Allogeneic Hematopoietic Stem Cell Transplantation for Adult Metachromatic Leukodystrophy (MLD): A Case Series

被引：0
|
作者：
Riedel, Andreas [1 ]
Faul, Christoph [1 ]
Reuss, Kristina [1 ]
Lang, Peter [2 ]
Lengerke, Claudia [1 ]
Weissert, Nadine [3 ]
Hengel, Holger [3 ]
Schoels, Ludger [3 ]
Bethge, Wolfgang Andreas [1 ]
机构：
[1] Univ Hosp Tuebingen, Dept Internal Med 2, Hematol Oncol Clin Immunol & Rheumatol, Stuttgart, Germany
[2] Univ Childrens Hosp Tubingen, Dpt Gen Pediat Hematol & Oncol 1, Tubingen, Germany
[3] Univ Hosp Tuebingen, Deutsch Zentrum Neurodegenerat Erkrankungen DZNE, Tubingen, Germany
来源：
BLOOD | 2023年 / 142卷
关键词：
D O I：
10.1182/blood-2023-187317
中图分类号：
R5 [内科学];
学科分类号：
1002 ; 100201 ;
摘要：
引用
收藏
页数：3
相关论文
共 50 条
  • [31] Long-term outcomes after allogeneic hematopoietic stem cell transplantation for metachromatic leukodystrophy: the largest single-institution cohort report
    Boucher, Alexander A.
    Miller, Weston
    Shanley, Ryan
    Ziegler, Richard
    Lund, Troy
    Raymond, Gerald
    Orchard, Paul J.
    ORPHANET JOURNAL OF RARE DISEASES, 2015, 10
  • [32] Lentiviral (LV) Hematopoietic Stem Cell Gene Therapy (HSC-GT) for Metachromatic Leukodystrophy (MLD) Provides Sustained Clinical Benefit
    Fumagalli, F.
    Calbi, V
    Sessa, M.
    Zambon, A.
    Baldoli, C.
    Cugnata, F.
    Rancoita, P.
    Acquati, S.
    Redaelli, D.
    Miglietta, S.
    Magni, S.
    Recupero, S.
    Ferrua, F.
    Barzaghi, F.
    Cicalese, M.
    Migliavacca, M.
    Tucci, F.
    Gallo, V
    Ciotti, F.
    Fraschini, M.
    Sarzana, M.
    Silvani, P.
    Spiga, I
    Ferrari, M.
    Facchini, M.
    Locatelli, S.
    Antonioli, G.
    Zancan, S.
    Calabria, A.
    Montini, E.
    Farinelli, G.
    Morena, F.
    Segovia, J.
    Schwab, L.
    Downey, G.
    Gabaldo, M.
    Martino, S.
    Di Serio, C.
    Ciceri, F.
    Comi, G.
    Sora, Natali M.
    Bernardo, M.
    Naldini, L.
    Biffi, A.
    Aiuti, A.
    ANNALS OF NEUROLOGY, 2019, 86 : S163 - S164
  • [33] A case of adult onset metachromatic leukodystrophy
    Ito, Kae
    Miura, Nobuyoshi
    Awata, Shuichi
    Nanba, Eiji
    Matsuoka, Hiroo
    PSYCHIATRY AND CLINICAL NEUROSCIENCES, 2009, 63 (01) : 127 - 127
  • [34] Correction of metachromatic leukodystrophy in the mouse model by transplantation of genetically modified hematopoietic stem cells
    Biffi, A
    De Palma, M
    Quattrini, A
    Del Carro, U
    Amadio, S
    Visigalli, I
    Sessa, M
    Fasano, S
    Brambilla, R
    Marchesini, S
    Bordignon, C
    Naldini, L
    JOURNAL OF CLINICAL INVESTIGATION, 2004, 113 (08): : 1118 - 1129
  • [35] A Mutation-Agnostic Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy
    Antony, Justin S.
