Publication & Citation Trends
Publications
0 total
Lentiviral haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1/2 trial and expanded access OA
Cited by 183
Semantic Scholar
Retrieval of vector integration sites from cell-free DNA
Cited by 31
Semantic Scholar
Toward Reference Intervals of ARSA Activity in the Cerebrospinal Fluid: Implication for the Clinical Practice of Metachromatic Leukodystrophy.
Cited by 8
Semantic Scholar
Lentiviral hematopoietic stem and progenitor cell gene therapy (HSPC-GT) for metachromatic leukodystrophy (MLD): Clinical outcomes from 33 patients
Cited by 10
Semantic Scholar
Extensive Metabolic Correction of Hurler Disease By Hematopoietic Stem Cell-Based Gene Therapy: Preliminary Results from a Phase I/II Trial OA
Cited by 4
Semantic Scholar
Correction to: c-MYC overexpression induces choroid plexus papillomas through a T-cell mediated inflammatory mechanism OA
Cited by 0
Semantic Scholar
PS1220 EX-VIVO HEMATOPOIETIC STEM CELL GENE THERAPY (GT) FOR MUCOPOLYSACCHARIDOSIS TYPE I HURLER (MPSIH): PRELIMINARY RESULTS FROM A PHASE I/II CLINICAL STUDY OA
Cited by 0
Semantic Scholar
Lentiviral haematopoietic stem cell gene therapy (HSC-GT) for metachromatic leukodystrophy (MLD): Preliminary results from a clinical trial with a cryopreserved formulation of OTL-200
Cited by 1
Semantic Scholar
Research Topics
Glioma Diagnosis and Treatment
(5)
Lysosomal Storage Disorders Research
(5)
Virus-based gene therapy research
(4)
Neuroinflammation and Neurodegeneration Mechanisms
(4)
Neutrophil, Myeloperoxidase and Oxidative Mechanisms
(3)
Affiliations
Vita-Salute San Raffaele University
Queen Mary University of London
The San Raffaele Telethon Institute for Gene Therapy
Istituti di Ricovero e Cura a Carattere Scientifico
Istituto di Ricovero e Cura a Carattere Scientifico San Raffaele