Efficient in Utero Gene Transfer to the Mammalian Inner Ears by the Synthetic Adeno-Associated Viral Vector Anc80L65

Chin Ju Hu; Ying Chang Lu; Yi Hsiu Tsai; Haw Yuan Cheng; Hiroki Takeda; Chun Ying Huang; Ru Xiao; Chuan Jen Hsu; Jin Wu Tsai; Luk H. Vandenberghe; Chen Chi Wu; Yen Fu Cheng

doi:10.1016/j.omtm.2020.06.019

Efficient in Utero Gene Transfer to the Mammalian Inner Ears by the Synthetic Adeno-Associated Viral Vector Anc80L65

Chin Ju Hu^*, Ying Chang Lu, Yi Hsiu Tsai, Haw Yuan Cheng, Hiroki Takeda, Chun Ying Huang, Ru Xiao, Chuan Jen Hsu, Jin Wu Tsai, Luk H. Vandenberghe, Chen Chi Wu, Yen Fu Cheng

^*Corresponding author for this work

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

17 Scopus citations

Abstract

This study demonstrates that Anc80L65, a synthetic AAV vector validated to exhibit high expression in the inner ears of neonatal and adult mice, can target both cochlear and vestibular hair cells and spiral ganglion neurons when delivered in utero. These findings support the potential use of Anc80L65 in prenatal gene therapy of hereditary hearing loss.

Original language	English
Pages (from-to)	493-500
Number of pages	8
Journal	Molecular Therapy - Methods and Clinical Development
Volume	18
DOIs	https://doi.org/10.1016/j.omtm.2020.06.019
State	Published - 11 Sep 2020

Keywords

AAV2/Anc80L65
adeno-associated virus
gene therapy
hereditary deafness
in utero microinjection
inner ears

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10.1016/j.omtm.2020.06.019

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Hu, C. J., Lu, Y. C., Tsai, Y. H., Cheng, H. Y., Takeda, H., Huang, C. Y., Xiao, R., Hsu, C. J., Tsai, J. W., Vandenberghe, L. H., Wu, C. C., & Cheng, Y. F. (2020). Efficient in Utero Gene Transfer to the Mammalian Inner Ears by the Synthetic Adeno-Associated Viral Vector Anc80L65. Molecular Therapy - Methods and Clinical Development, 18, 493-500. https://doi.org/10.1016/j.omtm.2020.06.019

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title = "Efficient in Utero Gene Transfer to the Mammalian Inner Ears by the Synthetic Adeno-Associated Viral Vector Anc80L65",

abstract = "This study demonstrates that Anc80L65, a synthetic AAV vector validated to exhibit high expression in the inner ears of neonatal and adult mice, can target both cochlear and vestibular hair cells and spiral ganglion neurons when delivered in utero. These findings support the potential use of Anc80L65 in prenatal gene therapy of hereditary hearing loss.",

keywords = "AAV2/Anc80L65, adeno-associated virus, gene therapy, hereditary deafness, in utero microinjection, inner ears",

author = "Hu, {Chin Ju} and Lu, {Ying Chang} and Tsai, {Yi Hsiu} and Cheng, {Haw Yuan} and Hiroki Takeda and Huang, {Chun Ying} and Ru Xiao and Hsu, {Chuan Jen} and Tsai, {Jin Wu} and Vandenberghe, {Luk H.} and Wu, {Chen Chi} and Cheng, {Yen Fu}",

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year = "2020",

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doi = "10.1016/j.omtm.2020.06.019",

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AU - Hu, Chin Ju

AU - Lu, Ying Chang

AU - Tsai, Yi Hsiu

AU - Cheng, Haw Yuan

AU - Takeda, Hiroki

AU - Huang, Chun Ying

AU - Xiao, Ru

AU - Hsu, Chuan Jen

AU - Tsai, Jin Wu

AU - Vandenberghe, Luk H.

AU - Wu, Chen Chi

AU - Cheng, Yen Fu

PY - 2020/9/11

Y1 - 2020/9/11

N2 - This study demonstrates that Anc80L65, a synthetic AAV vector validated to exhibit high expression in the inner ears of neonatal and adult mice, can target both cochlear and vestibular hair cells and spiral ganglion neurons when delivered in utero. These findings support the potential use of Anc80L65 in prenatal gene therapy of hereditary hearing loss.

AB - This study demonstrates that Anc80L65, a synthetic AAV vector validated to exhibit high expression in the inner ears of neonatal and adult mice, can target both cochlear and vestibular hair cells and spiral ganglion neurons when delivered in utero. These findings support the potential use of Anc80L65 in prenatal gene therapy of hereditary hearing loss.

KW - AAV2/Anc80L65

KW - adeno-associated virus

KW - gene therapy

KW - hereditary deafness

KW - in utero microinjection

KW - inner ears

UR - http://www.scopus.com/inward/record.url?scp=85088651043&partnerID=8YFLogxK

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DO - 10.1016/j.omtm.2020.06.019

M3 - Article

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SN - 2329-0501

VL - 18

SP - 493

EP - 500

JO - Molecular Therapy - Methods and Clinical Development

JF - Molecular Therapy - Methods and Clinical Development

ER -

Efficient in Utero Gene Transfer to the Mammalian Inner Ears by the Synthetic Adeno-Associated Viral Vector Anc80L65

Abstract

Keywords

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