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2017 ; 38
(6
): 738-753
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Therapeutic gene editing: delivery and regulatory perspectives
#MMPMID28392568
Shim G
; Kim D
; Park GT
; Jin H
; Suh SK
; Oh YK
Acta Pharmacol Sin
2017[Jun]; 38
(6
): 738-753
PMID28392568
show ga
Gene-editing technology is an emerging therapeutic modality for manipulating the
eukaryotic genome by using target-sequence-specific engineered nucleases. Because
of the exceptional advantages that gene-editing technology offers in facilitating
the accurate correction of sequences in a genome, gene editing-based therapy is
being aggressively developed as a next-generation therapeutic approach to treat a
wide range of diseases. However, strategies for precise engineering and delivery
of gene-editing nucleases, including zinc finger nucleases, transcription
activator-like effector nuclease, and CRISPR/Cas9 (clustered regularly
interspaced short palindromic repeats-associated nuclease Cas9), present major
obstacles to the development of gene-editing therapies, as with other
gene-targeting therapeutics. Currently, viral and non-viral vectors are being
studied for the delivery of these nucleases into cells in the form of DNA, mRNA,
or proteins. Clinical trials are already ongoing, and in vivo studies are
actively investigating the applicability of CRISPR/Cas9 techniques. However, the
concept of correcting the genome poses major concerns from a regulatory
perspective, especially in terms of safety. This review addresses current
research trends and delivery strategies for gene editing-based therapeutics in
non-clinical and clinical settings and considers the associated regulatory
issues.
|*Gene Editing
[MESH]
|*Gene Transfer Techniques
[MESH]
|*Genetic Therapy
[MESH]
|Clustered Regularly Interspaced Short Palindromic Repeats/*genetics
[MESH]