Navigation Links
Trial Of DMD Gene Therapy Initiated At Ohio

Jerry Mendell a neurologist, co-director of the Muscular Dystrophy Association (MDA) clinic at Columbus Children’s Hospital; professor of pediatrics, neurology and pathology at Ohio State University College of Medicine;// and head of the Neuromuscular Research Program and Gene Therapy Center at Columbus Children’s Research Institute. He administered an injection of Asklepios Biopharmaceutical Inc (AskBio’s) Biostrophin, which contains a functional gene for the muscle protein dystrophin, into the biceps of Andrew Kilbarger, 8, of Lancaster, Ohio.

He initiated the first U.S. human gene therapy trial directed at Duchenne muscular dystrophy (DMD) at Columbus (Ohio) Children’s Hospital. It is a long-awaited step in the overall plan to help kids with muscular dystrophy. He said that it is hoped by everyone that this effort will help guide us toward clinically meaningful results as we move forward with this form of treatment. Sharon Hesterlee, MDA Director of Research Development said that it was with great difficulty that this gene therapy trail was possible a sit had to overcome the scientific, technical and regulatory hurdles. The gene for dystrophin was identified in 1986 by MDA-supported researchers.

The transformation was very exciting from the academic institutions, where basic science concepts are developed into the industry, where concepts became products. She also said that it would not have been possible if Asklepios had not taken a big risk of combining business, academic and nonprofit players. 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 at the age of 20 as a result of respiratory or cardiac muscle failure. This is due to the gene mutation on the X chromosome. This results in the failure of the production of the essential muscle protein dystrophin. This gene is the largest known gene. Hence it was with great effort that it w as miniatured while retaining at the same time the crucial elements of the DNA.

AskBio’s Biostrophin therapy delivers the miniaturized replacement dystrophin gene with the help of Biological Nano Particles (BNP). BNP’s carry the minidystrophin gene to muscle cells. The Food and Drug Administration gave the team a green light to proceed with the human trial on March 3. The trial will test six boys with DMD, each of whom will receive minidystrophin genes in a muscle of one arm and a placebo in the other arm. Neither the investigators nor the participants will know which muscle got the dystrophin 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 would confirm the effectiveness of the therapy.
'"/>




Related medicine news :

1. Results Of Human Trials Of Hungarian Bird-Flu Vaccine Expected Today
2. Vaccine For Dengue Fever On Trial In Asia-Pacific
3. DNA Vaccine Trials Against HIV Virus In Sweden Nurture Hope
4. Clinical Trials of New Drug Febuxostat Show Promise in Gout Treatment
5. Sex, Race Influence Lung Cancer Trial Participation
6. Clinical Trials Of New Anti-Cancer Drug To Be Initiated In New Zealand
7. Diarrhea Vaccine Trials Promises Prevention of Rotavirus Infection
8. New Generation Clinical Trials Could Save Time And Money, Improve Patient Care
9. China Begins Human Trials For HIV/AIDS Drug
10. Patients Should Be Entitled to "Humane Compensation" if Drug Trials Go Wrong
11. HIV/AIDS Vaccine Trial To Be Initiated Soon
Post Your Comments:
*Name:
*Comment:
*Email:


(Date:6/27/2016)... (PRWEB) , ... June 27, 2016 , ... "FCPX editors ... customizable inside of Final Cut Pro X," said Christina Austin - CEO of Pixel ... style. Final Cut Pro X users can now reveal the media of ...
(Date:6/27/2016)... ... June 27, 2016 , ... TopConsumerReviews.com recently awarded their highest five-star rating ... Millions of individuals in the United States and Canada wear eyeglasses. Once considered to ... correct vision and make a fashion statement. Even celebrities use glasses as a way ...
(Date:6/26/2016)... Carolina (PRWEB) , ... June 26, 2016 , ... ... of a new product that was developed to enhance the health of felines. The ... centuries. , The two main herbs in the PawPaws Cat Kidney Support ...
(Date:6/25/2016)... ... June 25, 2016 , ... Austin residents seeking Mohs surgery services, can ... Surgery and to Dr. Russell Peckham for medical and surgical dermatology. , Dr. Dorsey ... cancer. The selective fellowship in Mohs Micrographic Surgery completed by Dr. Dorsey was under ...
(Date:6/25/2016)... ... June 25, 2016 , ... As a lifelong Southern Californian, ... and his M.D from the David Geffen School of Medicine at UCLA. He trained ... Angeles to complete his fellowship in hematology/oncology at the UCLA-Olive View-Cedars Sinai program where ...
Breaking Medicine News(10 mins):
(Date:6/23/2016)... 23, 2016 Research and Markets has ... Market Analysis 2016 - Forecast to 2022" report to ... report contains up to date financial data derived from varied ... major trends with potential impact on the market during the ... segmentation which comprises of sub markets, regional and country level ...
(Date:6/23/2016)... Ill. and INDIANAPOLIS , ... students receiving a Lilly Diabetes Tomorrow,s Leaders Scholarship is ... 2016 scholarship winners, announced today online at www.diabetesscholars.org ... let type 1 diabetes stand in the way of ... has supported the Foundation,s scholarship program since 2012, and ...
(Date:6/23/2016)... Research and Markets has announced the addition ... States, China, Japan, Brazil, United Kingdom, Germany, France, Italy, ... Surgical Procedure Volumes: Global ... surgical procedure volume data in a geographic context. The ... of growth drivers and inhibitors, including world population growth, ...
Breaking Medicine Technology: