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Delivery Approaches for Therapeutic Genome Editing and Challenges.

ABSTRACT: Impressive therapeutic advances have been possible through the advent of zinc-finger nucleases and transcription activator-like effector nucleases. However, discovery of the more efficient and highly tailorable clustered regularly interspaced short palindromic repeats (CRISPR) and associated proteins (Cas9) has provided unprecedented gene-editing capabilities for treatment of various inherited and acquired diseases. Despite recent clinical trials, a major barrier for therapeutic gene editing is the absence of safe and effective methods for local and systemic delivery of gene-editing reagents. In this review, we elaborate on the challenges and provide practical considerations for improving gene editing. Specifically, we highlight issues associated with delivery of gene-editing tools into clinically relevant cells.

SUBMITTER: Ates I

PROVIDER: S-EPMC7597956 | biostudies-literature | 2020 Sep

REPOSITORIES: biostudies-literature

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Delivery Approaches for Therapeutic Genome Editing and Challenges.

Ates Ilayda I Rathbone Tanner T Stuart Callie C Bridges P Hudson PH Cottle Renee N RN

Genes 20200923 10

Impressive therapeutic advances have been possible through the advent of zinc-finger nucleases and transcription activator-like effector nucleases. However, discovery of the more efficient and highly tailorable clustered regularly interspaced short palindromic repeats (CRISPR) and associated proteins (Cas9) has provided unprecedented gene-editing capabilities for treatment of various inherited and acquired diseases. Despite recent clinical trials, a major barrier for therapeutic gene editing is ...[more]

PMID: 32977396

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