...
(Date:10/31/2018)... TRIANGLE PARK, N.C. (PRWEB) , ... October 30, ... ... the appointment of Brad Heidinger to Director of Operations. With more than 20 ... executive with proven visionary leadership in the biomanufacturing industry. Heidinger joins Azzur from ...
Breaking Biology News(10 mins):
(Date:11/5/2018)... ... November 05, 2018 , ... GlycoMark, Inc., the ... professionals through its website, http://www.glycomark.com , as part of National Diabetes Month. ... option via the website in November from 9 a.m. – 5 p.m. ET ...
(Date:10/31/2018)... ... October 30, 2018 , ... Myoscience, ... at the Campbell Clinic in Memphis, Tennessee. This study enrolled 125 patients ... of care for managing pain after total knee replacement surgery (TKA). , The ...
(Date:10/25/2018)... ... October 24, 2018 , ... To prepare for radiological and ... will work Argentum Medical, LLC to repurpose its commercial Silverlon ® ... technology, the privately held company has been awarded a three-year, $8.4 million contract with ...
(Date:10/18/2018)... ... October 18, 2018 , ... R3 Stem Cell is ... day regenerative therapy training workshop. The discount is good through November 7th, 2018, with ... stem cell and PRP training workshop includes an amazing useful array of hands on ...
Breaking Biology Technology:
Cached News: