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:12/15/2014)... 12, 2014 Research and Markets ... the "Global Facial Recognition Market 2015-2019" ... http://photos.prnewswire.com/prnh/20130307/600769 Facial recognition is ... Facial recognition system measures the overall facial feature ... mouth, and the distance between eyes. Facial recognition ...
(Date:12/10/2014)... , Dec. 8, 2014 You,ve been here before: ... but can,t remember your password, site key or the answer ... your first grade teacher? Today, Hoyos ... app that will finally put an end to the ... 1U TM . 1U leverages a user,s smartphone to ...
(Date:12/10/2014)... , December 9, 2014 ... BCARD Platform   ... meeting scheduling solutions for business-to-business (B2B) events, today ... leader in mobile near-field communication (NFC), Bluetooth low ...      (Logo: http://photos.prnewswire.com/prnh/20140612/691055) , Jifflenow will ...
Breaking Biology News(10 mins):Global Facial Recognition Market 2015-2019: Key Vendors are 3M Cogent, Cognitec Systems, NEC and Safran Group 2Global Facial Recognition Market 2015-2019: Key Vendors are 3M Cogent, Cognitec Systems, NEC and Safran Group 3The Password is Finally Dead: Launch of 1U Mobile App Eliminates Need for All Usernames and Passwords 2The Password is Finally Dead: Launch of 1U Mobile App Eliminates Need for All Usernames and Passwords 3Jifflenow And ITN International Bring Cutting-Edge Badge Scanning Technology To B2B Events 2Jifflenow And ITN International Bring Cutting-Edge Badge Scanning Technology To B2B Events 3
(Date:12/19/2014)... Ed Bilsky, Ph.D., vice president for Research and ... the Neurosciences (CEN), and professor of pharmacology at the ... member of the prestigious Dana Alliance for Brain Initiatives ... receive this honor. , The Dana Alliance ... Foundation. Its mission is to advance public education ...
(Date:12/19/2014)... Diego, CA (PRWEB) December 18, 2014 ... a limited time special on Sartorius Biohit products ... Free on mLINE pipettes, Picus Electronic Pipette Trade-in Program, ... by Health and Safety officers around the world. They ... manual pipetting. Other features include:, ,     Full ...
(Date:12/17/2014)... York, NY (PRWEB) December 16, 2014 ... significant changes since 2013, which is why IBISWorld updated ... this industry continues to benefit from an intensified focus ... the proposal for new emission standards for power plants ... industry operators. According to IBISWorld Industry Analyst Sarah Kahn, ...
(Date:12/17/2014)... Once more, EMAAR has selected BioShaft ... water treatment plant to phase one of BayLaSun, a ... years ago Bioshaft successfully supplied and operated a packaged ... towers with an occupancy of 900 residences. The ... per day and was signed on September 16th, 2014, ...
Breaking Biology Technology:UNE's Ed Bilsky Named Member of Dana Alliance for Brain Initiatives 2UNE's Ed Bilsky Named Member of Dana Alliance for Brain Initiatives 3Major Pipette Distributor Pipette.com Announces Sartorius Year End Specials 2Activated Carbon Manufacturing in the US Industry Market Research Report Has Been Updated from IBISWorld 2Activated Carbon Manufacturing in the US Industry Market Research Report Has Been Updated from IBISWorld 3
Cached News: