Marina Cavazzana

Délégation Paris 5 — France

Biochemistry, Genetics and Molecular Biology · Life Sciences

97h-index41.4kcitations599works13.32yr avg
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Research Topics

Virus-based gene therapy research(164), Immunodeficiency and Autoimmune Disorders(139), Hematopoietic Stem Cell Transplantation(113), CAR-T cell therapy research(112), Hemoglobinopathies and Related Disorders(94)

Publications598 total

Securing raw materials, reagents, and consumable supplies in the academic bioproduction UNITC network: because the chain is only as strong as its weakest link
Bone Marrow Transplantation·2025Open Access
Tracking HIV-1 DNA fate from Cell Culture to Humanized mice Tissues
2025Open Access
Expanding access to haploidentical transplantation : Clinical feasibility of a centralized nationwide distribution model for fresh TCRα/β-depleted grafts in France
Blood·2025Open Access
GMP-manufactured allogeneic human t lymphoid progenitors (HTLP) promote early and sustained T-cell recovery after CD34+-selected haploidentical HSCT in SCID patients : Final results of the htlp-SCID study
Blood·2025Open Access
Long-term follow-up demonstrates durable clinical benefits of exagamglogene autotemcel for sickle cell disease with recurrent vaso-occlusive crises: Final results of climb SCD-121
Blood·2025Open Access
Ex vivo-generated T cell precursors enhance thymic regeneration and T cell immunity after allogeneic hematopoietic cell transplantation
Blood·2025
Lymphedema in patients with X-linked severe combined immunodeficiency
Journal of Allergy and Clinical Immunology Global·2025Open Access
Beyond conventional adoptive T-cell therapy
Journal of Allergy and Clinical Immunology·2025Open Access
Severe inflammation and lineage skewing are associated with poor engraftment of engineered hematopoietic stem cells in patients with sickle cell disease
Nature Communications·2025· 10 citedOpen Access
Maternal and perinatal outcomes of sickle cell disease in pregnancy: A nationwide study in France
British Journal of Haematology·2025· 4 citedOpen Access
Gene Therapy for Inborn Errors of Immunity
Elsevier eBooks·2025
Betibeglogene Autotemcel (Beti-cel) Gene Addition Therapy Results in Durable Hemoglobin A (HbA) Production with up to 10 Years of Follow-up in Participants with Transfusion-Dependent β-Thalassemia
Transplantation and Cellular Therapy·2025· 1 cited
Durable Clinical Benefits in Severe Sickle Cell Disease with Exagamglogene Autotemcel
Transplantation and Cellular Therapy·2025
Human oncostatin M deficiency underlies an inherited severe bone marrow failure syndrome
Journal of Clinical Investigation·2025· 4 citedOpen Access
Haematopoietic gene therapy of non-conditioned patients with Fanconi anaemia-A: results from open-label phase 1/2 (FANCOLEN-1) and long-term clinical trials
The Lancet·2024· 20 cited
Durable Clinical Benefits with Exagamglogene Autotemcel for Severe Sickle Cell Disease
Blood·2024· 2 citedOpen Access
Ex Vivo Generation of Human T-Cell Progenitors from Cord Blood CD34+ Cells Can Accelerate Immune Reconstitution after Umbilical Cord Blood Transplantation in Adult Patient with Hematologic Malignancies
Blood·2024
SMART101 Donor T-Lymphoid Progenitors to Accelerate Immune Reconstitution Post HLA Mismatched Peripheral Blood Stem Cell Transplantation with Post-Transplant Cyclophosphamide: Reset-02 First-in-Human Phase I/II
Blood·2024· 1 citedOpen Access
Betibeglogene Autotemcel (beti-cel) Gene Addition Therapy Results in Durable Hemoglobin a Production with up to 10 Years of Follow-up with Transfusion-Dependent β-Thalassemia
Blood·2024· 2 cited
Gene Therapy for Artemis-SCID and Artemis-Leaky-SCID Patients: Preliminary Results of the French Artegene Phase I/II Clinical Trial
Blood·2024· 1 citedOpen Access
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Frequent Co-authors

Alain Fischer(37), Françoise Le Deist(14), Salima Hacein‐Bey‐Abina(14), Despina Moshous(12), Bénédicte Neven(12), Capucine Pïcard(11), Stéphane Blanche(9), Paul Veys(9), Jean‐Laurent Casanova(8), Stéphane Blanche(8), N. M. Wulffraat(7), Geneviève de Saint Basile(7), Laure Caccavelli(7), Nizar Mahlaoui(7), Jean‐Pierre de Villartay(7), Adrian J. Thrasher(7), Andrew J. Cant(7), Wilhelm Friedrich(7), Mary Slatter(7), Frederic D. Bushman(6)