Harvard scientists find Compound protects nerve cells targeted by diseases
Harvard scientists find evidence that ALS and SMA could be treated with a common drug
Harvard Stem Cell Institute (HSCI) researchers have identified a compound that helps protect the cells destroyed by spinal muscular atrophy (SMA), the most frequent fatal genetic disease in children under 2 years of age.
SMA is a neurodegenerative disease targeting motor neurons, the long nerve cells that relay messages from the brain to the muscles and that are, consequently, responsible for bodily movements, including walking, swallowing, and even breathing. Patients with milder forms of SMA experience muscle wasting that may confine them to a wheelchair, while the more severe forms cause paralysis and death before the second birthday.
The findings were published in the journal Cell Reports.
“This discovery opens up new lines of drug interrogation,” said Lee Rubin, HSCI principal faculty member and the senior author on the study. Rubin’s lab, which operates out of in Harvard’s Department of Stem Cell and Regenerative Biology, uses induced pluripotent stem cells (iPS cells) to make human models of neurological diseases.
This work was supported by the SMA Foundation, National Institute of Neurological Disorders and Stroke grant P01 NS066888, National Institutes of Health grants NS045523 and NS075672, Massachusetts Spinal Cord Injury Research Trust, and the Harvard Stem Cell Institute.
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