Navigation Links
Muscle-targeted gene therapy reverses rare muscular dystrophy in mice

Gene therapy methods that specifically target muscle may reverse the symptoms of a rare form of muscular dystrophy, according to new research in mice conducted by medical geneticists at Duke University Medical Center. Infants born with the inherited muscular disorder called Pompe disease usually die before they reach the age of two. The researchers also said their approach of targeting corrective genes to muscles may have application in treating other muscular dystrophies.

Patients with Pompe disease have a defect in a key enzyme that converts glycogen, a stored form of sugar, into glucose, the body's primary energy source. As a result, glycogen builds up in muscles throughout the body, including the heart, causing muscles to degenerate.

Using genetically altered mice in which the gene for the enzyme had been rendered nonfunctional, the researchers demonstrated they could introduce the functioning gene and correct glycogen buildup in heart and skeletal muscle. The findings suggest that such an approach should be considered as a potential gene therapy strategy for Pompe disease patients, the researchers report in a forthcoming issue of Molecular Therapy (now available online).

"Gene therapy in muscular dystrophies presents a unique challenge, because replacement of deficient, therapeutic proteins invokes an immune response that limits the efficacy of the treatment," said Duke medical geneticist Dwight Koeberl, M.D., senior author of the study. "By restricting the expression of introduced genes to muscle, the immune response can be prevented or attenuated."

The muscle-targeted gene therapy might therefore apply to other forms of muscular dystrophy, they added. Muscular dystrophies include many genetic diseases, all of which are characterized by progressive weakness and degeneration of the skeletal muscles which control movement.

The Muscular Dystrophy Association and Genzyme Corporation supported the research.

Several for
'"/>

Source:Duke University Medical Center


Page: 1 2 3

Related biology news :

1. Adding Radiation Therapy To Chemotherapy Improves Survival In Patients With High-risk Breast Cancer
2. Columbia research lifts major hurdle to gene therapy for cancer
3. Combination therapy boosts effectiveness of telomere-directed cancer cell death
4. Gene therapy converts dead bone graft to new, living tissue
5. Study identifies predictors of HIV drug resistance in patients beginning triple therapy
6. New imaging method gives early indication if brain cancer therapy is effective, U-M study shows
7. New therapy for HIV/AIDS eliminates needles and excessive toxicity
8. New Treatment Rivals Chemotherapy For Lymphoma, Study Finds
9. Gene therapy for Parkinsons disease moves forward in animals
10. Pulsating ultrasound enhances gene therapy for tumors
11. Antiretroviral therapy may prevent excess risk of some cancers in people with HIV
Post Your Comments:
(Date:12/3/2014)... , Dec. 2, 2014 As part ... readers, Inception Technologies is pleased to announce the release ... for customers to collect the workforce data that they ... that have been left by existing readers. Many such ... interface, connectivity and modern technology. Older models force users ...
(Date:11/21/2014)... , Nov. 20, 2014 C-Labs LLC, a ... Internet of Things (IoT), today announced the appointment of ... operating officer. Previously a strategic advisor to the firm, ... operations. Mr. Traynor is based out of the C-Labs ... reports to Chris Muench , Chief Executive Officer. ...
(Date:11/18/2014)... Md. , Nov. 17, 2014 The Parenteral ... and U.S. regulatory agencies will speak and at least seven ... Metrics Conference at the Omni Shoreham Hotel in ... pleased once again to have significant support from the regulatory ... Europe in our effort to help ...
Breaking Biology News(10 mins):Inception Technologies to Release New Biometric Reader 2C-Labs Names Former Microsoft and Bsquare Executive as Chief Operating Officer 2FDA's Janet Woodcock, EMA's Emer Cooke Headline PDA Quality Metrics Conference 2
... 854 children with Crohn,s disease shows a 5-year cumulative risk ... studies. The findings of the study, led by Hasbro Children,s ... 16 had an increased risk of bowel surgery, and that ... not alter the risk of surgery. The study is published ...
... MD (August 31, 2010) -- A new imaging system ... the eyeball (ocular fundus) may pave the way for ... the eye, such as age-related macular degeneration and diabetic ... Review of Scientific Instruments, which is published by ...
... MD (August 31, 2010) -- In December 2010, IceCube ... located beneath the Antarctic ice -- will finally be ... in the AIP,s Review of Scientific Instruments, ... and his colleague Spencer Klein of Lawrence Berkeley National ...
Cached Biology News:Risk of surgery for Crohn's disease lower than reported in recent studies 2LEDs illuminate eye for ocular disease screening 2IceCube neutrino observatory nears complete 2
(Date:12/19/2014)... Dec. 19, 2014 Naurex Inc., a biopharmaceutical ... for diseases of the central nervous system, today announced ... executive officer, will present at the 33 rd ... take place at 3:00 p.m. PST on Tuesday, January ... San Francisco, Calif. ...
(Date:12/19/2014)... DUBLIN , Dec. 19, 2014 Research ... announced the addition of the "Technology Innovations ... their offering. http://photos.prnewswire.com/prnh/20130307/600769 ... Innovations in Smart Fabrics" offers a detailed assessment ... Key Findings: 1. ...
(Date:12/19/2014)... 18, 2014 Ed Bilsky, Ph.D., vice ... Center for Excellence in the Neurosciences (CEN), and professor ... been selected as a member of the prestigious Dana ... neuroscientist from Maine to receive this honor. , ... entirely by the Dana Foundation. Its mission is ...
(Date:12/19/2014)... (PRWEB) December 19, 2014 The ... to the rising ageing population and technological innovations ... and nutraceutical industries are catalysing the growth of ... @ http://www.marketsandmarkets.com/Market-Reports/empty-capsules-market-218018190.html , Scope of Report ... as well as new entrants/smaller firms to gauge ...
Breaking Biology Technology:Naurex to Present at 33rd Annual J.P. Morgan Healthcare Conference 2Technology Innovations in Smart Fabrics (Technical Insights) 2UNE's Ed Bilsky Named Member of Dana Alliance for Brain Initiatives 2UNE's Ed Bilsky Named Member of Dana Alliance for Brain Initiatives 3Empty Capsules Market Hit to Reach $1,820 Million by 2019 - New Research Report by MarketsandMarkets 2Empty Capsules Market Hit to Reach $1,820 Million by 2019 - New Research Report by MarketsandMarkets 3Empty Capsules Market Hit to Reach $1,820 Million by 2019 - New Research Report by MarketsandMarkets 4
... scattering detector for ambient HPLC. Contains ... hardware for two external devices such ... contains an analog output from the ... chart recorders. Includes an input for ...
... The FDSS6000 System is an imaging ... assay development and high throughput screening ... luminescence modalities., The Hamamatsu light sources ... our companys decades-long experience in photon ...
... supplies, for 10 chips, provides the ... standard-sensitivity RNA analysis (nanogram levels) with ... electrophoresis system. Supplied are 1,250 microliters ... stain, 20 microliters RNA ladder, 900 ...
... White solid. PROTECT FROM LIGHT. PACKAGED ... compound that acts as a selective, ... PTP1B (IC 50 = 4 ... 403 and PTP1B 298 , ...
Biology Products: