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Marina Cavazzana is a pediatrician, Professor of Hematology since 2000, Director of the Department of Biotherapy at Necker Hospital, Paris Descartes University. She is the Director of the Inserm / Assistance Publique - Hôpitaux de Paris GHU Ouest Biotherapy Clinical Investigation Center and is a member of the research Human Lymphohematopoiesis laboratory at Imagine Institute.
Her main research and clinical interests are the development of the hematopoietic immune system, and cell and gene therapy for inherited and acquired disease of the hematopoietic system. Her group studies the means to improve the clinical results of hematopoietic stem cell transplantation, crossing HLA-barriers, and the differentiation of mouse and human stem cells towards lymphocyte lineages. She has initiated several clinical trials based on the use of ex vivo gene modified hematopoietic stem cells to treat patients with inherited disorders, the preliminary clinical results of which are encouraging. She is the author or co-author of several patents and of more than 250 publications in peer-reviewed journals and was awarded 2 ERC (2011 and 2016) and one state funding from the French National Research Agency (ANR) Investments for the Future programme (PIA) (Recherche Hospitalo-Universitaire en santé) (2019).
Her work was rewarded by the American Society of Hematology (Award on Clinical Research in Gene Therapy in 1999), by the French Academy of Sciences (Special Medical Award in 2000 and Jean-Pierre Lecocq Award on Gene Therapy in 2004). She was awarded the title of Officier de l'Ordre National de la Légion d'honneur in 2011, and given the Irène Joliot Curie 2012 award "Scientific Women of the Year" (Science Academy and French Ministry of Education and Research). She was also awarded with the French National Academy of Medicine in 2016 and the 2017 Ernest Beutler Lecture and Prize for Clinical Science (American Society of Hematology). She has also been elected as an International member of the National Academy of Medicine in 2019.
Resources & publications
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Journal (source)Stem Cells Dev
Gene Therapy with Hematopoietic Stem Cells: The Diseased Bone Marrow's Point ...
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Journal (source)Mol Ther Methods Clin Dev
Successful Preclinical Development of Gene Therapy for Recombinase-Activating...
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Journal (source)Mol Ther Methods Clin Dev
Biosafety Studies of a Clinically Applicable Lentiviral Vector for the Gene T...
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Journal (source)Front Immunol
Disease Evolution and Response to Rapamycin in Activated Phosphoinositide 3-K...
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Journal (source)Blood Adv
Baboon envelope LVs efficiently transduced human adult, fetal, and progenitor...
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Journal (source)Blood
A combination of cyclophosphamide and interleukin-2 allows CD4+ T cells conve...
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Journal (source)Blood
Reticular dysgenesis: international survey on clinical presentation, transpla...
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Journal (source)J Allergy Clin Immunol
An in vivo genetic reversion highlights the crucial role of Myb-Like, SWIRM, ...
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Journal (source)J Allergy Clin Immunol
The BLNK adaptor protein has a nonredundant role in human B-cell differentiat...
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Journal (source)J Allergy Clin Immunol
Successful RAG1-SCID gene therapy depends on the level of RAG1 expression.
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Journal (source)J Allergy Clin Immunol
Recombination-activating gene 1 (Rag1)-deficient mice with severe combined im...
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Journal (source)Bone Marrow Transplant
Ex vivo generated human T-lymphoid progenitors as a tool to accelerate immune...
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Journal (source)Front Immunol
Disease Evolution and Response to Rapamycin in Activated Phosphoinositide 3-K...
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Journal (source)JAMA
Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndr...
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Journal (source)Am J Transplant
Donor-targeted serotherapy as a rescue therapy for steroid-resistant acute GV...
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Journal (source)Nat Immunol
Single-cell analysis of FOXP3 deficiencies in humans and mice unmasks intrins...
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Journal (source)Cell Mol Immunol
A DL-4- and TNFα-based culture system to generate high numbers of nonmodified...
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Journal (source)Nat Commun
Transient mTOR inhibition rescues 4-1BB CAR-Tregs from tonic signal-induced d...
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Journal (source)Nat Med
Long-term outcomes of lentiviral gene therapy for the β-hemoglobinopathies: t...
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Journal (source)Nat Commun
Base-editing-mediated dissection of a γ-globin cis-regulatory element for the...
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Journal (source)Mol Ther Nucleic Acids
Novel lentiviral vectors for gene therapy of sickle cell disease combining ge...
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Journal (source)Annu Rev Genomics Hum Genet
Sickle Cell Disease: From Genetics to Curative Approaches.
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Journal (source)Blood
Adenine base editor-mediated correction of the common and severe IVS1-110 (G>...
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Journal (source)Mol Ther
Combination of lentiviral and genome editing technologies for the treatment o...
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Journal (source)Haematologica
Mutations in the adaptor-binding domain and associated linker region of p110δ...
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Journal (source)Haematologica
Extensive multilineage analysis in patients with mixed chimerism after alloge...
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Journal (source)Blood
Lentiviral and genome-editing strategies for the treatment of β-hemoglobinopa...
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Journal (source)Hum. Mol. Genet.
Gene therapy of hemoglobinopathies: progress and future challenges.
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Journal (source)Nat Rev Drug Discov
Gene therapy targeting haematopoietic stem cells for inherited diseases: prog...
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Journal (source)Mol Ther Methods Clin Dev
A Nontoxic Transduction Enhancer Enables Highly Efficient Lentiviral Transduc...
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Journal (source)Haematologica
Plerixafor enables safe, rapid, efficient mobilization of hematopoietic stem ...
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Journal (source)J. Allergy Clin. Immunol.
Generation of adult human T-cell progenitors for immunotherapeutic applications.
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Journal (source)Hum. Gene Ther.
Gene Therapy for X-Linked Severe Combined Immunodeficiency: Where Do We Stand?
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Journal (source)Stem Cells Dev.
Gene Therapy with Hematopoietic Stem Cells: The Diseased Bone Marrow's Point ...
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Journal (source)Blood
Gene-corrected human Munc13-4-deficient CD8+ T cells can efficiently restrict...
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Journal (source)J. Clin. Invest.
Loss of ARHGEF1 causes a human primary antibody deficiency.
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Journal (source)Blood Adv
Gene transfer into hematopoietic stem cells reduces HLH manifestations in a m...
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Journal (source)J. Allergy Clin. Immunol. 2017
Clinical and immunologic phenotype associated with activated phosphoinositide...
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Journal (source)J. Clin. Invest.
A human immunodeficiency caused by mutations in the PIK3R1 gene.
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Journal (source)J. Allergy Clin. Immunol.
X-linked primary immunodeficiency associated with hemizygous mutations in the...
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Journal (source)Blood Adv
Gene transfer into hematopoietic stem cells reduces HLH manifestations in a m...
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Journal (source)Cell Death Dis
AK2 deficiency compromises the mitochondrial energy metabolism required for d...
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Journal (source)Stem Cells
Human T-lymphoid progenitors generated in a feeder-cell-free Delta-like-4 cul...
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Journal (source)Blood
Clonal tracking in gene therapy patients reveals a diversity of human hematop...
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Journal (source)Haematologica
A gain-of-function RAC2 mutation is associated with bone-marrow hypoplasia an...
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Journal (source)Blood
Clonal tracking in gene therapy patients reveals a diversity of human hematop...
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Journal (source)J Immunol
Seletalisib for Activated PI3Kδ Syndromes: Open-Label Phase 1b and Extension ...