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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: <em>The Scientist</em> on The Pulse, December 4

The Scientist on The Pulse, December 4

By | December 4, 2015

Are precision gene editing technologies, such as CRISPR, ready for prime time?

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image: Gene Editing Treats Leukemia

Gene Editing Treats Leukemia

By | November 6, 2015

One-year-old Layla Richards has remained cancer-free months after receiving an experimental gene editing therapy.

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image: Phase 3 Win for Gene Therapy

Phase 3 Win for Gene Therapy

By | October 6, 2015

The treatment restored sight among people with an inherited visual impairment.

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image: Hearing Channel Components Mapped

Hearing Channel Components Mapped

By | September 4, 2015

Localization of two proteins important for inner ear hair cell function suggests they are part of the elusive mechanotransduction channel. 

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image: Redirecting Gene Therapy Restores Sight

Redirecting Gene Therapy Restores Sight

By | August 17, 2015

By targeting rhodopsin genes to neurons, scientists help blind mice see.

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image: Ancient Viruses as Gene Therapy Vectors

Ancient Viruses as Gene Therapy Vectors

By | July 31, 2015

Researchers deploy ancestors of today’s adeno-associated viruses to deliver gene therapies without immune system interference.

2 Comments

image: Gene Therapy Fixes Mouse Hearing

Gene Therapy Fixes Mouse Hearing

By | July 9, 2015

Expressing a gene for a component of the inner ear’s hair cells treated a form of genetic deafness.

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image: CF Gene Therapy Shows Promise

CF Gene Therapy Shows Promise

By | July 6, 2015

The results of a Phase 2 trial suggest that delivering normal copies of the gene that causes cystic fibrosis may slow lung decline.

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image: Celladon Shutters Its R&D

Celladon Shutters Its R&D

By | June 26, 2015

Biotech firm likely to pull the plug after its gene therapy product fails.

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