Mps Ii

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CLINICAL_TRIALS trial_result · 2022-09-27
Clinical Trial Update: Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II [Phase 1, Phase 2]
An ascending-dose study of the ZFN genome-editing therapy SB-913 in MPS II (Mucopolysaccharidosis II) was terminated; results have been post…