HOME >> BIOLOGY >> NEWS
Effects of Muscular Dystrophy reversed by gene therapy, reports team fromChildren's National Medical Center and the University of Pittsburgh

SAN FRANCISCO, Oct. 19 -- Animal muscles crippled due to a form of muscular dystrophy can be repaired, both in size and strength, through an innovative gene therapy, according to a team of investigators from the Children's National Medical Center in Washington, D.C., and the University of Pittsburgh. The results, presented Friday, Oct. 22, at the annual meeting of the American Society of Human Genetics (ASHG) in San Francisco, offer the first evidence that whole muscles can be functionally rescued in any type of muscular dystrophy using an extremely safe gene vector.

"We are very excited by these preliminary results, which suggest that we can use a non-toxic virus to safely shuttle a gene for an important muscle protein that is improperly made in people suffering limb girdle muscular dystrophy," said Devin Dressman, who is presenting the results and who is a graduate student at the Center for Genetic Medicine, Children's National Medical Center, and the University of Pittsburgh's School of Medicine. "These findings demonstrate the feasibility of using the gene therapy approach in treating affected patients."

In their experiments, the investigators used a non-replicating adeno-associated virus (AAV) to carry a gene for the sarcoglycan protein, an important constituent of skeletal muscle. Unlike several other available viral vectors, AAV does not provoke an immune response from the body, either by itself or when it resides within cells. Furthermore, it can infect non-dividing cells and it takes up residence permanently, thus allowing long-term production of a critical protein.

In a collaborative effort, Xiao Xiao, Ph.D., assistant professor of molecular genetics and biochemistry at the University of Pittsburgh, injected this AAV-sarcoglycan gene combination into hamster leg muscles ravaged by limb girdle dystrophy. After one month, Jon Watchko, M.D., associate professor of pediatrics and of obstetrics, gynecology and reproductive sciences at the
'"/>

Contact: Lauren Ward
wardle@msx.upmc.edu
412-624-2607
University of Pittsburgh Medical Center
22-Oct-1999


Page: 1 2

Related biology news :

1. Effects of maternal myasthenia gravis on pregnancy and birth examined
2. Effects of rare, devastating disease linked to shrinking of cells telomeres
3. Effects of global warming already being felt on plants and animals worldwide
4. Effects of iron-deficiency anemia in infants linger
5. Effects of ozone pollution threaten agricultural production on Long Island, NY, says Cornell plant pathologist
6. Urine Tests Will Do Better Than Pelvic Exams To Save Teens From Serious Effects Of Sexually Transmitted Diseases, UCSF Analysis Shows
7. Experimental Drug Derived From Snake Venom Shows Promise In Reversing Effects Of Stroke
8. Vanderbilt Medical Students Study Effects Of Vibration On Infant Health
9. Early Nutrition In Babies May Have Long Term Effects On The Brain
10. Alcohol Consumption, Resistance To Its Effects Related To Levels Of Neurotransmitter, Say UW Researchers
11. DDT, PCBs, And Invasive Plants - USGS Scientists Examine The Effects Of Contaminant And Invasive Species On Wildlife

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


(Date:6/19/2020)... ... June 18, 2020 , ... ... Your Lab with Software for Data Decisions,” for pharmaceutical and biotech executives, ... integrate and automate cutting-edge scientific instruments and laboratory equipment from dozens of ...
(Date:6/5/2020)... (PRWEB) , ... June 04, 2020 , ... Murrieta ... filed a new patent application for simplified sample preparation for Next Generation Genomic ... will greatly reduce cost, time, and complexity of sample preparation. , “I have been ...
(Date:5/28/2020)... ... 27, 2020 , ... CrucialTrak, a global designer and manufacturer of biometric access ... (CEG) to market and sell its complete line of biometric readers and access ... infrastructure optimization solutions . , Brett Mason, CrucialTrak’s Director of Channel Sales for ...
Breaking Biology News(10 mins):
(Date:6/11/2020)... ... ... Reducing carbon emissions has been and will continue to be a major ... ways to reduce CO2 is something we can’t avoid. , To protect the ... produced today are created from recycled cooking oil or crops. Unfortunately, these sources are ...
(Date:6/2/2020)... ... June 01, 2020 , ... As ... encourage positive change in the world, enterprise security solutions provider bitsIO donated time ... people working from home during self-quarantines and shelter-in-place orders, companies are increasingly concerned ...
(Date:5/30/2020)... ... May 29, 2020 , ... The Conference Forum ... newsletter solely dedicated to the unique role of the Chief Medical Officer in ... the responsibilities, problem-solving, best practices and career development of CMOs and Heads of ...
(Date:5/28/2020)... SPRING, Md. (PRWEB) , ... May 28, 2020 , ... ... distinguished biometrics and security leaders as the 2020 winners of the Women in ... co-presented with sponsors IDEMIA , Biometric Update and the SIA ...
Breaking Biology Technology:
Cached News: