The Capsid Race: Multiplexed Analysis and Comparison of Rationally Engineered AAV CNS Capsids In Vivo

被引：0

作者：

Pimentel, Elena ^{[1
]}

Kieper, Sebastian ^{[2
]}

Moreno, Fiona ^{[2
]}

Van der Zon, Tom ^{[1
]}

Bosma, Bas ^{[1
]}

Bashir, Noun ^{[1
]}

Waaijer, Stijn ^{[1
]}

Espinosa, Pedro ^{[1
]}

Hoogland, Tycho ^{[1
]}

Valls, Mercedes ^{[1
]}

Pesic, Milja ^{[1
]}

Kusuma, Angga ^{[3
]}

机构：

[1] UniQure Biopharma BV, Amsterdam, Netherlands

[2] UniQure BV, Amsterdam, Netherlands

[3] UniQure b, Amsterdam, Netherlands

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：

448

引用

页码：231 / 232

页数：2

共 39 条

[21] Superior In vivo Transduction of Human Hepatocytes Using Engineered AAV3 Capsid
Vercauteren, Koen
Hoffman, Brad E.
Zolotukhin, Irene
Keeler, Geoffrey D.
Xiao, Jing W.
Basner-Tschakarjan, Etiena
High, Katherine A.
Ertl, Hildegund C. J.
Rice, Charles M.
Srivastava, Arun
de Jong, Ype P.
Herzog, Roland W.
MOLECULAR THERAPY, 2016, 24 (06) : 1042 - 1049
[22] Intravenous administration of engineered AAV gene therapy capsid demonstrates improved CNS transduction in adult mice
Murlidharan, G.
Adachi, K.
Wang, H.
Nguyen, T.
Johnson, B.
Liu, L.
Zhou, J.
Felix-Ortiz, A.
Christensen, E.
Aubin, J.
Goulet, M.
Thompson, J.
Carter, T.
Hou, J.
Sah, D.
HUMAN GENE THERAPY, 2018, 29 (12) : A79 - A79
[23] AAV Capsid-Promoter Interactions Determine CNS Cell Selective Gene Expression In Vivo
Powell, Sara Kathleen
Samulski, R. Jude
McCown, Thomas J.
MOLECULAR THERAPY, 2020, 28 (04) : 225 - 225
[24] Characterization of engineered AAV capsids in the retina of mice and in two novel human retina ex vivo models
Schoen, C.
Duechs, M.
Blazevic, D.
Schonberger, T.
Strobel, B.
Ciossek, T.
du Plessis, F.
Sun, M.
Oti, M.
Alanis-Lobato, G.
Loskill, P.
Kuehn, M. H.
Kreuz, S.
Maier, U.
Michelfelder, S.
HUMAN GENE THERAPY, 2022, 33 (23-24) : A27 - A28
[25] Evolution of Blood-Brain Barrier Penetrant AAV Capsids in Non-Human Primates Using a Multiplexed Transcription Dependent Capsid Engineering Platform
Tiffany, Matthew R.
Frias, Stephanye
Andrews, Lori
Nanjaraj, Ankitha
Bendana, Yuri
Darst, Russell
Wist, Stephen
Tran, Hung
Kennard, Kenneth
Mueller, Sarah
Ojala, David
Pooler, Amy
MOLECULAR THERAPY, 2023, 31 (04) : 573 - 573
[26] Discovering Rationally-Engineered AAV5-derived Capsid Variants for Superior CNS Tropism and Peripheral Tissue De-targeting in Non-Human Primate via Systemic Administration
Zhu, Sha
Wang, Hongxing
Cabrera, Gabriela Toro
Zhang, Weiqun
Liu, Na
Zhao, Lingzhi
Shen, Joan
MOLECULAR THERAPY, 2024, 32 (05) : 11 - 11
[27] An AAV Capsid Programmed to Bind Human and Macaque Receptors Mediates Enhanced Gene Delivery to the CNS and Skeletal Muscle In Vivo
Wu, Jason
Botticello-Romero, Nuria R.
Chan, Ken Y.
Lin, Chin-Yen
Huang, Qin
Beddow, Thomas
Brauer, Pamela
Chan, Yujia A.
Deverman, Benjamin E.
MOLECULAR THERAPY, 2024, 32 (04) : 116 - 117
[28] A Novel Engineered AAV2 Capsid Variant CereAAV™ for Efficient In Vivo Gene Transduction into Mouse and Marmoset Brain
Tanaka, Y.
Nariki, H.
Ueda, S.
Tanaka, R.
Nishie, T.
Takashima, F.
Okamoto, S.
Enoki, T.
Mineno, J.
HUMAN GENE THERAPY, 2022, 33 (23-24) : A31 - A31
[29] AAV-Mediated CNS-Wide Gene Delivery via a De Novo Engineered Capsid-Human Receptor Interaction
Chan, Ken Y.
Huang, Qin
Johnston, Jencilin
Barry, Andrew J.
Zheng, Qingxia
Chen, Albert T.
Beddow, Thomas
Keyes, Casey
Pacouret, Simon
Brauer, Pamela P.
Lou, Shan
Clouse, Gabrielle
Wu, Jason
Navarrete, Nuria R. R.
Eid, Fatma Elzahraa
Chan, Yujia A.
Deverman, Benjamin E.
MOLECULAR THERAPY, 2023, 31 (04) : 56 - 56
[30] SMRT Sequencing Allows High-Throughput Analysis of a Whole Capsid Shuffled AAV Capsid Library Following CNS Selection in Mice and NHPs
Casy, Widler
Chen, Xin
Dong, Thomas
Hu, Yuhui
Kanchwala, Mohammed
Xing, Chao
Braun, Stephen
Bunnell, Bruce
Gray, Steven
MOLECULAR THERAPY, 2020, 28 (04) : 79 - 79

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