Hematopoietic stem cell transplantation for metachromatic leukodystrophy

Expert Opinion on Orphan Drugs

Volumn 3, Issue 8, 2015, Pages 911-919

  Lorioli, Laura ^a,b,c,d   Biffi, Alessandra ^a,b,c,d  

^a SAN RAFFAELE SCIENTIFIC INSTITUTE   (Italy)

^b ASST Santi Carlo e Paolo   (Italy)

^c SAN RAFFAELE HOSPITAL   (Italy)

^d VITA SALUTE SAN RAFFAELE UNIVERSITY   (Italy)

Author keywords

Gene therapy; hematopoietic stem cells; metachromatic leukodystrophy; transplantation

Indexed keywords

BUSULFAN; CEREBROSIDE SULFATASE; COMPLEMENTARY DNA; LENTIVIRUS VECTOR;

COMPARATIVE EFFECTIVENESS; FOLLOW UP; GENE THERAPY; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; METACHROMATIC LEUKODYSTROPHY; PRIORITY JOURNAL; PROGNOSIS; REVIEW; RISK BENEFIT ANALYSIS; TREATMENT INDICATION;

EID: 84937390306     PISSN: None     EISSN: 21678707     Source Type: Journal    
DOI: 10.1517/21678707.2015.1062754     Document Type: Review

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