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image: Gene Therapy Temporarily Reverses Type 1 Diabetes in Mice

Gene Therapy Temporarily Reverses Type 1 Diabetes in Mice

By | January 4, 2018

Pancreatic cells engineered to produce insulin did not immediately provoke an immune response.

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image: Child Receives Transgenic Skin

Child Receives Transgenic Skin

By | January 1, 2018

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

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image: CRISPR Proves Promising for Treating ALS in Mice

CRISPR Proves Promising for Treating ALS in Mice

By | December 21, 2017

The gene-editing tool was effective in disabling a defective gene responsible for some forms of amyotrophic lateral sclerosis. 

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image: CRISPR Helps Mice Hear

CRISPR Helps Mice Hear

By | December 20, 2017

Researchers reduce the severity of hereditary deafness in mice with the delivery of CRISPR-Cas9 protein-RNA complexes that inactivate a mutant gene in their inner ears. 

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Upping a gene’s expression in rat brains made them better learners and normalized the activity of hundreds of other genes to resemble the brains of younger animals.

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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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