A recent review provides insight into the clinical progression of treatment-naive patients with spinal muscular atrophy type 3 who have lost ambulation. Patients with spinal muscular atrophy (SMA) ...
Proteins long known to be essential for hearing have been hiding a talent: they also act as gatekeepers that shuffle fatty molecules across cell membranes. When this newly discovered function goes ...
In a recent Molecular Therapy: Nucleic Acids study, researchers demonstrate enhanced loss-of-function editing of clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated ...
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