Sentences with phrase «sma gene replacement»
The SMA gene replacement therapy developed by the Mendell and Kaspar teams at Nationwide Children's Hospital and collaborators at Ohio State University has the realistic potential to cure a devastating neurological disorder of newborn infants.
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«Encouraging data for gene replacement therapy for SMA type I, phase 1 study shows.»
Hereditary diseases like SMA maybe a prime therapeutic indications for gene therapy, where gene therapy will provide a functional replacement for a dysfunctional gene.