1 Department of Clinical Sciences and Community Health, Università degli Studi di Milano, Milan, Italy.
2 Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Milan, Italy.
3 Department of Pediatrics, Division of Hematology, Children's Hospital of Philadelphia, Philadelphia, PA, United States.
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hemoglobin disorders, including beta-thalassemia (BT) and sickle-cell
disease (SCD), are the most common monogenic diseases worldwide, with a
global carrier frequency of over 5%. With
migration, they are becoming more common worldwide, making their
management and care an increasing concern for health care systems.
Bone Marrow Transplantation (BMT)
Clinical Trials Based on Gene Therapy and Gene Editing
Conditioning in Clinical Trials and New Preclinical Development
CD34+ Hematopoietic Progenitor Cells Mobilization
From Clinical Trials to Drug Commercialization, the Challenges of Pivotal GT Studies
Lentiviral Vectors Manufacturing
HCS Transduction and Handling
Summary and Conclusions