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Review

Delivery Approaches for Therapeutic Genome Editing and Challenges

1
Department of Bioengineering, Clemson University, Clemson, SC 29634, USA
2
College of Medicine, Medical University of South Carolina, Charleston, SC 29425, USA
*
Author to whom correspondence should be addressed.
Received: 16 August 2020 / Revised: 16 September 2020 / Accepted: 18 September 2020 / Published: 23 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. View Full-Text
Keywords: gene editing; delivery methods; CRISPR-Cas; ZFNs; TALENs; base editors; viral delivery; nonviral delivery gene editing; delivery methods; CRISPR-Cas; ZFNs; TALENs; base editors; viral delivery; nonviral delivery
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MDPI and ACS Style

Ates, I.; Rathbone, T.; Stuart, C.; Bridges, P.H.; Cottle, R.N. Delivery Approaches for Therapeutic Genome Editing and Challenges. Genes 2020, 11, 1113. https://0-doi-org.brum.beds.ac.uk/10.3390/genes11101113

AMA Style

Ates I, Rathbone T, Stuart C, Bridges PH, Cottle RN. Delivery Approaches for Therapeutic Genome Editing and Challenges. Genes. 2020; 11(10):1113. https://0-doi-org.brum.beds.ac.uk/10.3390/genes11101113

Chicago/Turabian Style

Ates, Ilayda, Tanner Rathbone, Callie Stuart, P. H. Bridges, and Renee N. Cottle 2020. "Delivery Approaches for Therapeutic Genome Editing and Challenges" Genes 11, no. 10: 1113. https://0-doi-org.brum.beds.ac.uk/10.3390/genes11101113

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