The delivery challenge: fulfilling the promise of therapeutic genome editing
Abstract
Genome editing has the potential to treat an extensive range of incurable monogenic and complex diseases. In particular, advances in sequence-specific nuclease technologies have dramatically accelerated the development of therapeutic genome editing strategies that are based on either the knockout of disease-causing genes or the repair of endogenous mutated genes. These technologies are progressing into human clinical trials. However, challenges...
Paper Details
Title
The delivery challenge: fulfilling the promise of therapeutic genome editing
Published Date
Jun 29, 2020
Journal
Volume
38
Issue
7
Pages
845 - 855
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