Delivery Approaches for Therapeutic Genome Editing and Challenges

Genes

P.Hudson Bridges¹  Tanner Rathbone²  Callie Stuart²  ReneeN. Cottle²  Ilayda Ates² 

[1] College of Medicine, Medical University of South Carolina, Charleston, SC 29425, USA;Department of Bioengineering, Clemson University, Clemson, SC 29634, USA;

关键词: gene editing;    delivery methods;    CRISPR-Cas;    ZFNs;    TALENs;    base editors;   

DOI  :  10.3390/genes11101113

来源: DOAJ

【 摘 要 】

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.

【 授权许可】

Unknown