Health Discoveries in Movement Disorders, Parkinsons etc
Major grant funds gene therapy for muscular dystrophy
August 12, 2010
The Nationwide Children's Hospital in Columbus, Ohio, has received a $600,000 grant to conduct clinical testing of gene therapy aimed at improving muscle function in those with muscular dystrophy.
The award by the Parent Project Muscular Dystrophy (PPMD), a non-profit organization, focused on finding a cure for the disease.
Dr. Jerry Mendell, director of the hospital's Center for Gene Therapy, and researcher Brian Kaspar will head the project, which will involve injecting participants with a vector carrying the gene to stimulate muscle growth. The injection site will be the quadriceps muscles that are particularly weakened by muscular dystrophy.
The study is expected to start in early 2011.
"This is the first time a gene therapy approach has been used to supply genes that generically stimulate muscle growth rather than directly replacing missing muscle proteins, " said Sharon Hesterlee, PPMD's senior director of research and advocacy. "Other applications could include the treatment of muscles that have been injured directly through accidents or indirectly through disuse."
Within the North Shore-LIJ Health System, the Neuromuscular and Peripheral Nerve Disease Program addresses numerous diseases that affect the peripheral nervous system such as muscular dystrophy, Bell's palsy, amyotrophic lateral sclerosis (ALS) or Lou Gehrig's Disease, myasthenia gravis, fibromuscular dysplasia and shingles.
The award by the Parent Project Muscular Dystrophy (PPMD), a non-profit organization, focused on finding a cure for the disease.
Dr. Jerry Mendell, director of the hospital's Center for Gene Therapy, and researcher Brian Kaspar will head the project, which will involve injecting participants with a vector carrying the gene to stimulate muscle growth. The injection site will be the quadriceps muscles that are particularly weakened by muscular dystrophy.
The study is expected to start in early 2011.
"This is the first time a gene therapy approach has been used to supply genes that generically stimulate muscle growth rather than directly replacing missing muscle proteins, " said Sharon Hesterlee, PPMD's senior director of research and advocacy. "Other applications could include the treatment of muscles that have been injured directly through accidents or indirectly through disuse."
Within the North Shore-LIJ Health System, the Neuromuscular and Peripheral Nerve Disease Program addresses numerous diseases that affect the peripheral nervous system such as muscular dystrophy, Bell's palsy, amyotrophic lateral sclerosis (ALS) or Lou Gehrig's Disease, myasthenia gravis, fibromuscular dysplasia and shingles.