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:7/31/2015)... SHENZHEN , Chine, 31 juillet 2015 /PRNewswire/ ... (ICG-10, www.icg-10.org ) sera organisée par le ... Shenzhen en Chine. ... année. Depuis son inauguration en 2006, l,ICG est ... influentes dans le domaine des « omiques » et c,est ...
(Date:7/30/2015)... Calif. , July 30, 2015 ... services for gene function analysis and biomarker discovery, ... RNA (sgRNA) Knockout Library targeting all human protein ... and permanently "knock out" a gene,s function. Cellecta,s ... a high throughput screening tool so that researchers ...
(Date:7/21/2015)... , July 21, 2015 Today, ZTE announced ... received as well as expected revenues in 2015 that relate to ... communicated revenue guidance of approximately 2,200 MSEK for 2015. ... a leading smartphone manufacturer in China and ... 02 5 for Axon , ...
Breaking Biology News(10 mins):La 10e International Conference on Genomics (ICG-10) doit s'ouvrir en octobre 2La 10e International Conference on Genomics (ICG-10) doit s'ouvrir en octobre 3CELLECTA, INC. Announces Launch of Human Whole Genome CRISPR Knockout Library 2FPC's Touch Fingerprint Sensor FPC1025 in ZTE's Smartphone Axon 2
(Date:7/30/2015)... HIGHLIGHTS:Q2 2015 Results (all changes ... Reported sales were $697 million compared to $701 million ... by 8%, and changes in foreign currency exchange rates ... 1%. , By business unit, organic sales growth ... in SAFC Commercial. , Reported diluted EPS was ...
(Date:7/29/2015)... 2015 Sanofi, un leader ... résultats pour le premier semestre 2015. Le Directeur ... Visionner l,interview vidéo et lire la ... Au sommaire de l,interview :  ... croissance - Diabète - ...
(Date:7/29/2015)...  HealthSouth Corporation (NYSE: HLS ), ... healthcare services, offering both facility-based and home-based post-acute ... the second quarter ended June 30, 2015. ... volume growth in both segments and an 11.0% ... President and Chief Executive Officer of HealthSouth. "While ...
(Date:7/29/2015)... , July 29, 2015  AmnioChor Inc., an early ... is very pleased to announce that the Musculoskeletal Transplant ... agreed to invest in their seed round of development ... tissue storage capabilities. AmnioChor,s technology allows ... the human stem cells living within those tissues. Amnion ...
Breaking Biology Technology:Sigma-Aldrich (NASDAQ - SIAL) Reports Q2 2015 Sales Of $697 Million And Adjusted Diluted EPS Of $1.01 2Sigma-Aldrich (NASDAQ - SIAL) Reports Q2 2015 Sales Of $697 Million And Adjusted Diluted EPS Of $1.01 3Sigma-Aldrich (NASDAQ - SIAL) Reports Q2 2015 Sales Of $697 Million And Adjusted Diluted EPS Of $1.01 4Sigma-Aldrich (NASDAQ - SIAL) Reports Q2 2015 Sales Of $697 Million And Adjusted Diluted EPS Of $1.01 5Sigma-Aldrich (NASDAQ - SIAL) Reports Q2 2015 Sales Of $697 Million And Adjusted Diluted EPS Of $1.01 6Sigma-Aldrich (NASDAQ - SIAL) Reports Q2 2015 Sales Of $697 Million And Adjusted Diluted EPS Of $1.01 7Sigma-Aldrich (NASDAQ - SIAL) Reports Q2 2015 Sales Of $697 Million And Adjusted Diluted EPS Of $1.01 8Sigma-Aldrich (NASDAQ - SIAL) Reports Q2 2015 Sales Of $697 Million And Adjusted Diluted EPS Of $1.01 9Sigma-Aldrich (NASDAQ - SIAL) Reports Q2 2015 Sales Of $697 Million And Adjusted Diluted EPS Of $1.01 10Sigma-Aldrich (NASDAQ - SIAL) Reports Q2 2015 Sales Of $697 Million And Adjusted Diluted EPS Of $1.01 11Sigma-Aldrich (NASDAQ - SIAL) Reports Q2 2015 Sales Of $697 Million And Adjusted Diluted EPS Of $1.01 12Sigma-Aldrich (NASDAQ - SIAL) Reports Q2 2015 Sales Of $697 Million And Adjusted Diluted EPS Of $1.01 13Sigma-Aldrich (NASDAQ - SIAL) Reports Q2 2015 Sales Of $697 Million And Adjusted Diluted EPS Of $1.01 14Sigma-Aldrich (NASDAQ - SIAL) Reports Q2 2015 Sales Of $697 Million And Adjusted Diluted EPS Of $1.01 15Sigma-Aldrich (NASDAQ - SIAL) Reports Q2 2015 Sales Of $697 Million And Adjusted Diluted EPS Of $1.01 16Sigma-Aldrich (NASDAQ - SIAL) Reports Q2 2015 Sales Of $697 Million And Adjusted Diluted EPS Of $1.01 17Sigma-Aldrich (NASDAQ - SIAL) Reports Q2 2015 Sales Of $697 Million And Adjusted Diluted EPS Of $1.01 18Sigma-Aldrich (NASDAQ - SIAL) Reports Q2 2015 Sales Of $697 Million And Adjusted Diluted EPS Of $1.01 19Sigma-Aldrich (NASDAQ - SIAL) Reports Q2 2015 Sales Of $697 Million And Adjusted Diluted EPS Of $1.01 20HealthSouth Reports Results for Second Quarter 2015 2HealthSouth Reports Results for Second Quarter 2015 3HealthSouth Reports Results for Second Quarter 2015 4HealthSouth Reports Results for Second Quarter 2015 5HealthSouth Reports Results for Second Quarter 2015 6HealthSouth Reports Results for Second Quarter 2015 7HealthSouth Reports Results for Second Quarter 2015 8HealthSouth Reports Results for Second Quarter 2015 9HealthSouth Reports Results for Second Quarter 2015 10HealthSouth Reports Results for Second Quarter 2015 11HealthSouth Reports Results for Second Quarter 2015 12HealthSouth Reports Results for Second Quarter 2015 13HealthSouth Reports Results for Second Quarter 2015 14HealthSouth Reports Results for Second Quarter 2015 15HealthSouth Reports Results for Second Quarter 2015 16HealthSouth Reports Results for Second Quarter 2015 17
Cached News: