Development of a novel AAV capsid with improved PNS tropism for treating Pompe disease by intravenous administration

被引：0
|
作者：
Kevany, B. M. [1 ]
Padegimas, L. [1 ]
Miller, T. J. [1 ]
机构：
[1] Abeona Therapeut Inc, Dallas, TX USA
来源：
HUMAN GENE THERAPY | 2019年 / 30卷 / 11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：
P007
引用
收藏
页码：A21 / A21
页数：1
相关论文
共 50 条
  • [21] Bioengineered AAV3B Capsid with Improved Human Hepatocyte Tropism and Evasion of Pre-Existing Neutralizing Antibodies
    Rana, Jyoti
    Marsic, Damien
    Zou, Chenhui
    Munoz-Melero, Maite
    Li, Xin
    Kondratov, Oleksandr
    Li, Ning
    de Jong, Ype P.
    Zolotukhin, Sergei
    Biswas, Moanaro
    MOLECULAR THERAPY, 2023, 31 (04) : 673 - 673
  • [22] Directed Evolution of Rationally-Engineered AAV Capsid Variants with Enhanced Central Nervous System (CNS) Tropism Following Systematic Administration
    Wang, Hongxing
    Zhu, Sha
    Zhang, Weiqun
    Toro, Gabriela C.
    Liu, Na
    Zhao, Lingzhi
    Shen, Joan
    MOLECULAR THERAPY, 2024, 32 (04) : 480 - 480
  • [23] Development of the novel AAV-based vectors with selective tropism to human cancer cells
    Sayroo, Rana
    Yin, Zifei
    Nolasco, Diego
    Pandya, Munjal
    Ling, Chen
    Aslanidi, George
    CANCER RESEARCH, 2015, 75
  • [24] Intravenous administration of mitochondria for treating experimental Parkinson's disease
    Shi, Xianxun
    Zhao, Ming
    Fu, Chen
    Fu, Ailing
    MITOCHONDRION, 2017, 34 : 91 - 100
  • [25] Novel caprine adeno-associated virus (AAV) capsid (AAV-Go.1) is closely related to the primate AAV-5 and has unique tropism and neutralization properties
    Arbetman, AE
    Lochrie, M
    Zhou, SZ
    Wellman, J
    Scallan, C
    Doroudchi, MM
    Randlev, B
    Patarroyo-White, S
    Liu, TY
    Smith, P
    Lehmkuhl, H
    Hobbs, LA
    Pierce, GF
    Colosi, P
    JOURNAL OF VIROLOGY, 2005, 79 (24) : 15238 - 15245
  • [26] Development of an improved novel AAV capsids for intramuscular delivery
    Padegimas, L.
    Kerns, S.
    Kevany, B.
    Miller, T.
    HUMAN GENE THERAPY, 2019, 30 (11) : A26 - A27
  • [27] 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
  • [28] Effects of Sexual Dimorphism and Genetic Background on AAV Tissue Transduction in Mice Following Intravenous Administration of a Diverse Capsid Pool
    Firnberg, Elad
    Egley, Jenny M.
    Qiao, Chunping
    Yost, Samantha A.
    Giles, April R.
    Henry, William M.
    Janczura, Karolina J.
    Glenn, Justin
    McDougald, Devin S.
    Elliot, Kirk
    Qian, Randolph
    Karumuthil-Melethil, Subha
    Liu, Ye
    Danos, Olivier
    Mercer, Andrew C.
    MOLECULAR THERAPY, 2021, 29 (04) : 48 - 48
  • [29] Process and Formulation Development for a Novel Blood-Brain Barrier Penetrant AAV Capsid
    Kim, Taeho
    Xie, Zelong
    Ghazvini, Saba
    Padilla, Jada
    Ward, Alex M.
    Tiffany, Matthew R.
    Ojala, David
    Patel, Nikul
    Chikere, Anthony
    Ramsey, Phillip
    Yadav, Sandeep
    MOLECULAR THERAPY, 2024, 32 (04) : 504 - 504
  • [30] Development of novel AAV serotype 6 based vectors with selective tropism for human cancer cells
    R Sayroo
    D Nolasco
    Z Yin
    Y Colon-Cortes
    M Pandya
    C Ling
    G Aslanidi
    Gene Therapy, 2016, 23 : 18 - 25
12345