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Delivery Approaches for Therapeutic Genome Editing and Challenges. | LitMetric

Delivery Approaches for Therapeutic Genome Editing and Challenges.

Genes (Basel)

Department of Bioengineering, Clemson University, Clemson, SC 29634, USA.

Published: September 2020

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.

Download full-text PDF

Source
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7597956PMC
http://dx.doi.org/10.3390/genes11101113DOI Listing

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