MPSV

临床评论者：医学博士Shelby Elenburg和MD STN的Leah Crisafi：125755/0

ABCD1 Adenosine triphosphate binding cassette, subfamily D, member 1 AC Advisory Committee ALDP Adrenoleukodystrophy Protein Allo-HSCT Allogeneic hematopoietic stem cell tr​​ansplant ALT Alanine aminotransferase ANC Absolute neutrophil count AMN Adrenomyeloneuropathy AST Aspartate aminotransferase BLA Biologics License Application BM Bone marrow BMCs Bone marrow cells c/dg Copies per diploid genome CALD Cerebral adrenoleukodystrophy CBC Complete blood count CD15+ Cluster of differentiation 15 positive CD34+ Cluster of differentiation 34 positive cDNA Complementary deoxyribonucleic acid Chr Chromosome CI Confidence interval DAT Direct antiglobulin test DNA Deoxyribonucleic acid DP Drug product Eli-cel Elivaldogene autotemcel FDA Food and Drug Administration FISH Fluorescence in situ hybridization G-CSF Granulocyte colony stimulating factor GdE Gadolinium enhancement GLP Good laboratory practice GT Gene therapy GVHD Graft versus host disease Hgb Hemoglobin HLA Human leukocyte antigen HSC Hematopoietic stem cell HSCT Hematopoietic stem cell tr​​ansplant ISA Integration site analysis ISS Integrated Summary of Safety ITT Intent-to-treat KM Kaplan-Meier LTFU Long-term follow-up LTR Long terminal repeat LVCF Last visit carried forward LVV Lentiviral vector MD Matched donor MDS Myelodysplastic syndrome MFD Major functional disability MPSV Myeloproliferative sarcoma virus MRD Matched相关供体（兄弟姐妹除外）MRI磁共振成像mRNA Messenger核糖核酸