One-Time Treatment: Base Editing Shows Promise vs. SMA : vim

A research team led by gene editing pioneer David R. Liu, PhD, has published how it has applied the technology to develop a one-time treatment for spinal muscular atrophy (SMA) that showed effectiveness in cell and mouse models.

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San Diego , California , United States , Massachusetts , Harvard University , Netherlands , Keith Joung , Benjaminp Kleinstiver , Mandana Arbab , Alexisc Komor , Nicolem Gaudelli , Davidr Liu , Andrew Anzalone , Christiano Alves , University Of California , Harvard Medical School , Broad Institute , Genentech , Novartis , Biogen Ionis Pharmaceuticals , Harvard Medical School Department Of Neurobiology , Howard Hughes Medical Institute , Genomic Medicine , Massachusetts General Hospital , Beam Therapeutics , Assistant Professor , Harvard Medical , Harvard Medical School Department , Boston Children , Rosamund Stone Zander Translational Neuroscience , Senior Director , Gene Editing Platform Technologies , Ionis Pharmaceuticals ,