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My professional goal as a pediatrician specializing in immunology, has been to challenge the limits of "inexplicable" and "untreatable" diseases, and apply current scientific knowledge to understand the mechanisms of impaired cellular immune function underlying the clinical manifestations in order to develop curative treatments.Following the completion of my pediatrics residency, I received training in molecular and cellular immunology in France (UNICET, Lyon) and the United States (DNAX Research Institute of Molecular and Cellular Biology, Palo Alto) under scientists, who critically influenced my scientific development. There, I was first exposed to the importance of integrating in depth laboratory research with clinical observations to develop a translational research approach to science. I then worked for fifteen years at the San Raffaele Scientific Institute in the Telethon Institute for Gene Therapy (HSR-TIGET), where I focused on dissecting the genetic and immunological basis of primary immunodeficiencies with autoimmune manifestations that might be treated by gene therapy.
In the coming years, I plan to further determine the genetic and immunological basis of diseases with autoimmunity or immune dysregulation in children. I believe that much can still be learned from the in depth mechanistic studies of pediatric autoimmune diseases. Genomic analysis of the patients' samples has become possible which may provide a rapid indication of altered target molecules. I plan to implement robust functional studies to define the consequences of these genetic abnormalities and bridge them to the patient's clinical phenotype.Understanding functional consequences of gene mutations in single case/family first and then validating the molecular and cellular defects in other patients with similar phenotypes, will anticipate and complement cellular and gene therapy strategies.For further information please visit the Bacchetta Lab website:http://med.stanford.edu/bacchettalab.html
CD4^LVFOXP3 in Participants With IPEX
This first-in-human, Phase 1 clinical trial will test the feasibility of the manufacturing
and the safety of the administration of CD4^LVFOXP3 in up to 36 evaluable human participants
with IPEX and evaluate the impact of the CD4^LVFOXP3 infusion on the disease.
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Stem Cell Transplant From Donors After Alpha Beta Cell Depletion in Children and Adults With T-allo10 Cells Addback
The purpose of this study is to determine the safety of a cell therapy, T-allo10, after
αβdepleted-HSCT in the hopes that it will boost the adaptive immune reconstitution of the
patient while sparing the risk of developing severe Graft-versus-Host Disease (GvHD).
The primary objective of Phase 1 is to determine the recommended Phase 2 dose (RP2D)
administered after infusion of αβdepleted-HSCT in children and young adults with hematologic
A Phase 1b extension will occur after dose escalation, enrolling at the RP2D for the T-allo10
cells determined in the Phase 1 portion to evaluate the safety and efficacy of infusion of
T-allo10 after receipt of αβdepleted-HSCT. Additionally, Phase 1b aims to explore
improvements in immune reconstitution.
All participants on this study must be enrolled on another study: NCT04249830