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:


(Date:1/13/2016)... New York , January 13, 2016 ... Market Research has published a new market report titled ... Growth, Trends, and Forecast, 2015 - 2023. According to the ... in 2014 and is anticipated to reach US$1,625.8 mn ... 2015 to 2023. In terms of volume, the biometric ...
(Date:1/8/2016)... and MANCHESTER, United Kingdom , Jan. ... developer of innovative sensor-based diagnostic products, today announced the closing ... new and existing investors.  Proceeds from the financing will be ... , a hand-held device for detecting early-stage pressure ulcers. ... Ireland after receiving CE Mark approval. The device,s ...
(Date:1/7/2016)... -- A United States District Court in Illinois ... to interpret a biometric privacy statute in a decision ... photo website Shutterfly brought by the law firm Carey Rodriguez ... SHUTTERFLY, INC.; and THISLIFE, INC ( N.D. Ill ., ... Illinois Biometric Privacy Act by collecting and scanning face ...
Breaking Biology News(10 mins):
(Date:2/8/2016)... program for the development of future natural products for crop ... and development center in Israel and ... Stockton has a variety of products adapted to ... in more than 35 counties worldwide. ... product Timorex Gold ® is used to control a ...
(Date:2/5/2016)... ATCC, the premier global biological materials resource ... and life science researchers that are working to address ... CDC website . --> CDC ... a single-stranded RNA virus of the Flaviviridae family, genus ... Chikungunya Viruses. Zika virus is transmitted to humans primarily ...
(Date:2/4/2016)... ... February 04, 2016 , ... Morf Media ... announced an interactive FDA compliance training course, Writing Effective SOPs ... Society) accredited interactive course on Morf Playbook—now conveniently available on smartphones and PCs--provides ...
(Date:2/4/2016)... , Feb. 4, 2016 Beike Biotechnology, ... various medical institutions attended a ceremony in late 2015 ... personalized cell therapy in 2016. --> ... Clinical Translation Platform for Personalized Cell Therapy" was hosted ... Cell Production Center, both subsidiaries of Beike Biotechnology Co., ...
Breaking Biology Technology:
Cached News: