Therapeutic In Vivo Gene Editing Achieved by a Hypercompact CRISPR‐Cas12f1 System Delivered with All‐in‐One Adeno‐Associated Virus (Adv. Sci. 19/2024)

Therapeutic In Vivo Gene Editing Achieved by a Hypercompact CRISPR‐Cas12f1 System Delivered with All‐in‐One Adeno‐Associated Virus (Adv. Sci. 19/2024)

CRISPR Gene Therapy In article number 2308095, Wei Li, Fei Teng, and colleagues introduce a hypercompact CRISPR‐Cas12f1 system characterized by enhanced gene editing capabilities and minimal off‐target effects. Applied to the treatment of inherited retinal diseases, this system shows significant the...

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Veröffentlicht in:Advanced science 2024-05, Vol.11 (19), p.n/a
Hauptverfasser: Cui, Tongtong, Cai, Bingyu, Tian, Yao, Liu, Xin, Liang, Chen, Gao, Qingqin, Li, Bojin, Ding, Yali, Li, Rongqi, Zhou, Qi, Li, Wei, Teng, Fei
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Sprache:eng
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Zusammenfassung:CRISPR Gene Therapy In article number 2308095, Wei Li, Fei Teng, and colleagues introduce a hypercompact CRISPR‐Cas12f1 system characterized by enhanced gene editing capabilities and minimal off‐target effects. Applied to the treatment of inherited retinal diseases, this system shows significant therapeutic outcomes, highlighting its substantial promise for gene therapy applications.
ISSN:2198-3844
2198-3844
DOI:10.1002/advs.202470108