Publications by authors named "Francesco Calzatini"
Article Synopsis
- A study involving 57 pediatric patients (ages 0.5-11.4 years) focused on harvesting autologous hematopoietic stem progenitor cells (HSPC) for gene therapy aimed at certain inherited disorders like ADA-SCID, Wiskott-Aldrich syndrome, and metachromatic leukodystrophy.
- The research highlighted that a minimum dose of CD34+ cells needed for effective infusion was 2 × 10/kg, with an optimal target of 5-10 × 10/kg, and the average volume of bone marrow harvested was 34.2 ml/kg.
- Findings indicated that larger volumes (over 30 ml/kg) harvested from children, even infants with these
Successful Treatment With Ledipasvir/Sofosbuvir in an Infant With Severe Combined Immunodeficiency Caused by Adenosine Deaminase Deficiency With HCV Allowed Gene Therapy with Strimvelis.
Francesca Tucci Valeria Calbi Federica Barzaghi Maddalena Migliavacca Francesca Ferrua Francesco Calzatini
Hepatology
December 2018