Translational potential of base-editing tools for gene therapy of monogenic diseases
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DOI: 10.3389/fbioe.2022.942440
Abstract: Millions of people worldwide have rare genetic diseases that are caused by various mutations in DNA sequence. Classic treatments of rare genetic diseases are often ineffective, and therefore great hopes are placed on gene-editing methods.… read more here.
Keywords: potential base; monogenic diseases; base editing; translational potential ... See more keywords