Navigation Links
No small feat: First ever gene therapy success for muscular dystrophy achieved

Researchers from the University of Pittsburgh report the first study to achieve success with gene therapy for the treatment of congenital muscular dystrophy (CMD) in mice, demonstrating that the formidable scientific challenges that have cast doubt on gene therapy ever being feasible for children with muscular dystrophy can be overcome. Moreover, their results, published in this week's online edition of the Proceedings of the National Academy of Sciences (PNAS), indicate that a single treatment can have expansive reach to muscles throughout the body and significantly increase survival.

CMD is a group of some 20 inherited muscular dystrophies characterized by progressive and severe muscle wasting and weakness first noticed soon after birth. No effective treatments exist and children usually die quite young.

Despite gene therapy being among the most vigorously studied approaches for muscular dystrophy, it has been beset with uniquely difficult hurdles. The genes to replace those that are defective in CMD are larger than most, so it has not been possible to apply the same methods successfully used for delivering other types of genes. And because CMD affects all muscles, an organ that accounts for 40 percent of body weight, gene therapy can only have real therapeutic benefit if it is able to reverse genetic defects in every cell of the body's 600 muscle groups.

By using a miniature gene, similar in function to the one defective in CMD, and applying a newly developed method for "systemic" gene delivery, the Pitt researchers have shown that gene therapy for muscular dystrophy is both feasible and effective in a mouse model of especially profound disease. Using this approach, the team, led by Xiao Xiao, Ph.D., associate professor of orthopaedic surgery and molecular genetics and biochemistry at the University of Pittsburgh School of Medicine, report that treated mice had physiological improvements in the muscles of the heart, diaphragm, abdomen and l
'"/>

Source:University of Pittsburgh Medical Center


Page: 1 2 3

Related biology news :

