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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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Scientists shut down cancer-causing fusion genes with CRISPR.

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image: Smallest Cas9 Ortholog Reported

Smallest Cas9 Ortholog Reported

By | February 23, 2017

Researchers demonstrate the delivery of AAV-packaged CRISPR-CjCas9 into the muscles and eyes of mice.

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image: Mouse Immunology Paper Retracted

Mouse Immunology Paper Retracted

By | December 16, 2016

A finding of misconduct spurs the retraction of a Science paper claiming to have identified a protein in mice that boosted immunity to both viruses and cancer.

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image: TS Picks: August 9, 2016

TS Picks: August 9, 2016

By | August 9, 2016

Gene therapy money-back guarantee; the brain benefits from bilingualism; Q&A with a science watchdog

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image: Improving Gene Therapy with In Vivo Selection

Improving Gene Therapy with In Vivo Selection

By | June 8, 2016

Scientists have developed a way to selectively expand genetically modified hepatocytes in mice.

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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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