Duchenne muscular dystrophy
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.
CRISPR Therapy in a Dish
CRISPR Therapy in a Dish
Kerry Grens | Dec 8, 2015
Redirecting the gene-editing tool to modulate gene expression, researchers restore protein function in cells from a child with Duchenne muscular dystrophy.
Fatal-Disease Drug in Limbo
Fatal-Disease Drug in Limbo
Jef Akst | Nov 25, 2015
A panel of experts advised the US Food and Drug Administration that BioMarin Pharmaceutical has not demonstrated efficacy of its new drug for Duchenne muscular dystrophy.
(Re)Programming Director
Karen Hopkin | Oct 1, 2012
Unwilling to accept the finality of terminal differentiation, Helen Blau has honed techniques that showcase the flexibility of cells to adopt different identities.