Publication & Citation Trends
Most Cited Works
Publications
272 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 PDF
Cited by 269
OpenAlex
Post-Transcriptional Genetic Silencing of <i>BCL11A</i> to Treat Sickle Cell Disease PDF
Cited by 419
OpenAlex
A comprehensive single cell transcriptional landscape of human hematopoietic progenitors PDF
Cited by 370
OpenAlex
Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1/2 trial
Cited by 500
OpenAlex
Preclinical Safety and Efficacy of Human CD34+ Cells Transduced With Lentiviral Vector for the Treatment of Wiskott-Aldrich Syndrome (vol 21, pg 175, 2013)
Cited by 1
Semantic Scholar
Combined cell and gene therapy to treat merosin deficient congenital muscular dystrophy
Cited by 0
Semantic Scholar
Research Topics
Lysosomal Storage Disorders Research
(49)
Virus-based gene therapy research
(44)
Acute Lymphoblastic Leukemia research
(34)
Childhood Cancer Survivors' Quality of Life
(29)
Hematopoietic Stem Cell Transplantation
(24)
Frequent Co-Authors
Affiliations
Broad Institute
Boston Children's Hospital
Hudson Institute
Harvard University
University of Padua