Positive Trial Results for Experimental DMD Gene Therapy
Preliminary data from Sarepta Therapeutics has exceeded expectations, causing a surge in company stock prices this week.
Positive Trial Results for Experimental DMD Gene Therapy
Positive Trial Results for Experimental DMD Gene Therapy

Preliminary data from Sarepta Therapeutics has exceeded expectations, causing a surge in company stock prices this week.

Preliminary data from Sarepta Therapeutics has exceeded expectations, causing a surge in company stock prices this week.

Duchenne muscular dystrophy
Nonviral CRISPR Delivery a Success
Nonviral CRISPR Delivery a Success
Abby Olena | Oct 2, 2017
Researchers use gold nanoparticles to deliver CRISPR-Cas9 and correct a point mutation in a mouse model of Duchenne muscular dystrophy. 
Dogs with Duchenne Treated with Gene Therapy
Dogs with Duchenne Treated with Gene Therapy
Diana Kwon | Jul 25, 2017
Researchers restored muscle function in animals with muscular dystrophy.
CRISPR Restores Muscle Function in Mice
CRISPR Restores Muscle Function in Mice
Diana Kwon | Jul 17, 2017
Scientists use the gene-editing tool to treat animals with a rare form of congenital muscular dystrophy.
RNA-Seq Reveals Previously Hidden, Genetic Disorder–Causing Mutations
RNA-Seq Reveals Previously Hidden, Genetic Disorder–Causing Mutations
Anna Azvolinsky | Apr 19, 2017
Adding RNA sequencing analysis to genomic sequencing helps scientists uncover mutations likely responsible for genetic disorders they might otherwise miss.
CRISPR Corrects Duchenne-Causing Mutations
CRISPR Corrects Duchenne-Causing Mutations
Anna Azvolinsky | Apr 12, 2017
Using CRISPR-Cpf1 gene editing, researchers have fixed mutations that cause a form of muscular dystrophy in cultured human cardiomyocytes and a mouse model.
Conditional FDA Approval for Fatal-Disease Drug
Conditional FDA Approval for Fatal-Disease Drug
Tracy Vence | Sep 19, 2016
The agency OKs Sarepta Therapeutics’s treatment for Duchenne muscular dystrophy through its accelerated approval pathway, which requires a confirmatory clinical trial.
Another Fatal-Disease Drug in Limbo
Another Fatal-Disease Drug in Limbo
Jef Akst | Apr 26, 2016
A federal advisory panel votes against Sarepta Therapeutics’s treatment for Duchenne muscular dystrophy.
First Data from Anti-Aging Gene Therapy
First Data from Anti-Aging Gene Therapy
Kerry Grens | Apr 25, 2016
A biotech company reports that an experimental treatment elongated its CEO’s telomeres. 
CRISPR Improves Disease in Adult Mice
CRISPR Improves Disease in Adult Mice
Kerry Grens | Jan 4, 2016
Three groups of researchers used the gene-editing method to restore a protein deficient in Duchenne muscular dystrophy.