AANS Neurosurgeon | Volume 27, Number 3, 2018

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Transplanted Hematopoietic Stem Cells Reverse Damage Caused by Neuro-Muscular Disorder

In mouse model of Friedreich’s ataxia, a single infusion measurably restored normal cellular functions

Researchers at University of California San Diego School of Medicine report that a single infusion of wildtype hematopoietic stem and progenitor cells (HSPCs) into a mouse model of Friedreich’s ataxia (FA) measurably halted cellular damage caused by the degenerative disease.

The findings suggest a potential therapeutic approach for a disease that currently is considered incurable. 

Friedreich’s ataxia is an inherited, degenerative neuromuscular disorder that initially impairs motor function, such as gait and coordination, but can lead to scoliosis, heart disease, vision loss and diabetes. Cognitive function is not affected. The disease is progressively debilitating, and ultimately requires full-time use of a wheelchair. One in 50,000 Americans has FA. 

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