    Daniel-Moreno, Alberto
    Lamsfus-Calle, Andres
    Raju, Janani
    Kaftancioglu, Merve
    Urena-Bailen, Guillermo
    Rottenberger, Jennifer
    Hou, Yujuan
    Santhanakumaran, Vidiyaah
    Lee, Jun-Hoe
    Heumos, Lukas
    Boehringer, Judith
    Kraegeloh-Mann, Ingeborg
    Handgretinger, Rupert
    Mezger, Markus
    CRISPR JOURNAL, 2022, 5 (01): : 66 - 79
  • [36] LENTIVIRAL HAEMATOPOIETIC STEM CELL GENE THERAPY (ATIDARSAGENE AUTOTEMCEL) FOR LATE JUVENILE METACHROMATIC LEUKODYSTROPHY (MLD)
    Gallo, Vera
    Calbi, Valeria
    De Mattia, Fabiola
    Zambon, Alberto
    Recupero, Salvatore
    Fratini, Elena
    Ciotti, Francesca
    Fraschini, Maddalena
    Sangalli, Mara
    Sarzana, Marina
    Clerici, Alessandra
    Morena, Francesco
    Martino, Sabata
    Brooks, Jean
    Richardson, Alan
    Campbell, Laura
    Bernardo, Maria Ester
    Aiuti, Alessandro
    Fumagalli, Francesca
    BONE MARROW TRANSPLANTATION, 2024, 59 : 95 - 96
  • [37] Lentiviral hematopoietic stem and progenitor cell gene therapy (HSPC-GT) for metachromatic leukodystrophy (MLD): Clinical outcomes from 33 patients
    Fumagalli, Francesca
    Calbi, Valeria
    Sessa, Maria
    Zambon, Alberto
    Baldoli, Cristina
    Rancoita, Paula M. V.
    Acquati, Serena
    de Mattia, Fabiola
    Tucci, Francesca
    Gallo, Vera
    Zancan, Stefano
    Montini, Eugenio
    Schwab, Laetitia
    Downey, Gerald
    Sharpe, John
    Gabaldo, Michela
    Martino, Sabata
    De Serio, Clelia
    Ciceri, Fabio
    Filippi, Massimo
    Sora, Maria Grazia Natali
    Bernardo, Maria Ester
    Naldini, Luigi
    Biffi, Alessandra
    Aiuti, Alessandro
    MOLECULAR GENETICS AND METABOLISM, 2020, 129 (02) : S59 - S59
  • [38] Lentiviral Hematopoietic Stem and Progenitor Cell Gene Therapy (HSPC-GT) for Metachromatic Leukodystrophy (MLD): Clinical Outcomes from 33 Patients
    Calbi, Valeria
    Fumagalli, Francesca
    Sessa, Maria
    Zambon, Alberto
    Baldoli, Cristina
    Cugnata, Federica
    Rancoita, Paola M. V.
    Acquati, Serena
    Redaelli, Daniela
    De Mattia, Fabiola
    Fratini, Elena
    Ferrua, Francesca
    Barzaghi, Federica
    Cicalese, Maria Pia
    Migliavacca, Maddalena
    Tucci, Francesca
    Gallo, Vera
    Ciotti, Francesca
    Fraschini, Maddalena
    Sarzana, Marina
    Silvani, Paolo
    Spiga, Ivana
    Facchini, Marcella
    Locatelli, Sara
    Antonioli, Gigliola
    Zancan, Stefano
    Calabria, Andrea
    Montini, Eugenio
    Farinelli, Giada
    Morena, Francesco
    Segovia, Jesus
    Schwab, Laetitia C.
    Downey, Gerald
    Sharpe, John
    Gabaldo, Michela
    Martino, Sabata
    Di Serio, Clelia
    Ciceri, Fabio
    Filippi, Massimo
    Sora, Maria Grazia Natali
    Bernardo, Maria Ester
    Naldini, Luigi
    Biffi, Alessandra
    Aiuti, Alessandro
    BONE MARROW TRANSPLANTATION, 2020, 55 (SUPPL 1) : 72 - 73
  • [39] Dermoscopic Evolution of Mycosis Fungoides After Allogeneic Hematopoietic Stem Cell Transplantation: a Case Series
    Kaminska-Winciorek, Grazyna
    Szlauer-Stefanska, Anastazja
    Mendrek, Wlodzimierz
    Giebel, Sebastian
    DERMATOLOGY PRACTICAL & CONCEPTUAL, 2023, 13 (01):
  • [40] Allogeneic hematopoietic stem cell transplantation
    Kai, S
    Hara, T
    THERAPEUTIC APHERESIS AND DIALYSIS, 2003, 7 (03) : 285 - 291
12345