The Latest Biology News And Medical NewsBiology News 2Health News 2Biology News 3Health News 3
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:
(Date:11/23/2009)... By producing some of the highest resolution image...tists have a deeper understanding how biomolecules... lead to a new treatment for kidney stones using b...e Nov. 23 online edition of the journal Proceedin...how peptides interact with mineral surfaces by acc...
(Date:11/23/2009)...versity of New Hampshire microbiologist Louis Tisa...nce understanding of the actinorhizal plants, wide...ent-poor and contaminated soils. , The United S...a, a professor of molecular, cellular and biomedic...ationship between the bacteria Frankia and Casuari...
(Date:11/23/2009)...string of recent discoveries about the multiple he... this multi-purpose nutrient, increased awareness ... it, spurred research and even led to an appreciat...ranging from the health of your immune system to p...to influenza, vitamin D is now seen as one of the ...
Breaking Biology News(10 mins):Scientists watch as peptides control crystal growth with 'switches, throttles and brakes' 2Scientists watch as peptides control crystal growth with 'switches, throttles and brakes' 3UNH prof. receives nearly $500,000 to research environmentally significant plants 2Multiple health concerns surface as winter, vitamin D deficiences arrive 2Multiple health concerns surface as winter, vitamin D deficiences arrive 3DaVita to Present Three Abstracts for the International Society for Peritoneal Dialysis 55368 1DaVita to Present Three Abstracts for the International Society for Peritoneal Dialysis 55368 2Vestara Announces Strategic Agreement with Leading Contract Services Company Novation 13691 1Vestara Announces Strategic Agreement with Leading Contract Services Company Novation 13691 2New Study Reveals Mouthguards are Protective but can Cause Harm 55364 1New Study Reveals Mouthguards are Protective but can Cause Harm 55364 2New Study Reveals Mouthguards are Protective but can Cause Harm 55364 3
(Date:11/23/2009)...--PearlTherapeuticsInc.,abiopharmaceuticalcompanyd...icrespiratorydiseases,todayannouncedtheappointment...nhas25yearsofexperienceinthepharmaceuticalandbiote...esandALZACorporation.Hisappointmentincreasesthenum...ngsawealthofproductdevelopment,commercializationan...
(Date:11/20/2009)...opportunity to take the lead in two small biotechn...Valencia (UV), working together with the IDICHUS F...he Spanish biotechnology industry. Their conclusio... clout than those in English-speaking countries. ,... been overlooked by the leading countries in this ...
(Date:11/20/2009)...ire-FirstCall/ -- Novavax, Inc. (Nasdaq: NVAX ) t...public offering of 6,800,000 shares of its common ... The approximately $21 million of net proceeds, a...issions, will be used for preclinical studies and ...l research and development programs, working capit...
(Date:11/20/2009)...ntly announced that a free webinar, “Roller ...ticle Characterization”, will be presented o...ion of in situ particle characterization is demons...es of roller compaction runs while varying raw mat... , (PRWEB) November 20, 20...
Breaking Biology Technology:Pearl Therapeutics Appoints Howie Rosen to Board of Directors 2Spanish biotechnology should focus on food and plant sectors to be more competitive 2Novavax Prices Public Offering of Common Stock 2Roller Compaction Process Optimization Using At-Line Particle Characterization 2
Other News:
...disease diagnosis and drug development, biochip re...ted to an international audience of top nanoscient...Hugh Roy and Lillie Cranz Cullen Distinguished Pro...has been selected as one of the plenary speakers a...
...ve found a way to mimic epileptic seizures in the ...the worm a powerful model for unraveling the molec...cts two percent of the population.......Guy A. Cal...stitute's (HHMI) Undergraduate Research Intern Pro...
...tch on to human cells has been published by UK, Fr...y detailed model of one of the tools used by some ...erichia coli, to stick to and gain entry to host c...ws, Reader in Chemical and Structural Biology at I...
...ancer among women worldwide. Research has shown th...sive cervical cancer is associated with the onset ...ing a mouse model of cervical cancer to explore th...ression, Douglas Hanahan and colleagues, from the ...
Disease diagnosis, drug development focus of UH prof's biochip research 2Convulsions in worms mimic epileptic seizures 2Convulsions in worms mimic epileptic seizures 3Stuck on you: Scientists lay bare secrets of bacterial attachment proteins 2
A team of researchers at Virginia Commonwealth University Massey Cancer Center has discovered an entirely new mechanism of action for a novel pharmacological agent currently in clinical trials in pati
...THACA, N.Y. -- The most detailed analysis to date ...evel shows strong evidence that natural selection ...cording to researchers from Cornell University, Ce...y published in the Oct. 20 issue of the journal Na...
...CTOBER 17, 2005 (BRUSSELS, BELGIUM) For the fourt...obal supplier of prosthetic and orthotic devices, ...panies. Ossur was listed No. 185 in 2005 Europe's...ing companies published by Europe's 500 Entreprene...
NEW BRUNSWICK/PISCATAWAY, N.J. Safe, versatile and environmentally friendly chemicals could replace hazardous, petroleum-based solvents used in science labs and industrial plants. Chemists at Rutgers
VCU Massey Cancer Center researchers find new agent inhibits Leukemia cell survival 2Cornell finds natural selection in humans 2Cornell finds natural selection in humans 3Ossur one of Europe's 500 fastest growing companies 2Rutgers findings a step toward safer chemicals in labs and industry 2Rutgers findings a step toward safer chemicals in labs and industry 3