In Vivo Selection in Non-Human Primates Identifies AAV Capsids Having Enhanced Transgene Expression and Biodistribution in Spinal Cord after Intrathecal Administration

被引：0
|
作者：
Maguire, Casey A. [1 ]
Hanlon, Killian S. [1 ]
Kozarsky, Karen [2 ]
Clark, Sean [3 ]
Gong, Yi [4 ]
Cheng, Ming [1 ]
De la Cruz, Demitri [5 ]
Patel, Nikita [1 ]
Santoscoy, Miguel [1 ]
Nguyen, Diane M. [1 ]
Nammour, Josette [1 ]
机构：
[1] Massachusetts Gen Hosp, Charlestown, MA USA
[2] SwanBio Therapeut Ltd, Bala Cynwyd, PA USA
[3] SwanBio Therapeut Ltd, Princeton, NJ USA
[4] Massachusetts Gen Hosp, Boston, MA USA
[5] Massachusetts Gen Hosp, Neurol, Charlestown, 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：
455
引用
收藏
页码：235 / 235
页数：1
相关论文
共 50 条
  • [41] 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
    MOLECULAR THERAPY, 2022, 30 (04) : 556 - 557
  • [42] Physiologically Based Pharmacokinetic Modeling for the Biodistribution of Adeno-Associated Virus Serotype 8 after Intravenous Administration in Mice and Non-Human Primates
    Sun, Kefeng
    Zhang, Zhiwei
    Ko, Glen
    Bradshaw, Erica L.
    MOLECULAR THERAPY, 2021, 29 (04) : 129 - 130
  • [43] Dorsal root ganglion toxicity after AAV intra-CSF delivery of a RNAi expression construct into non-human primates and mice
    Hawley, Zachary C. E.
    Pardo, Ingrid D.
    Cao, Shaolong
    Zavodszky, Maria I.
    Casey, Fergal
    Ferber, Kyle
    Luo, Yi
    Hana, Sam
    Chen, Shukkwan K.
    Doherty, Jessica
    Costa, Raquel
    Cullen, Patrick
    Liu, Yuqing
    Carlile, Thomas M.
    Chowdhury, Twinkle
    Doyle, Benjamin
    Clarner, Pete
    Mangaudis, Kevin
    Guilmette, Edward
    Bourque, Shawn
    Koske, David
    Nadella, Murali V. P.
    Trapa, Patrick
    Hawes, Michael L.
    Raitcheva, Denitza
    Lo, Shih-Ching
    MOLECULAR THERAPY, 2025, 33 (01) : 215 - 234
  • [44] Global CNS Gene Delivery in Non-Human Primates by Intra-CSF Administration: Evasion of Anti-AAV Neutralizing Antibodies but Not a Transgene-Specific CTL Response
    Gray, Steven J.
    Kalburgi, Sahana Nagabhushan
    McCown, Thomas J.
    Samulski, R. Jude
    MOLECULAR THERAPY, 2013, 21 : S46 - S46
  • [45] 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.
    Venu, Samitha
    Yang, Jason
    Firnberg, Elad
    Yost, Samantha A.
    Liu, Ye
    Danos, Olivier F.
    Mercer, Andrew C.
    MOLECULAR THERAPY, 2023, 31 (04) : 429 - 430
  • [46] 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
    MOLECULAR THERAPY, 2022, 30 (04) : 69 - 69
  • [47] STRV5, a Liver Detargeted AAV Capsid Demonstrates Improved Safety Profile After Intravenous Administration of High Doses in Non-Human Primates
    Gulledge, Travis
    Austin, Bridget
    Bandoski-Gralinski, Cheryl
    Asokan, Aravind
    Rivera, Ruth Castellanos
    MOLECULAR THERAPY, 2022, 30 (04) : 339 - 339
  • [48] Immunological response and durability of expression following sequential intravitreal administration of AAV2.7m8 gene therapy to the contralateral eye in non-human primates
    Cepeda, Diana
    Yeh, Edward
    Nieves, Julio
    Diaz, Carolina Ceballos
    Rosario, Awilda
    Greengard, Judith
    Gelfman, Claire
    Gasmi, Mehdi
    INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2019, 60 (09)
  • [49] Targeted Gene Insertion of Vectorized Monoclonal Antibodies in Non-Human Primates Overcomes AAV Genome Silencing in the Liver and Supports High, Sustained In Vivo Expression of Functional Antibodies
    Sattler, Rachel
    LoMastro, Anthony
    Ezemba, Juanita
    Sterlacci, Tara
    Koehler-Stec, Ellen-Marie
    Parveen, Asma
    Ivanova, Elena
    Pascal, Kristen
    Sabin, Leah
    Kyratsous, Christos
    MOLECULAR THERAPY, 2024, 32 (04) : 106 - 107
  • [50] Sustained AAV9-mediated expression of a non-self protein in the CNS of non-human primates after immunomodulation (vol 13, e0198154, 2018)
    Ramsingh, Arlene I.
    Gray, Steven J.
    Reilly, Andrew
    Koday, Michael
    Bratt, Debbie
    Koday, Merika Treants
    Munson, Paul
    Murnane, Robert
    Smedley, Jeremy
    Hu, Yuhui
    Messer, Anne
    Fuller, Deborah Heydenburg
    PLOS ONE, 2018, 13 (11):
12345