HOME >> BIOLOGY >> NEWS
First clinical trial of gene therapy for muscular dystrophy now under way

The first gene therapy human trial in the United States for a form of muscular dystrophy is under way.

The clinical trial for Duchenne muscular dystrophy (DMD) tests the safety and effectiveness of a therapy that was developed over two decades by scientists at the University of North Carolina at Chapel Hill's School of Medicine and the University of Pittsburgh.

The trial was launched March 28, at Columbus Children's Hospital in Ohio, an affiliate of Ohio State University's School of Medicine. In the trial, six boys with DMD will receive replacement genes for an essential muscle protein.

Each of the boys will receive replacement genes via injection into a bicep of one arm and a placebo in the other arm. Neither the investigators nor the participants will know which muscle got the genes. After several weeks, an analysis of the injected muscle tissue's microscopic appearance, as well as extensive testing of the health and strength of the trial participants, will reveal whether gene therapy for DMD is likely to be safe and whether it's likely to result in persistent production of the essential protein in muscle cells.

Muscular dystrophies are genetic disorders characterized by progressive muscle wasting and weakness that begin with microscopic changes in the muscle. As muscles degenerate over time, the person's muscle strength declines.

Duchenne muscular dystrophy is a genetic disease that begins in early childhood, causes progressive loss of muscle strength and bulk, and usually leads to death in the 20s from respiratory or cardiac muscle failure. DMD occurs when a gene on the X chromosome fails to make the essential muscle protein dystrophin. One of nine types of muscular dystrophy, DMD primarily affects boys.

Currently, the best medical therapy can only slow the progressive muscle weakness of DMD.

The gene for dystrophin is one of the largest genes in the human body, and miniaturizing it, while retaining the cru
'"/>

Contact: Stephanie Crayton
scrayton@unch.unc.edu
919-966-2860
University of North Carolina at Chapel Hill
29-Mar-2006


Page: 1 2 3 4

Related biology news :

1. First case of successful ovarian tissue transplantation between two, nonidentical sisters
2. First new multiple sclerosis gene found in 30 years
3. First genome-wide study of infectious disease opens new avenues for HIV treatment, vaccines
4. First all-African produced genetically engineered maize is resistant to maize streak virus
5. First baby is born after oocytes were matured in the lab and frozen
6. First confirmed common genetic risk factors for breast cancer
7. First demonstration of new hair follicle generation in an animal model
8. First research to show that diabetes damages DNA in mens sperm and may affect fertility
9. First demonstration of muscle restoration in an animal model of Duchenne muscular dystrophy
10. First systematic study of Chinas one-child policy reveals complexity, effectiveness
11. First international conference on self-healing materials

Post Your Comments:
*Name:
*Comment:
*Email:
TAG: First clinical trial gene therapy for muscular dystrophy now under way

(Date:3/26/2015)... March 26, 2015 The Granite ... recreation and athletic club, today announced it has implemented ... freedom of movement for members and staff, while restricting ... comprehensive process, we selected FST,s IMID Access system because ... for our members and staff, in addition to unparalleled ...
(Date:3/23/2015)... SoundView Technology Group issues a new research update following the ... wallet. SoundView was one of the selected user groups to provide ... in multiple scenarios and outlets. Soundview shares ... both debit and credit card payments.  Soundview ... meets their plans in 2015, it would push our IV ...
(Date:3/20/2015)... DUBLIN , Mar. 19, 2015 Research and Markets ... the "Hand Geometry - Global Strategic Business Report" ... worldwide markets for Hand Geometry in US$ Thousands. The report ... , Japan , Europe ... America , and Rest of World. Annual ...
Breaking Biology News(10 mins):Granite Club First Private Club in North America to Implement FST Biometrics Secure Access Solution, as Part of Multi-million Dollar Expansion 2Technology Research Note Update from SoundView; Real-World Usage and Feedback of Wocket Smart Wallet at CVS, Whole Foods and other Retailers 2Technology Research Note Update from SoundView; Real-World Usage and Feedback of Wocket Smart Wallet at CVS, Whole Foods and other Retailers 3Technology Research Note Update from SoundView; Real-World Usage and Feedback of Wocket Smart Wallet at CVS, Whole Foods and other Retailers 4Hand Geometry - Global Strategic Business Report 2015: An Essential Security Component for both Government & Enterprise Sector 2Hand Geometry - Global Strategic Business Report 2015: An Essential Security Component for both Government & Enterprise Sector 3Hand Geometry - Global Strategic Business Report 2015: An Essential Security Component for both Government & Enterprise Sector 4
(Date:3/31/2015)... KY (PRWEB) March 31, 2015 ... 50 schools for online bachelor’s of criminal justice ... to connecting people to inspiring ideas and high ... an e-mail to the University. We are confident ... University.” , This is the site’s first ...
(Date:3/31/2015)... (PRWEB) March 31, 2015 Pittcon 2015, ... Applied Spectroscopy, ended on March 12, in New Orleans ... $21 million as estimated by the New Orleans Convention ... than 14,200 conferees and exhibitor personnel. Those who ... the conferee number. For the third year, the co-location ...
(Date:3/31/2015)... March 31, 2015  Bayer HealthCare (Bayer) has ... Massachusetts Institute of Technology (MIT) and Harvard University ... goal of this new part of the alliance ... help create new cardiovascular therapies. "We ... Broad Institute to the area of cardiovascular genomics ...
(Date:3/31/2015)...  The Myeloma Crowd Research Initiative (MCRI) completed ... proposals that will now be evaluated for further ... validated funding initiative uncovering solutions for high-risk multiple ... MCRI included preliminary vetting by the Scientific Advisory ... of innovative projects focusing on high-risk myeloma, we ...
Breaking Biology Technology:EKU Ranked in Top 50 Among Online Criminal Justice Degrees 2Pittcon 2015 Hits the Big Easy in a Big Way 2Pittcon 2015 Hits the Big Easy in a Big Way 3Pittcon 2015 Hits the Big Easy in a Big Way 4Bayer HealthCare Expands Collaboration with Broad Institute of MIT and Harvard to Develop Therapies for Cardiovascular Disease 2Bayer HealthCare Expands Collaboration with Broad Institute of MIT and Harvard to Develop Therapies for Cardiovascular Disease 3Myeloma Crowd Research Initiative Selects 10 High-Risk Multiple Myeloma Proposals For Further Review 2
Cached News: