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
A phase 1 gene transfer trial in nine babies with type 1 spinal muscular atrophy has opened at Nationwide Children's Hospital in Columbus, Ohio
A phase 1 trial to test the safety and efficacy of gene transfer therapy in infants with type 1 spinal muscular atrophy (SMA) who are 9 months old or younger has opened at Nationwide Children's Hospital in Columbus, Ohio, under the direction of neurologist Jerry Mendell.
A look at how today's gene therapy research for DMD is overcoming obstacles
The year was 1986, and the Duchenne muscular dystrophy (DMD) community was more excited than it had been for decades. A group of MDA-supported researchers had identified the genetic cause of the disorder — any of a number of different flaws (“mutations”) in the gene for a newly identified muscle protein, one that would come to be called dystrophin.
Researcher Carmen Bertoni is using DNA-like material to fix flawed genes
When Carmen Bertoni came to the University of Tulane in New Orleans from her native Italy back in 1995 to study the burgeoning science of molecular genetics, she knew she wanted to study genetic disorders but not solely to advance the field. "I wanted to work on something that would potentially be curable within my lifetime," she says.
Dongsheng Duan is tweaking dystrophin genes to improve their therapeutic efficacy, designing better viral vectors in which to package the genes, and aims to treat the heart with gene transfer
By the time Dongsheng Duan finished medical school at West China University of Medical Sciences in Sichuan Province in the late 1980s, he knew he wanted to study biological sciences in the United States. Political turmoil in China temporarily derailed his plans, but in 1993, he left his home country. By the following year, he was at the University of Pennsylvania, working with Katherine High, an...
Charles Gersbach is using “nuclease-based genome correction” to turn flawed dystrophin genes into functional ones
In 2001, Charles Gersbach began his graduate studies in biomedical engineering at the Georgia Institute of Technology in Atlanta, joining a center that was working on constructing semi-artificial muscle, bone and cartilage tissues.
He was interested in constructing new materials and making tissue implants, but what intrigued him more were the genetic underpinnings of tissue growth and...
Jeffrey Chamberlain believes that providing new dystrophin genes is the best way to treat Duchenne muscular dystrophy, although combining gene transfer with other strategies interests him.
Jeffrey Chamberlain’s interest in Duchenne muscular dystrophy (DMD) began in 1986, when mutations in the dystrophin gene were first identified as the underlying cause of the disease.
At that time, Chamberlain was working in a lab at Baylor College of Medicine in Houston, trying to find out what was causing weakness in mice that seemed to have some type of muscular dystrophy. He began to think the...