The first three infants in a gene therapy trial for spinal muscular atrophy have been treated; the trial continues, and additional trials are planned
Dallas-based biotechnology company AveXis, which is developing gene therapy for spinal muscular atrophy (SMA), announced in October that administration of its experimental gene transfer compound to the first three patients in a
An MDA-supported research team has found in animal experiments that plasmapheresis can reduce the number of unwanted antibodies to gene therapy delivery vehicles
A research group supported in part by MDA has found that a blood-filtering strategy known as plasmapheresis looks promising as a way to help overcome one type of unwanted immune response to gene transfer therapy. Gene transfer therapy is the addition of therapeutic genes to treat disease, and it is often administered via transport vehicles made from viruses.
Louis Chicoine at Nationwide Children'...
Early data show follistatin gene transfer may be safe and effective in BMD and safe in IBM, in which efficacy has not yet been evaluated
Preliminary results from a trial to test the safety of injecting follistatin genes into the thigh muscles of adults with Becker muscular dystrophy (BMD)or sporadic (nongenetic) inclusion-body myositis (sIBM) suggest that the experimental therapeutic approach is safe in both types of patients
In a podcast from Nationwide Children’s Hospital, longtime MDA grantee Jeffrey Chamberlain describes advances in gene transfer therapy and stem cell therapy for Duchenne muscular dystrophy
In a July 2013 podcast from Nationwide Children’s Hospital in Columbus, Ohio, longtime MDA grantee Jeffrey Chamberlain discusses recent advances in the development of gene therapy (gene transfer) and stem cell therapy (transplantation) for Duchenne muscular dystrophy (DMD), the results of which may apply to other types of muscular dy
A new zebrafish research model may speed myofibrillar myopathy research; published results of a gene transfer study in LGMD are now accessible
Update (Aug. 8, 2012): This story was updated to reflect the availability of a podcast on the dysferlin gene transfer study.
Zebrafish research models mimic myofibrillar myopathy
Investigators have safely injected gamma-sarcoglycan genes into an arm muscle in nine people with limb-girdle muscular dystrophy type 2C; high-dose recipients produced gamma-sarcoglycan protein
A March 2012 podcast from Nationwide Children's Hospital in Columbus, Ohio, presents the results of a phase 1 trial of gene therapy for gamma-sarcoglycan-deficient limb-girdle muscular dystrophy (LGMD), also known as LGMD2C.
Two longtime MDA grantees receive prestigious award
On May 3, 2009, molecular biologist Louis Kunkel at Children’s Hospital in Boston and Harvard University, and biophysicist Kevin Campbell at the University of Iowa, received the prestigious March of Dimes Prize in Developmental Biology. The prize includes a $250,000 cash award.