Base and Prime Editing in the Retina—From Preclinical Research toward Human Clinical Trials
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DOI: 10.3390/ijms232012375
Abstract: Inherited retinal diseases (IRDs) are a clinically and genetically heterogeneous group of diseases that are one of the leading causes of vision loss in young and aged individuals. IRDs are mainly caused by a loss… read more here.
Keywords: editing retina; retina preclinical; prime editing; research toward ... See more keywords
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