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National Ataxia Foundation

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CRISPR

Sunrise of Gene Therapy for Friedreich’s Ataxia

Written by Dr. Marija Cvetanovic   Edited by Dr. Ronald Buijsen Researchers from the University of California show they can “edit” the Frataxin gene in human cells from Friedreich’s Ataxia and transplant them into mice. This lays the groundwork for this method to be tested for safety. Friedreich’s ataxia is a Read More…

Snapshot: What are stem cells?

Embryonic and adult stem cells Stem cells are cells that provide new cells during growth, and replace cells that are damaged or lost during life. They have the following two important properties that enable them to do this: The ability to develop (differentiate) into many other, different cell types, for Read More…

Snapshot: What is CRISPR?

A common nuisance for bacteria is the bacteriophage: a virus that uses the internal machinery of a bacteria to replicate its own genetic material. Bacteriophages do this by latching onto bacteria and injecting their DNA into the cell. As the cell grows and divides, the bacteriophage’s hope is that their Read More…

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