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The Scientist

» Duchenne muscular dystrophy

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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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