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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: Image of the Day: Fragile Brain

Image of the Day: Fragile Brain

By | October 3, 2017

In Fragile X syndrome—a genetic mishap that results in cognitive delays—the lack of a translation-repressing protein leads to the rampant accumulation of other proteins in the mouse brain.

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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: Infographic: Macrophages Around the Body

Infographic: Macrophages Around the Body

By | October 1, 2017

In addition to circulating in the blood as immune sentinels, macrophages play specialized roles in different organs around the body.

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image: Macrophages Are the Ultimate Multitaskers

Macrophages Are the Ultimate Multitaskers

By | October 1, 2017

From guiding branching neurons in the developing brain to maintaining a healthy heartbeat, there seems to be no job that the immune cells can’t tackle.

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image: Water Level in a Cell Can Determine Its Fate

Water Level in a Cell Can Determine Its Fate

By | September 27, 2017

Adding or removing water changes how stem cells differentiate.

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image: CRISPR Used in Human Embryos to Probe Gene Function

CRISPR Used in Human Embryos to Probe Gene Function

By | September 20, 2017

OCT4 is necessary for blastocyst formation in the human embryo, researchers report.

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A study of a simple marine animal suggests that the common ancestor of cnidarians and bilaterians may have had three germ layers instead of two.

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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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