Gene Therapy for GM1 Gangliosidosis Mediated by AAV Vector Carrying BBB-Penetrable Enzyme

被引:0
|
作者
Matsushima, Saki [1 ]
Iizuka, Sayoko [1 ]
Kinoshita, Masafumi [2 ]
Takagi, Haruna [2 ]
Iizuka, Shunsuke [2 ]
Ohtsuki, Kota [2 ]
Imakiire, Atsushi [2 ]
Nagashima, Takashi [3 ]
Okamoto, Shinichiro [4 ,5 ]
Higuchi, Takashi [1 ]
Shimada, Yohta [1 ]
Hioki, Hiroyuki [4 ]
Sonoda, Hiroyuki [2 ]
Watabe, Ayako M. [3 ]
Ohashi, Toya [6 ]
Kobayashi, Hiroshi [1 ]
机构
[1] Jikei Univ, Div Gene Therapy, Res Ctr Med Sci, Sch Med, Tokyo, Japan
[2] JCR Pharmaceut Co Ltd, Kobe, Hyogo, Japan
[3] Jikei Univ, Inst Clin Med & Res, Res Ctr Med Sci, Sch Med, Chiba, Japan
[4] Juntendo Univ, Dept Neuroanat, Grad Sch Med, Tokyo, Japan
[5] Juntendo Univ, Adv Res Inst Hlth Sci, Tokyo, Japan
[6] Jikei Univ, Sch Nursing, Tokyo, Japan
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
1344
引用
收藏
页码:635 / 636
页数:2
相关论文
共 50 条
  • [21] Adeno-Associated Viral (AAV) Vector-Mediated GLB1 Gene Transfer Reduces Substrate Accumulations in a Murine Model of GM1 Gangliosidosis
    Ni, Yan G.
    Gelb, Michael
    Herbst, Zachary
    Vrentas, Catherine
    Messick, Elyse
    Weinstein, David A.
    Browne, Susan E.
    Wilson, James M.
    Hinderer, Christian
    MOLECULAR THERAPY, 2023, 31 (04) : 388 - 388
  • [22] Investigational AAV Gene Therapy LYS-GM101 for GM1-Gangliosidosis
    Hocquemiller, Michael
    Olivier, Sophie
    Parker, Samantha
    Aiach, Karen
    Laufer, Ralph
    MOLECULAR THERAPY, 2020, 28 (04) : 562 - 563
  • [23] IV Gene Therapy Corrects Feline GM1 Gangliosidosis Long Term
    Edwards, Heather
    Randle, Ashley
    Sena-Esteves, Miguel
    Martin, Douglas
    MOLECULAR THERAPY, 2016, 24 : S144 - S145
  • [24] Development of a Hematopoietic Stem Cell Gene Therapy for Infantile GM1 Gangliosidosis
    Bucciarelli, Linda
    Fabris, Camilla
    Scarabello, Elena
    Accardo, Massimo
    Poletti, Valentina
    Biffi, Alessandra
    MOLECULAR THERAPY, 2024, 32 (04) : 733 - 734
  • [25] Mesenchymal stem cell and lentiviral gene therapy for feline GM1 gangliosidosis
    Martin, DR
    Haack, K
    Krum, BK
    Hathcock, TL
    Cox, NR
    Kafri, T
    Niemeyer, GP
    Baker, HJ
    MOLECULAR THERAPY, 2003, 7 (05) : S253 - S253
  • [26] Phase 1/2 trial of AXO-AAV-GM1 (AAV9-GLB1) gene therapy for infantile- and juvenile-onset GM1 gangliosidosis
    Tifft, Cynthia
    D'Souza, Precilla
    Johnston, Jean M.
    Acosta, Maria T.
    Nicoli, Elena-Raluca
    Rothermel, Caroline
    Thurm, Audrey
    Karunakara, Ajith
    Thorp, Benjamin
    Ross, Peter
    Jameson, John
    Sheehan, Michael
    Vaughn, Toby
    Valencia, Donna
    De Boever, Erika
    Corcoran, Gavin
    MOLECULAR GENETICS AND METABOLISM, 2022, 135 (02) : S119 - S119
  • [27] Phase 1/2 Trial of AXO-AAV-GM1 Gene Therapy for the Treatment of Infantile- and Juvenile-onset GM1 Gangliosidosis
    Tifft, C.
    D'Souza, P.
    Johnston, J. M.
    Acosta, M. T.
    Nicoli, E. R.
    Rothermel, C.
    Thurm, A.
    Karunakara, A.
    Thorp, B.
    Ross, P.
    Jameson, J.
    Sheehan, M.
    Vaughn, T.
    Valencia, D.
    De Boever, E.
    Corcoran, G.
    HUMAN GENE THERAPY, 2021, 32 (19-20) : A11 - A12
  • [28] A phase 1/2 trial of AXO-AAV-GM1 gene therapy for the treatment of infantile- and juvenile-onset GM1 gangliosidosis
    Tift, Cynthia
    D'Souza, Precilla
    Johnston, Jean
    Acosta, Maria
    Nicoli, Elena-Raluca
    Rothermel, Caroline
    Thurm, Audrey
    Karunakara, Ajith
    Thorp, Benjamin
    Ross, Peter
    Jameson, John
    Sheehan, Michael
    Vaughn, Toby
    Valencia, Donna
    De Boever, Erika
    GENETICS IN MEDICINE, 2022, 24 (03) : S164 - S164
  • [29] Gene therapy approaches for GM1 gangliosidosis: Focus on animal and cellular studies
    Hosseini, Kamran
    Fallahi, Jafar
    Tabei, Seyed M. B.
    Razban, Vahid
    CELL BIOCHEMISTRY AND FUNCTION, 2023, 41 (08) : 1093 - 1105
  • [30] ENZYME HISTOCHEMICAL AND ULTRASTRUCTURAL FEATURES OF BOVINE GM1 GANGLIOSIDOSIS
    SHEAHAN, BJ
    DONNELLY, WJC
    NEUROPATHOLOGY AND APPLIED NEUROBIOLOGY, 1975, 1 (03) : 317 - 317