Long-Term Effects of In Vivo Genome Editing in the Mouse Retina Using Campylobacter jejuni Cas9 Expressed via Adeno-Associated Virus

Dong Hyun Jo; Taeyoung Koo; Chang Sik Cho; Jin Hyoung Kim; Jin Soo Kim; Jeong Hun Kim

doi:10.1016/j.ymthe.2018.10.009

Long-Term Effects of In Vivo Genome Editing in the Mouse Retina Using Campylobacter jejuni Cas9 Expressed via Adeno-Associated Virus

Dong Hyun Jo, Taeyoung Koo, Chang Sik Cho, Jin Hyoung Kim, Jin Soo Kim, Jeong Hun Kim

Research output: Contribution to journal › Article › peer-review

47 Citations (Scopus)

Abstract

Jo et al. demonstrated that intravitreally administered adeno-associated virus encoding the Cas9 ortholog from Campylobacter jejuni targeted to Hif1a did not induce histologic and functional aberration in the retina. Despite the accumulation of on-target insertions and deletions (indels), there were no indels at the potential off-target sites.

Original language	English
Pages (from-to)	130-136
Number of pages	7
Journal	Molecular Therapy
Volume	27
Issue number	1
DOIs	https://doi.org/10.1016/j.ymthe.2018.10.009
Publication status	Published - 2 Jan 2019

Bibliographical note

Publisher Copyright:
© 2018 The American Society of Gene and Cell Therapy

Keywords

Campylobacter jejuni
genome editing
long-term effect
retina

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10.1016/j.ymthe.2018.10.009

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title = "Long-Term Effects of In Vivo Genome Editing in the Mouse Retina Using Campylobacter jejuni Cas9 Expressed via Adeno-Associated Virus",

abstract = "Jo et al. demonstrated that intravitreally administered adeno-associated virus encoding the Cas9 ortholog from Campylobacter jejuni targeted to Hif1a did not induce histologic and functional aberration in the retina. Despite the accumulation of on-target insertions and deletions (indels), there were no indels at the potential off-target sites.",

keywords = "Campylobacter jejuni, genome editing, long-term effect, retina",

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AU - Kim, Jin Hyoung

AU - Kim, Jin Soo

AU - Kim, Jeong Hun

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AB - Jo et al. demonstrated that intravitreally administered adeno-associated virus encoding the Cas9 ortholog from Campylobacter jejuni targeted to Hif1a did not induce histologic and functional aberration in the retina. Despite the accumulation of on-target insertions and deletions (indels), there were no indels at the potential off-target sites.

KW - Campylobacter jejuni

KW - genome editing

KW - long-term effect

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Long-Term Effects of In Vivo Genome Editing in the Mouse Retina Using Campylobacter jejuni Cas9 Expressed via Adeno-Associated Virus

Abstract

Bibliographical note

Keywords

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