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Long-Term Follow-up Study after Lentiviral Hematopoietic Stem/Progenitor Cell Gene Therapy for Wiskott - Aldrich Syndrome

Archive ouverte | Magnani, Alessandra | CCSD

International audience. Wiskott Aldrich syndrome (WAS) is a rare X-linked primary immunodeficiency associated with thrombocytopenia, eczema, infectious, autoimmune complications, and lymphomas. Patients lacking an H...

Clonal tracking in gene therapy patients reveals a diversity of human hematopoietic differentiation programs

Archive ouverte | Six, Emmanuelle | CCSD

International audience. In gene therapy with human hematopoietic stem and progenitor cells (HSPCs), each gene-corrected cell and its progeny are marked in a unique way by the integrating vector. This feature enables...

Gene Therapy for X-Linked Severe Combined Immunodeficiency: Where Do We Stand?

Archive ouverte | Cavazzana, Marina | CCSD

International audience. More than 20 years ago, X-linked severe combined immunodeficiency (SCID-X1) appeared to be the best condition to test the feasibility of hematopoietic stem cell gene therapy. The seminal SCID...

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