Identification of Novel AAV Capsid Variants with Enhanced Tropism for the Canine Outer Retina Following Intravitreal Delivery

被引:0
作者
Byrne, Leah C. [1 ]
Visel, Meike [2 ]
Dufour, Valerie [3 ]
Marinho, Felipe Pompeo [3 ]
Aguirre, Gustavo D. [3 ]
Beltran, William A. [3 ]
Flannery, John G. [2 ]
Schaffer, David V. [2 ]
机构
[1] Univ Calif Berkeley, Sch Vet Med, Dept Clin Studies, Helen Wills Neurosci Inst,Univ Penn, Berkeley, CA 94720 USA
[2] Univ Calif Berkeley, Helen Wills Neurosci Inst, Berkeley, CA 94720 USA
[3] Univ Penn, Sch Vet Med, Dept Clin Studies, Philadelphia, PA 19104 USA
关键词
D O I
10.1016/S1525-0016(16)33425-6
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
617
引用
收藏
页码:S244 / S245
页数:2
相关论文
共 48 条
[21]   Intravenous delivery of a novel AAV capsid with improved PNS tropism reduces underlying Pompe disease pathology [J].
Kevany, Brian ;
Padegimas, Linas ;
Miller, Timothy .
MOLECULAR GENETICS AND METABOLISM, 2020, 129 (02) :S87-S87
[22]   Novel AAV variants isolated by directed evolution in primate display enhanced retinal transduction following intravitreal injection [J].
Choudhury, Shreyasi ;
Marsic, Damien ;
Peterson, James ;
Fajardo, Diego ;
Bennett, Antonette ;
Gamlin, Paul D. ;
Agbandje-McKenna, Mavis ;
Zolotukhin, Sergei ;
Boye, Sanford L. ;
Boye, Shannon Elizabeth .
INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2017, 58 (08)
[23]   Novel AAV Variants Isolated by Directed Evolution in Primate Display Enhanced Retinal Transduction Following Intravitreal Injection [J].
Boye, Sanford L. ;
Choudhury, Shreyasi ;
Marsic, Damien ;
Strang, Christianne ;
Peterson, James J. ;
Fajardo, Diego ;
Bennett, Antonette ;
Agbandje-McKenna, Mavis ;
Witherspoon, C. Douglas ;
Gamlin, Paul D. ;
Zolotukhin, Sergei ;
Boye, Shannon E. .
MOLECULAR THERAPY, 2017, 25 (05) :231-232
[24]   A novel dual affinity selection (DAS) capsid screening platform for engineering AAV capsids with enhanced photoreceptor transduction via intravitreal delivery [J].
Wang, Jianghui ;
Cui, Mengtian ;
Punzo, Claudio ;
Gao, Guangping .
MOLECULAR THERAPY, 2025, 33 (04)
[25]   Identification of AAV Capsid with Improved Tropism to Macular Retina Through Multi-Species Screening in Mice, Rabbits, Pigs, and Monkeys [J].
Liu, Shengjiang ;
Chen, Haifeng ;
Gong, Xiaoming ;
Peraramelli, Sameera ;
Luo, Ningguang ;
Chen, Alan ;
Li, Jinzhong ;
Santillan, Eduardo ;
Huynh, Derrick ;
Li, Shuyi ;
Li, Ying ;
Lin, Kevin ;
Dai, Minghua ;
Chen, Jianwu ;
Yu, Zhe ;
Song, June ;
Nguyen, Son Th Ai ;
Zhang, John ;
Ou, Li .
MOLECULAR THERAPY, 2024, 32 (04) :166-166
[26]   Machine-Guided Design Reveals AAV Variants Efficient in Transducing NHP Retina After Intravitreal Delivery [J].
Turunen, Heikki T. ;
Cramer, Megan ;
Gerold, Jeff ;
Kuchwara, Helene ;
Kwasnieski, Jamie C. ;
Lapan, Sylvain ;
Lin, Kathy ;
Locane, Elina ;
Marrogi, Eryney ;
McDonel, Patrick ;
Levitin, Hanna Mendes ;
Nagabhushana, Nishith ;
Otwinowski, Jakub ;
Reardon, Christopher ;
Sengel, Cem ;
Sinai, Sam ;
Wolock, Sam ;
Kelsic, Eric .
MOLECULAR THERAPY, 2022, 30 (04) :555-555
[27]   Analysis of Gene Expression, Tissue Tropism, and Safety of Novel AAV Variants in Mice Following Intravenous Administration [J].
Neal, Tawny ;
Tobia, Steven ;
Bauer, Alex ;
Ni, Ming ;
Gasmi, Mehdi ;
Keravala, Annahita .
MOLECULAR THERAPY, 2017, 25 (05) :52-52
[28]   Assessment of next-generation AAV variants in gerbil and non-human primate retina following intravitreal injection [J].
Keravala, Annahita ;
Tseng, Yu-Shan ;
Neal, Tawny ;
Ni, Ming ;
Chavez, Christopher ;
Gasmi, Mehdi .
INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2017, 58 (08)
[29]   Intravitreal Delivery of an AAV2-Based and Cell-Penetrating Peptide Grafted Novel Capsid Produces Enhanced Panretinal and Photoreceptor Transduction with Reduced Inflammation [J].
Wang, Jianghui ;
Cui, Mengtian ;
Liu, Hao ;
Wang, Dan ;
Tai, Phillip W. L. ;
Gao, Guangping .
MOLECULAR THERAPY, 2024, 32 (04) :683-684
[30]   Discovery of Novel AAV Capsids with Enhanced Skeletal Muscle Tropism Following Directed Evolution in Cynomolgus Macaque [J].
Jin, Quan ;
Hung, Ka Ying Sharon ;
Kashyap, Samneet ;
Rouse, Emily ;
Kelemen, Margaret ;
Xiang, Lucia ;
Utturkar, Aditya ;
Chittoda, Misha ;
Sampath, Harini ;
Bourque, Shawn ;
Desmarais, Samantha ;
Deng, Shuyun ;
Chen, Wei Tong ;
Gianni, Davide ;
Suh, Junghae ;
Yuan, Yuan .
MOLECULAR THERAPY, 2023, 31 (04) :556-556