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
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
  • [41] METACHROMATIC LIPODYSTROPHY ( METACHROMATIC LEUKODYSTROPHY )
    LISS, L
    FOLLIS, A
    MCCLURE, R
    REINER, C
    SAYERS, MP
    NEUROLOGY, 1964, 14 (03) : 270 - &
  • [42] Towards a substrate reduction therapy for metachromatic leukodystrophy
    Pujol, Aurora
    Jensen, Elizabeth
    Dwyer, Jacquelyn
    Fezoui, Malika
    Gao, Grace
    Keefe, Kelly
    Brezzani, Alexander
    Olszewski, Maureen
    Lim, Sungtaek
    He, Yan
    Kabiri, Mostafa
    Park, Hyejung
    Wang, Bing
    Dodge, James
    Yew, Nelson S. -S.
    Sardi, Pablo
    MOLECULAR GENETICS AND METABOLISM, 2023, 138 (02) : 108 - 108
  • [43] Lentiviral Haematopoietic Stem Cell Gene Therapy for Late Juvenile Metachromatic Leukodystrophy
    Calbi, Valeria
    Gallo, Vera
    De Mattia, Fabiola
    Zambon, Alberto
    Recupero, Salvatore
    Fratini, Elena Sophia
    Clerici, Alessandra
    Morena, Francesco
    Martino, Sabata
    Brooks, Jean
    Richardson, Alan
    Cambpell, Laura
    Bernardo, Maria Ester
    Aiuti, Alessandro
    Fumagalli, Francesca
    MOLECULAR THERAPY, 2024, 32 (04) : 887 - 887
  • [44] Is It Metachromatic Leukodystrophy?
    Zheng, M. M.
    Zhang, X. H.
    MULTIPLE SCLEROSIS JOURNAL, 2013, 19 (05) : 699 - 699
  • [45] Metachromatic leukodystrophy
    Svitakova, V
    Kodousek, R
    Urbanek, K
    Burval, S
    CESKA A SLOVENSKA NEUROLOGIE A NEUROCHIRURGIE, 1997, 60 (02) : 101 - 105
  • [46] METACHROMATIC LEUKODYSTROPHY - REPORT OF SIBLINGS WITH THE JUVENILE TYPE OF METACHROMATIC LEUKODYSTROPHY
    SATOH, T
    SUZUKI, H
    MONMA, N
    SATODATE, R
    TANAKA, H
    YAJIMA, H
    ACTA PATHOLOGICA JAPONICA, 1988, 38 (08): : 1041 - 1051
  • [47] MUTATIONS OF ARYLSULFASE-A (ASA) GENE IN METACHROMATIC LEUKODYSTROPHY
    ETO, Y
    HASEGAWA, Y
    KAWAME, H
    OHASHI, T
    IDA, H
    JOURNAL OF NEUROCHEMISTRY, 1993, 61 : S101 - S101
  • [48] Intracerebral gene therapy in children with metachromatic leukodystrophy: Results of a phase I/II trial
    Sevin, Caroline
    Roujeau, Thomas
    Cartier, Nathalie
    Baugnon, Thomas
    Adamsbaum, Catherine
    Piraud, Monique
    Martino, Sabata
    Mouiller, Philippe
    Couzinie, Celia
    Bellesme, Celine
    Lalande, Clemence Castaignede
    Cormary, Carine
    Souweidane, Mark M.
    Colle, Marie-Anne
    Adjali, Oumeya
    Sondhi, Dolan
    Crystal, Ronald G.
    Aubourg, Patrick
    Zerah, Michel
    MOLECULAR GENETICS AND METABOLISM, 2018, 123 (02) : S129 - S129
  • [49] Gene therapy for lysosomal storage disorders: recent advances for metachromatic leukodystrophy and mucopolysaccaridosis I
    Penati, Rachele
    Fumagalli, Francesca
    Calbi, Valeria
    Bernardo, Maria Ester
    Aiuti, Alessandro
    JOURNAL OF INHERITED METABOLIC DISEASE, 2017, 40 (04) : 543 - 554