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The Scientist

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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.

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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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image: Restored Sight Fades After Gene Therapy

Restored Sight Fades After Gene Therapy

By | May 5, 2015

Participants of two gene-therapy trials who experienced partial restoration of sight following treatment are now losing their vision once again.

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image: Protein Spurs T-Cell Proliferation

Protein Spurs T-Cell Proliferation

By | April 17, 2015

A newly discovered protein promotes immunity to viruses and cancer by triggering the production of cytotoxic T cells.

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image: Reducing Gene Therapy-Related Risk

Reducing Gene Therapy-Related Risk

By | January 22, 2015

In a mouse model of a rare disease, scientists have figured out how to reduce the elevated cancer risk tied to a gene therapy treatment.

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image: Next Generation: Fat-Targeted Gene Knockdown

Next Generation: Fat-Targeted Gene Knockdown

By | October 5, 2014

A small peptide helps a silencing construct home in on the adipocytes of obese mice.

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image: Gene Therapy Researcher Faked Data

Gene Therapy Researcher Faked Data

By | April 25, 2014

A former postdoc in a prominent gene therapy lab is branded a fraud by the US government more than three years after having a slew of papers retracted from various journals.

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