Publication & Citation Trends
Publications
462 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
A Global Effort to Define the Human Genetics of Protective Immunity to SARS-CoV-2 Infection PDF
Cited by 233
OpenAlex
Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent ß-thalassemia
Cited by 224
OpenAlex
Lentiviral haemopoietic stem/progenitor cell gene therapy for treatment of Wiskott-Aldrich syndrome: interim results of a non-randomised, open-label, phase 1/2 clinical study PDF
Cited by 203
OpenAlex
Gene therapy for ADA‐SCID, the first marketing approval of an <i>ex vivo</i> gene therapy in Europe: paving the road for the next generation of advanced therapy medicinal products PDF
Cited by 251
OpenAlex
In Vivo Tracking of Human Hematopoiesis Reveals Patterns of Clonal Dynamics during Early and Steady-State Reconstitution Phases PDF
Cited by 237
OpenAlex
Research Topics
Virus-based gene therapy research
(165)
CAR-T cell therapy research
(105)
Immunodeficiency and Autoimmune Disorders
(83)
RNA Interference and Gene Delivery
(53)
CRISPR and Genetic Engineering
(52)
Frequent Co-Authors
Affiliations
University of Siena
University of Rome Tor Vergata
European Medicines Agency
Apple (Israel)
Harvard University