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» Duchenne muscular dystrophy

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Adding RNA sequencing analysis to genomic sequencing helps scientists uncover mutations likely responsible for genetic disorders they might otherwise miss.

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image: CRISPR Corrects Duchenne-Causing Mutations

CRISPR Corrects Duchenne-Causing Mutations

By | April 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.

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image: Conditional FDA Approval for Fatal-Disease Drug

Conditional FDA Approval for Fatal-Disease Drug

By | September 19, 2016

The agency OKs Sarepta Therapeutics’s treatment for Duchenne muscular dystrophy through its accelerated approval pathway, which requires a confirmatory clinical trial.

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image: Another Fatal-Disease Drug in Limbo

Another Fatal-Disease Drug in Limbo

By | April 26, 2016

A federal advisory panel votes against Sarepta Therapeutics’s treatment for Duchenne muscular dystrophy.

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image: First Data from Anti-Aging Gene Therapy

First Data from Anti-Aging Gene Therapy

By | April 25, 2016

A biotech company reports that an experimental treatment elongated its CEO’s telomeres. 

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image: CRISPR Improves Disease in Adult Mice

CRISPR Improves Disease in Adult Mice

By | January 4, 2016

Three groups of researchers used the gene-editing method to restore a protein deficient in Duchenne muscular dystrophy.

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image: CRISPR Therapy in a Dish

CRISPR Therapy in a Dish

By | December 8, 2015

Redirecting the gene-editing tool to modulate gene expression, researchers restore protein function in cells from a child with Duchenne muscular dystrophy.

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image: Fatal-Disease Drug in Limbo

Fatal-Disease Drug in Limbo

By | November 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.

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image: (Re)Programming Director

(Re)Programming Director

By | October 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.

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