1. Stopping smallpox in its tracks: A new anti-viral approach
2. New World founders small in number
3. Secret of smallpoxs success may lead to bioterror cure
4. Circulating stem cells play small role in lung repair
5. LIAI scientists make major finding on potential smallpox treatment
6. Researchers develop new testing methods for potential monkeypox or smallpox outbreak
7. Solexa and collaborating scientists illuminate the small RNA component of the transcriptome
8. Delaware scientists make significant advance in study of small RNAs
9. Scientists learn to predict protein-stabilizing ability of small molecules
10. One small step means giant leap for spinal cord research
11. New study of the worlds smallest elephant
Post Your Comments:
(Date:7/21/2015)... NEW YORK , July 21, 2015 /PRNewswire/ ... critical systems, as seen in the recent U.S. ... enterprises a solution to the infosec conundrum, but ... data is stored. To address this dilemma, Biometrics-as-a-Service ... of its Biometric Tokenization SDK for third party ...
(Date:7/9/2015)...  Synaptics Inc. (NASDAQ: SYNA ), a leading developer ... report financial results for the fourth quarter and fiscal ... close of market. The company will host a corresponding ... PT (5:00 p.m. ET), during which management may discuss ... live call, analysts and investors should dial 888-455-2260 (conference ...
(Date:7/8/2015)... July 8, 2015 Summary Pancreatic cancer ... fourth most fatal, with a mortality rate of 10.9 ... of pancreatic cancer patients has highlighted a significant need ... not being met by the current market. A ... products with varying molecule types and mechanisms of action, ...
Breaking Biology News(10 mins):HYPR Corp. Launches Industry First Biometric Tokenization Platform 2HYPR Corp. Launches Industry First Biometric Tokenization Platform 3HYPR Corp. Launches Industry First Biometric Tokenization Platform 4Synaptics to Report Fourth Quarter, Fiscal Year 2015 Results on July 30 2Frontier Pharma: Pancreatic Cancer - Identifying and Commercializing First-in-Class Innovation 2Frontier Pharma: Pancreatic Cancer - Identifying and Commercializing First-in-Class Innovation 3
... University of California, Riverside biologists working on guppies ... of long-term studies report that rapid growth responses ... restriction early in life have repercussions in adulthood. ... guppies that grew rapidly as juveniles produced fewer offspring ...
... without a class system of managers and workers? That,s ... solved: How did a prehistoric, egalitarian people called the ... a "manager" hierarchy? Archaeologists from Southern Methodist University ... the Gila River Indian Community in Arizona have launched ...
... for a severe inflammatory disease that has spread along ... to the edge of Europe. University of Manchester ... have revealed some of the genetic mutations that lead ... Nature Genetics . Behet,s is a vascular ...
Cached Biology News:Rapid growth in adolescence leads to fewer offspring, UC Riverside biologists find 2Rapid growth in adolescence leads to fewer offspring, UC Riverside biologists find 3New research will unravel Arizona's prehistoric puzzle of the Hohokam ceramic industry 2New research will unravel Arizona's prehistoric puzzle of the Hohokam ceramic industry 3New research will unravel Arizona's prehistoric puzzle of the Hohokam ceramic industry 4Disease genes that followed the Silk Road identified 2
(Date:7/30/2015)... , July 30, 2015   GenoSpace , ... the interpretation and analysis of genomic and other ... has joined the company as Vice President ... http://photos.prnewswire.com/prnh/20150730/250931 "GenoSpace partners ... integrate, interpret, analyze and explore complex sets of ...
(Date:7/30/2015)... ... ... The 2015 Market Research Report on the Global Propanol Industry is ... with a focus on the Chinese situation. Major companies included in the propanol market ... Royal Dutch Shell, LG Chem and Zhejiang Xinhua Chemical. , The report provides a ...
(Date:7/30/2015)... San Diego , CA (PRWEB) , ... July ... ... Biopharma, announced the successful completion of a field clinical study of its canine ... being developed together with Aratana Therapeutics (Kansas City, KS) and will be marketed ...
(Date:7/30/2015)... 2015   Senomyx , Inc. (NASDAQ: SNMX ... to discover, develop, and commercialize novel flavor ingredients for ... financial results for the second quarter 2015. ... remain on track to achieve our commercial and financial ... of the Company. "Since our last quarterly earnings report, ...
Breaking Biology Technology:GenoSpace Introduces Matthew Fischer as Vice President of Sales and Marketing 2Global Propanol Market Technical Data and Manufacturing Analysis 2020 2Global Propanol Market Technical Data and Manufacturing Analysis 2020 3VetStem Biopharma Successfully Completes Large Milestone Efficacy Study for its Flagship Canine Osteoarthritis Stem Cell Product 2VetStem Biopharma Successfully Completes Large Milestone Efficacy Study for its Flagship Canine Osteoarthritis Stem Cell Product 3SENOMYX ANNOUNCES SECOND QUARTER 2015 FINANCIAL RESULTS 2SENOMYX ANNOUNCES SECOND QUARTER 2015 FINANCIAL RESULTS 3SENOMYX ANNOUNCES SECOND QUARTER 2015 FINANCIAL RESULTS 4SENOMYX ANNOUNCES SECOND QUARTER 2015 FINANCIAL RESULTS 5SENOMYX ANNOUNCES SECOND QUARTER 2015 FINANCIAL RESULTS 6SENOMYX ANNOUNCES SECOND QUARTER 2015 FINANCIAL RESULTS 7SENOMYX ANNOUNCES SECOND QUARTER 2015 FINANCIAL RESULTS 8SENOMYX ANNOUNCES SECOND QUARTER 2015 FINANCIAL RESULTS 9
... will bike down the California coast to ... and help those affected by arthritis, SAN FRANCISCO, Sept. ... annual Amgen California Coast Classic,Bike Tour on Saturday, September 29 ... over eight days, aim to raise more,than $1.3 million this ...
... N.C., Sept. 21 Campbell Alliance, the ... and biotech,industries, today announced that Ben Bonifant, ... practice, will moderate a panel discussion,at the ... Information. The conference will be held September ...
... Roche has,advised Maxygen, Inc. (Nasdaq: MAXY ) ... development of MAXY-alpha, also known as R7025.,MAXY-alpha is a ... B virus infections and is licensed to Roche. ... an,unexpected reduction of the pharmacodynamic and pharmacokinetic effects of,MAXY-alpha ...
Cached Biology Technology:More Than 230 Cyclists to Ride in Amgen California Coast Classic Bike Tour to Raise $1.3 Million for Arthritis Foundation 2Campbell Alliance to Moderate Senior-Level Panel Discussion at Windhover Pharmaceutical Strategic Alliances Conference 2Campbell Alliance to Moderate Senior-Level Panel Discussion at Windhover Pharmaceutical Strategic Alliances Conference 3Maxygen Announces Hold on MAXY-alpha Development Program 2Maxygen Announces Hold on MAXY-alpha Development Program 3
Rabbit polyclonal to Testosterone 19 ( Abpromise for all tested applications). Antigen: Testosterone 19 CME HSA (Human)...
Kinase Buffer can be used to assay protein kinase activity...
Anti-Kusabira Orange Monoclonal Antibody Description: 100 g Research Focus: tag Storage: -20C Shipping Temperature: 4C...
alpha/beta-Tubulin Antibody Ship: Hot Store: -20 C...
Biology Products: