Review of CSF and Peripheral Immune Responses Following Intrathecal Gene Transfer for Giant Axonal Neuropathy

被引:0
|
作者
Saade, Dimah [1 ]
Bharucha-Goebel, Diana [1 ,2 ]
Foley, A. Reghan [1 ]
Neuhaus, Sarah B. [1 ]
Soldatos, Ariane [3 ]
Hu, Ying [1 ]
Hewitt, Matthew [4 ]
Del Hoyo, Roberto Calcedo [5 ]
Jacobson, Steven [6 ]
Leibovitch, Emily [6 ]
Nath, Avindra [7 ]
Bailey, Rachel M. [8 ]
Grieger, Josh [9 ]
Samulski, R. Jude [9 ]
Gray, Steven J. [10 ]
Bonnemann, Carsten G. [1 ]
机构
[1] NINDS, Neuromusuclar & Neurogenet Disorders Childhood Se, NIH, Bldg 36,Rm 4D04, Bethesda, MD 20892 USA
[2] Childrens Natl Hlth Syst, Neurol, Washington, DC USA
[3] NINDS, Pediat Neurol, NIH, Bldg 36,Rm 4D04, Bethesda, MD 20892 USA
[4] Univ Penn, Gene Therapy Program, Immunol Core, Philadelphia, PA 19104 USA
[5] Univ Penn, Immunol Core, Philadelphia, PA 19104 USA
[6] NINDS, Neuroimmunol & Neurovirol, NIH, Bldg 36,Rm 4D04, Bethesda, MD 20892 USA
[7] NINDS, Sect Infect Nervous Syst, NIH, Bldg 36,Rm 4D04, Bethesda, MD 20892 USA
[8] Univ N Carolina, Gene Therapy Ctr, Chapel Hill, NC 27515 USA
[9] Bamboo Therapeut, Chapel Hill, NC USA
[10] Univ Texas Southwestern Med Ctr Dallas, Dept Pediat, Dallas, TX USA
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
999
引用
收藏
页码:456 / 457
页数:2
相关论文
共 50 条
  • [31] Two novel mutations in the GAN gene causing giant axonal neuropathy
    Irad Normendez-Martinez, Monica
    Monterde-Cruz, Lucero
    Martinez, Roberto
    Marquez-Harper, Magdalena
    Esquitin-Garduno, Nayelli
    Valdes-Flores, Margarita
    Casas-Avila, Leonora
    Ponce de Leon-Suarez, Valeria
    Javier Romero-Diaz, Viktor
    Hidalgo-Bravo, Alberto
    WORLD JOURNAL OF PEDIATRICS, 2018, 14 (03) : 298 - 304
  • [32] Giant axonal neuropathy (GAN) caused by mutations in the gigaxonin gene.
    Kuhlenbaumer, G
    Young, P
    Oberwittler, C
    Ringelstein, B
    Stogbauer, F
    AMERICAN JOURNAL OF HUMAN GENETICS, 2001, 69 (04) : 599 - 599
  • [33] Gene Therapy as a Platform: From Giant Axonal Neuropathy to the PaveGT Program
    Bonnemann, Carsten G.
    ANNALS OF NEUROLOGY, 2023, 94 : S273 - S273
  • [34] Two novel mutations in the GAN gene causing giant axonal neuropathy
    Monica Irad Normendez-Martínez
    Lucero Monterde-Cruz
    Roberto Martínez
    Magdalena Marquez-Harper
    Nayelli Esquitin-Garduño
    Margarita Valdes-Flores
    Leonora Casas-Avila
    Valeria Ponce de Leon-Suarez
    Viktor Javier Romero-Díaz
    Alberto Hidalgo-Bravo
    World Journal of Pediatrics, 2018, 14 : 298 - 304
  • [35] Postmortem Assessment of Vector Biodistribution in the First-in-Human Intrathecal scAAV9 Gene Therapy Trial for Giant Axonal Neuropathy
    Gray, Steven J.
    Bailey, Rachel M.
    Bharucha-Goebel, Diana
    Foley, A. Reghan
    Zaric, Violeta
    Crawford, Thomas O.
    Armao, Diane
    Sumner, Charlotte J.
    Hoke, Ahmet
    Chen, Liam
    Grieger, Joshua C.
    Samulski, R. Jude
    Mohassel, Payam
    Hooper, Jody E.
    Bonnemann, Carsten
    MOLECULAR THERAPY, 2017, 25 (05) : 139 - 139
  • [36] CHILDHOOD GIANT AXONAL NEUROPATHY - CASE-REPORT AND REVIEW OF THE LITERATURE
    TANDAN, R
    LITTLE, BW
    EMERY, ES
    GOOD, PS
    PENDLEBURY, WW
    BRADLEY, WG
    JOURNAL OF THE NEUROLOGICAL SCIENCES, 1987, 82 (1-3) : 205 - 228
  • [37] Intrathecal lithium reduces neuropathic pain responses in a rat model of peripheral neuropathy
    Shimizu, T
    Shibata, M
    Inoue, T
    Mashimo, T
    Yoshiya, I
    ANESTHESIOLOGY, 1999, 91 (3A) : U379 - U379
  • [38] Intrathecal lithium reduces neuropathic pain responses in a rat model of peripheral neuropathy
    Shimizu, T
    Shibata, M
    Wakisaka, S
    Inoue, T
    Mashimo, T
    Yoshiya, I
    PAIN, 2000, 85 (1-2) : 59 - 64
  • [39] GIANT AXONAL NEUROPATHY - ELECTROPHYSIOLOGIC EVIDENCE OF CENTRAL AND PERIPHERAL NERVOUS-SYSTEM INVOLVEMENT
    VEILLEUX, M
    LEMIEUX, B
    BERNIER, JP
    JARJOURA, S
    LAMARCHE, JB
    MUSCLE & NERVE, 1988, 11 (09) : 963 - 963
  • [40] Immune responses following intramuscular plasmid gene transfer: A serious limitation for gene therapy?
    Maule, J
    McMahon, J
    Foster, K
    Kingston, R
    Wells, KE
    Wells, DJ
    IMMUNOLOGY, 1996, 89 : B16 - B16