Counteracting the Common Shwachman–Diamond Syndrome-Causing SBDS c.258+2T>C Mutation by RNA Therapeutics and Base/Prime Editing
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DOI: 10.3390/ijms24044024
Abstract: Shwachman–Diamond syndrome (SDS) represents one of the most common inherited bone marrow failure syndromes and is mainly caused by SBDS gene mutations. Only supportive treatments are available, with hematopoietic cell transplantation required when marrow failure… read more here.
Keywords: diamond syndrome; sbds 258; mutation; shwachman diamond ... See more keywords