Publication & Citation Trends
Most Cited Works
Publications
19 total
Metachromatic leukodystrophy: A single‐center longitudinal study of 45 patients PDF
Cited by 72
OpenAlex
Mutation Update of ARSA and PSAP Genes Causing Metachromatic Leukodystrophy OA
Cited by 107
Semantic Scholar
Mutation Update of<i>ARSA</i>and<i>PSAP</i>Genes Causing Metachromatic Leukodystrophy
Cited by 139
OpenAlex
Shedding of clinical-grade lentiviral vectors is not detected in a gene therapy setting PDF
Cited by 14
OpenAlex
Metallothionein transcripts are dynamic markers for brain disease in lysosomal disorders OA
Cited by 0
Semantic Scholar
selection clinical trial reflect a benign integration bias and not oncogenic Lentiviral vector common integration sites in preclinical models and a
Cited by 0
Semantic Scholar
Research Topics
Lysosomal Storage Disorders Research
(10)
Virus-based gene therapy research
(5)
Trypanosoma species research and implications
(4)
RNA regulation and disease
(4)
CRISPR and Genetic Engineering
(3)
Frequent Co-Authors
Affiliations
Brigham and Women's Hospital
Harvard University
Vita-Salute San Raffaele University
San Raffaele University of Rome
The San Raffaele Telethon Institute for Gene Therapy