Improved Gene Therapy for Metachromatic Leukodystrophy

被引：0
|
作者：
Tricoli, Lucas [1 ]
Vanderver, Adeline [1 ]
Adang, Laura [1 ]
Chappell, Maxwell [1 ]
Breda, Laura [1 ]
Tanaka, Naoto [1 ]
Wenger, David [2 ]
Guerra, Amaliris [1 ]
Triebwasser, Michael [1 ]
Kurre, Peter [1 ]
Schlotawa, Lars [3 ]
Radhakrishnan, Karthikeyan [4 ]
Ahrens-Nicklas, Rebecca [1 ]
Hurwitz, Stephanie [5 ]
Rivella, Stefano [1 ]
机构：
[1] Childrens Hosp Philadelphia, Philadelphia, PA 19104 USA
[2] Thomas Jefferson Univ, Philadelphia, PA 19107 USA
[3] Univ Med Ctr Gottingen, Gottingen, Germany
[4] Bielefeld Univ, Bielefeld, Germany
[5] Hosp Univ Penn, Philadelphia, PA USA
来源：
MOLECULAR THERAPY | 2022年 / 30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：
876
引用
收藏
页码：415 / 416
页数：2
相关论文
共 50 条
  • [11] Compassionate use of lentiviral gene therapy for metachromatic leukodystrophy
    Orchard, Paul J.
    Gupta, Ashish
    Adang, Laura A.
    Braun, Jennifer
    Pillai, Nishitha R.
    Lund, Troy
    Eisengart, Julie B.
    MOLECULAR GENETICS AND METABOLISM, 2024, 141 (02)
  • [12] Gene therapy for metachromatic leukodystrophy: Lead candidate optimization
    Gingras, Jacinthe
    St Martin, Thia
    Gall, Katie
    Newman, Jennifer
    Selby, Donald E.
    Adjei, Samuel
    Hanscom, Terrence
    Prout, Jaime
    Przybylski, Dariusz S.
    Zheng, Alice
    Avila, Nancy
    Lotterhand, Jason
    Rivas, Israel
    Wright, Teresa
    Francone, Omar
    Seymour, Albert
    MOLECULAR GENETICS AND METABOLISM, 2023, 138 (02) : 47 - 47
  • [13] Gene therapy in advanced metachromatic leukodystrophy: tempering expectations
    Daphne HSchoenmakers
    Shanice Beerepoot
    Laura AAdang
    Marije ABCAsbreuk
    Caroline GBergner
    Annette EBley
    JaapJan Boelens
    Valeria Calbi
    Alejandra Darling
    Erik Eklund
    ngeles Garca Cazorla
    Sabine WGrnborg
    Samuel Groeschel
    Peter Mvan Hasselt
    Carla EMHollak
    Claire Horgan
    Simon Jones
    Tom de Koning
    Lucia Laugwitz
    Caroline Lindemans
    Pascal Martin
    Fanny Mochel
    Andreas berg
    Dipak Ram
    Caroline Sevin
    Ludger Schls
    Ayelet Zerem
    Nicole IWolf
    Francesca Fumagalli
    Protein & Cell, 2025, 16 (01) : 12 - 15
  • [14] Novel candidate disease for gene therapy: metachromatic leukodystrophy
    Biffi, Alessandra
    Naldini, Luigi
    EXPERT OPINION ON BIOLOGICAL THERAPY, 2007, 7 (08) : 1193 - 1205
  • [15] Gene therapy in advanced metachromatic leukodystrophy: tempering expectations
    Schoenmakers, Daphne H.
    Beerepoot, Shanice
    Adang, Laura A.
    Asbreuk, Marije A. B. C.
    Bergner, Caroline G.
    Bley, Annette E.
    Boelens, Jaap-Jan
    Calbi, Valeria
    Darling, Alejandra
    Eklund, Erik
    Garcia Cazorla, Angeles
    Gronborg, Sabine W.
    Groeschel, Samuel
    van Hasselt, Peter M.
    Hollak, Carla E. M.
    Horgan, Claire
    Jones, Simon
    de Koning, Tom
    Laugwitz, Lucia
    Lindemans, Caroline
    Martin, Pascal
    Mochel, Fanny
    Oberg, Andreas
    Ram, Dipak
    Sevin, Caroline
    Schoels, Ludger
    Zerem, Ayelet
    Wolf, Nicole, I
    Fumagalli, Francesca
    PROTEIN & CELL, 2024, 16 (01) : 12 - 15
  • [16] In vivo gene therapy of metachromatic leukodystrophy by lentiviral vectors
    Consiglio, A
    Follenzi, A
    Dolcetta, D
    Marchesini, S
    Orlacchio, A
    Bordignon, C
    Naldini, L
    FASEB JOURNAL, 2003, 17 (04): : A685 - A685
  • [17] Brain gene therapy clinical trial for Metachromatic Leukodystrophy
    Sevin, C.
    Piguet, F.
    Sondhi, D.
    Colle, M. A.
    Roujeau, T.
    Raoul, S.
    Deschamps, J. Y.
    Bouquet, C.
    Ahouansou, O.
    Vanier, M. T.
    Moullier, P.
    Cherel, Y.
    Hackett, N. R.
    Zerah, M.
    Crystal, R. G.
    Aubourg, P.
    Cartier, N.
    HUMAN GENE THERAPY, 2011, 22 (10) : A43 - A44
  • [18] Safety of arylsulfatase a overexpression for gene therapy of metachromatic leukodystrophy
    Capotondo, A.
    Cesani, M.
    Pepe, S.
    Fasano, S.
    Gregori, S.
    Tononi, L.
    Venneri, M. A.
    Brambilla, R.
    Quattrini, A.
    Ballabio, A.
    Cosma, M. P.
    Naldini, L.
    Biffi, A.
    HUMAN GENE THERAPY, 2007, 18 (09) : 821 - 836
  • [19] Lentiviral vectors for in vivo gene therapy of metachromatic leukodystrophy
    Consiglio, A
    Dolcetta, D
    Follenzi, A
    Luca, T
    Marchesini, S
    Gritti, A
    Vescovi, A
    Naldini, L
    Bordignon, C
    JOURNAL OF NEUROCHEMISTRY, 2003, 87 : 5 - 5
  • [20] Brain gene therapy for metachromatic leukodystrophy: towards clinical trial
    Piguet, F.
    Sondhi, D.
    Colle, M. A.
    Raoul, S.
    Roujeau, T.
    Vanier, M. T.
    Bouquet, C.
    Moullier, P.
    Cherel, Y.
    Hackett, N.
    Zerah, M.
    Aubourg, P.
    Crystal, R. G.
    Cartier, N.
    Sevin, C.
    HUMAN GENE THERAPY, 2010, 21 (10) : 1449 - 1450
12345