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:4/18/2014)... researchers have published soil organic carbon sequestration rates. ... organic carbon can be sequestered by simply switching from ... there is a growing body of research with evidence ... cover crops, small grains, and forages may not be ... , "Some studies have shown that both moldboard and ...
(Date:4/18/2014)... Durham, NC Seeds that sprout as soon as they,re ... plants need to be more careful. In the wild, a ... rainy day would risk disaster. More than just an insurance ... out that seed dormancy has long-term advantages too: Plants ... give rise to more species, finds in a team of ...
(Date:4/18/2014)... led by a University of California, Riverside professor of ... Department of Defense grant to uncover fundamental design rules ... predictable design of light-weight, tough and strong advanced materials ... and animals, including the mantis shrimp, toucan and bamboo. ... over millions of years and coming up with design ...
Breaking Biology News(10 mins):Researchers question published no-till soil organic carbon sequestration rates 2Researchers question published no-till soil organic carbon sequestration rates 3Researchers question published no-till soil organic carbon sequestration rates 4Plants with dormant seeds give rise to more species 2Mantis shrimp, toucan and trilobite, oh my 2Mantis shrimp, toucan and trilobite, oh my 3
(Date:1/15/2014)... Cynvenio, a cancer diagnostics company focused ... analysis of tumor cells in the bloodstream, today announced ... decision support application powered by CollabRx, Inc. , ... test, Therapy Finder will provide breast cancer patients and ...
(Date:1/15/2014)... More than 5 million Americans are ... 3 seniors will die with Alzheimer’s or another dementia, ... have shocked many Americans into looking for ways to ... tragic age-related cognitive disorders. Jonathan Weisman, president of Biohack ...
(Date:1/15/2014)... Freeslate, Inc ., the leading provider of ... one of India’s top five pharmaceutical companies, has received ... for high throughput solid form screening. , Lupin, ... range of quality, affordable generic and branded formulations and ...
(Date:1/14/2014)... 2014 Histogen, Inc., a regenerative medicine ... grown under simulated embryonic conditions, today announced that they ... Medical, Inc. for physician-dispensed aesthetic products containing Histogen’s proprietary ... agreement is an amendment to the existing license between ...
Breaking Biology Technology:Cynvenio Offers New Breast Cancer Therapy Finder Application for its ClearID Breast Cancer Genomic Test 2Cynvenio Offers New Breast Cancer Therapy Finder Application for its ClearID Breast Cancer Genomic Test 3Biohack Pure Offers 5 Tips for Increasing Memory in 2014 2Lupin Selects Freeslate’s CM Protégé PharmD System to Accelerate Polymorph Screening for Drug Development 2Lupin Selects Freeslate’s CM Protégé PharmD System to Accelerate Polymorph Screening for Drug Development 3Histogen and Suneva Medical Expand License for Cell Conditioned Media-based Aesthetic Products Internationally 2Histogen and Suneva Medical Expand License for Cell Conditioned Media-based Aesthetic Products Internationally 3
Cached News: