Directed Evolution of an AAV9 Library Identifies a Capsid Variant with Enhanced Brain Tropism and Liver De-Targeting in Non-Human Primates and Mice Following Systemic Administration

被引：0

作者：

Moyer, Tyler ^{[1
]}

Graham, Kristin ^{[1
]}

Liu, Jiachen ^{[1
]}

Ren, Xiaoqin ^{[1
]}

Clement, Joseph ^{[1
]}

Bloedel, Alexis ^{[1
]}

Kulkarni, Anupriya ^{[1
]}

Looby, Kailyn ^{[1
]}

Khalid, Hamza ^{[1
]}

Amy, Bruce ^{[1
]}

Egbuchulam, Chiso ^{[1
]}

Laks, Dan ^{[1
]}

Child, Matthew ^{[1
]}

Kaur, Rupinderjit ^{[1
]}

Sayed-Zahid, Ambreen ^{[1
]}

Grannan, Michael ^{[1
]}

Chung, Charlotte ^{[1
]}

Knox, Tatiana ^{[1
]}

Hoffman, Brett ^{[1
]}

Kutchin, Alex ^{[1
]}

Johnson, Amy ^{[1
]}

Cameron, Andrew ^{[1
]}

Grant, Kyle ^{[1
]}

Yao, Johnny ^{[1
]}

Pande, Nilesh ^{[1
]}

Thompson, Jeff ^{[1
]}

Heffner, Garrett ^{[1
]}

Carter, Todd A. ^{[1
]}

Nonnenmacher, Mathieu E. ^{[1
]}

机构：

[1] Voyager Therapeut, Cambridge, MA USA

来源：

MOLECULAR THERAPY | 2023年 / 31卷 / 04期

关键词：

D O I：

暂无

中图分类号：

Q81 [生物工程学（生物技术）]; Q93 [微生物学];

学科分类号：

071005 ; 0836 ; 090102 ; 100705 ;

摘要：

105

引用

页码：57 / 57

页数：1

共 11 条

[1] Directed Evolution of AAV9 Peptide Display Libraries Identifies a Family of Cross-Species Variants with Enhanced Brain Tropism in Non-Human Primates and Mice Following Systemic Administration
Moyer, Tyler
Li, Jiangyu
Laks, Dan R.
Child, Matthew A.
Tyson, Katherine
Kaur, Rupinderjit
Ren, Xiao-Qin
Sayed-Zahid, Ambreen
Grannon, Michael
Chung, Charlotte Hiu-Yan
Pande, Nilesh
Thompson, Jeff
Wang, Wei
Hasson, Samuel
Hefferan, Michael
Nonnenmacher, Mathieu
[J]. MOLECULAR THERAPY, 2022, 30 (04) : 556 - 557
[2] Directed Evolution of an AAV5 Capsid Library Identifies a Variant with Enhanced Transduction in Non-Human Primate and Rodent Brain Following Systemic Administration
Nonnenmacher, Mathieu E.
Ren, Amy Z.
Wang, Wei
Child, Matthew A.
Ren, Xiao-Qin
Tyson, Katherine
Laguna-Torres, Jessenia
Kulkarni, Anupriya
Pande, Nilesh
Sayed-Zahid, Ambreen
Chung, Charlotte H. Y.
Hefferan, Michael
Moyer, Tyler
Maura, Damien
[J]. MOLECULAR THERAPY, 2022, 30 (04) : 69 - 69
[3] A Novel AAV9 Variant Exhibiting Strong Preference for Neuronal Transduction in Mice and Non-Human Primates Following Systemic Administration
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Baggett, Helen R.
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[J]. MOLECULAR THERAPY, 2020, 28 (04) : 13 - 14
[4] AAV-DJ is superior to AAV9 for targeting brain and spinal cord, and de-targeting liver across multiple delivery routes in mice
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[J]. JOURNAL OF TRANSLATIONAL MEDICINE, 2024, 22 (01)
[5] Improving Neuronal Gene Transfer to the Brain After CSF Administration of AAV9 in Juvenile Non-Human Primates
Wright, Maggie
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[J]. MOLECULAR THERAPY, 2022, 30 (04) : 301 - 301
[6] Systemic gene expression following intravenous administration of AAV2/9 to fetal and neonatal mice and non-human primates
Rahim, A. A.
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[J]. HUMAN GENE THERAPY, 2011, 22 (10) : A106 - A107
[7] High Resolution Biodistribution Analysis Following Suprachoroidal Administration of a Pool of AAV3B, AAV8, and AAV9 Vectors to Non-Human Primates Reveals Spatial and Cell-Based Tropism Differences
Giles, April R.
Lilley, Brendan M.
Tejada, Sigmund K.
Trang Nguyen
Aprea, Christieann
Cho, Sungyeon
Clarke, Ryan N.
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[J]. MOLECULAR THERAPY, 2023, 31 (04) : 429 - 430
[8] Directed Evolution of AAV Targeting Lung Epithelia Using Aerosol Delivery Identifies 4D-A101, a Variant Demonstrating Robust Gene Delivery in Non-Human Primates
Kotterman, Melissa
Calton, Melissa
Nye, Julie
Beliakoff, Ghezal
Quezada, Melissa
Croze, Roxanne
Whittlesey, Kevin
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Francis, Peter
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[J]. MOLECULAR THERAPY, 2020, 28 (04) : 570 - 571
[9] Identification of a cell surface receptor utilized by an engineered BBB-penetrant capsid family with enhanced brain tropism in non-human primates and mice
Hoffman, B. A.
Knox, T.
Moyer, T.
Shah, I.
Kaur, R.
Child, M. A.
Laks, D. R.
Nonnenmacher, M.
[J]. HUMAN GENE THERAPY, 2022, 33 (23-24) : A32 - A32
[10] Cell Type-Specific TRAnscription-Dependent Directed Evolution (TRADE) Identifies Novel AAV Capsids Capable of Enhanced Neuronal Transduction in Mice and Non-Human Primates
Huang, Samuel J.
Adachi, Kei
Bagget, Helen R.
Song, Zhen
Dissen, Gregory A.
Ojeda, Sergio R.
Nakai, Hiroyuki
[J]. MOLECULAR THERAPY, 2019, 27 (04) : 24 - 25

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