Challenges and successes of retinal gene therapy with AAV vectors

被引：0
|
作者：
Auricchio, A. [1 ,2 ]
机构：
[1] Telethon Inst Genet & Med TIGEM, Naples, Italy
[2] Univ Naples Federico 2, Dept Pediat, Naples, Italy
来源：
HUMAN GENE THERAPY | 2011年 / 22卷 / 10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：
引用
收藏
页码：A11 / A11
页数：1
相关论文
共 50 条
  • [41] Adeno-associated virus (AAV) vectors in gene therapy
    Serra, C
    Zentilin, L
    Tafuro, S
    Falaschi, A
    Giacca, M
    MINERVA BIOTECNOLOGICA, 1997, 9 (03) : 155 - 165
  • [42] Gene therapy for hemophilia B by novel serotypes of AAV vectors
    Wang, LL
    Gao, GP
    Bell, P
    Sanmiguel, J
    Li, Y
    Nichols, TC
    Verma, IM
    Wilson, JM
    MOLECULAR THERAPY, 2003, 7 (05) : S26 - S26
  • [43] Application of AAV vectors for in vivo gene therapy of Gaucher disease
    Marshall, J
    McEachern, KA
    Kyros, JAC
    Nietupski, JB
    Lukason, MJ
    Barbon, C
    Taylor, K
    Ford, C
    Barranger, JA
    Schuchman, EH
    Desnick, RJ
    Lonning, S
    Cheng, SH
    MOLECULAR THERAPY, 2003, 7 (05) : S396 - S397
  • [44] Synthetic AAV Vectors for the Potential Gene Therapy of Hemophilia in Children
    Shoti, Jakob
    Qing, Keyun
    Srivastava, Arun
    MOLECULAR THERAPY, 2021, 29 (04) : 178 - 178
  • [45] AAV vectors for gene correction
    Cathomen, T
    CURRENT OPINION IN MOLECULAR THERAPEUTICS, 2004, 6 (04) : 360 - 366
  • [46] Improved dual AAV hybrid vectors for efficient and safe retinal gene transfer
    Trapani, I.
    Toriello, E.
    de Simone, S.
    Colella, P.
    Iodice, C.
    Somella, A.
    Rossi, S.
    Simonelli, F.
    Giunti, M.
    Bacci, M. L.
    Auricchio, A.
    HUMAN GENE THERAPY, 2015, 26 (10) : A100 - A100
  • [47] Comparative study of liver gene therapy with AAV vectors based on endogenous and engineered AAV capsids
    Wang, L.
    Bell, P.
    Somanathan, S.
    Wang, Q.
    He, Z.
    Yu, H.
    McMenamin, D.
    Goode, T.
    Calcedo, R.
    Wilson, J. M.
    HUMAN GENE THERAPY, 2015, 26 (10) : A101 - A101
  • [48] Retinal gene therapy for Stargardt disease with dual AAV intein vectors is both safe and effective in large animal models
    Ferla, Rita
    Pugni, Eugenio
    Lupo, Mariangela
    Tiberi, Paola
    Fioretto, Federica
    Perota, Andrea
    Duchi, Roberto
    Lagutina, Irina
    Gesualdo, Carlo
    Rossi, Settimio
    Ventrella, Domenico
    Elmi, Alberto
    Mcclinton, Benjamin
    Toomes, Carmel
    Xu, Tongzhou
    Molday, Robert S.
    Surace, Enrico M.
    Simonelli, Francesca
    Bacci, Maria L.
    Galli, Cesare
    Memon, Muhammad A.
    Shams, Naveed
    Auricchio, Alberto
    Trapani, Ivana
    SCIENCE ADVANCES, 2025, 11 (13):
  • [49] Development of Gene Therapy Vectors: Remaining Challenges
    Gupta, Vibhor
    Lourenco, Silvia P.
    Hidalgo, Ismael J.
    JOURNAL OF PHARMACEUTICAL SCIENCES, 2021, 110 (05) : 1915 - 1920
  • [50] Novel targeting non-AAV parvoviral vectors for gene therapy
    Itah, R
    Etingov, I
    Davis, C
    Tal, J
    MOLECULAR THERAPY, 2004, 9 : S303 - S304
12345