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image: Optogenetic Therapies Move Closer to Clinical Use

Optogenetic Therapies Move Closer to Clinical Use

By | November 16, 2017

With a clinical trial underway to restore vision optogenetically, researchers also see promise in using the technique to treat deafness, pain, and other conditions.

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image: Man Receives First In Vivo Gene-Editing Therapy

Man Receives First In Vivo Gene-Editing Therapy

By | November 15, 2017

The 44-year-old patient has Hunter syndrome, which doctors hope to treat using zinc finger nucleases.

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image: Child Receives Transgenic Skin Over Most of His Body

Child Receives Transgenic Skin Over Most of His Body

By | November 8, 2017

A combination gene-and-cell therapy has given a boy with a grievous skin disease a new lease on life, and has resolved a dermatology debate to boot.

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Spark Therapeutics’s Luxturna would be the first approved therapy in the U.S. that replaces or repairs a defective gene inherited from one’s parents.

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image: Nonviral CRISPR Delivery a Success

Nonviral CRISPR Delivery a Success

By | October 2, 2017

Researchers use gold nanoparticles to deliver CRISPR-Cas9 and correct a point mutation in a mouse model of Duchenne muscular dystrophy. 

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image: Skin Graft-based Gene Therapy Treats Diabetes in Mice

Skin Graft-based Gene Therapy Treats Diabetes in Mice

By | August 4, 2017

A small patch of engineered cells makes an enzyme that stimulates insulin release.

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image: Dogs with Duchenne Treated with Gene Therapy

Dogs with Duchenne Treated with Gene Therapy

By | July 25, 2017

Researchers restored muscle function in animals with muscular dystrophy.

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image: FDA Votes Yes on CAR T-Cell Therapy

FDA Votes Yes on CAR T-Cell Therapy

By | July 13, 2017

A Food and Drug Administration advisory panel unanimously calls for agency approval of the cell therapy for the treatment of resistant leukemia. 

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Sangamo Therapeutics will use zinc finger nucleases to introduce the gene for a missing clotting factor into the livers of men with hemophilia B.

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A small Phase 1 clinical trial reports that, even at high doses, the treatment did not lead to adverse reactions.

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