Presentation
Resources & publications
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2019Journal (source)Blood AdvGene transfer into hematopoietic stem cells reduces HLH manifestations in a m...
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2019Journal (source)Haematologica
Extensive multilineage analysis in patients with mixed chimerism after alloge...
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2019Journal (source)Blood
Lentiviral and genome-editing strategies for the treatment of β-hemoglobinopa...
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2019Journal (source)Hum. Mol. Genet.
Gene therapy of hemoglobinopathies: progress and future challenges.
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2019Journal (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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2019Journal (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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2016Journal (source)Hum. Gene Ther.
Gene Therapy for X-Linked Severe Combined Immunodeficiency: Where Do We Stand?
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2017Journal (source)Stem Cells Dev.
Gene Therapy with Hematopoietic Stem Cells: The Diseased Bone Marrow's Point ...
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2018Journal (source)Blood
Gene-corrected human Munc13-4-deficient CD8+ T cells can efficiently restrict...
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2019Journal (source)J. Clin. Invest.
Loss of ARHGEF1 causes a human primary antibody deficiency.
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2018Journal (source)Haematologica
Mutations in the adaptor-binding domain and associated linker region of p110δ...
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Journal (source)J. Allergy Clin. Immunol. 2017
Clinical and immunologic phenotype associated with activated phosphoinositide...
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2015Journal (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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2019Journal (source)Blood Adv
Gene transfer into hematopoietic stem cells reduces HLH manifestations in a m...
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2016Journal (source)Cell Death Dis
AK2 deficiency compromises the mitochondrial energy metabolism required for d...
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2013Journal (source)Stem Cells
Human T-lymphoid progenitors generated in a feeder-cell-free Delta-like-4 cul...
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2020Journal (source)Blood
Clonal tracking in gene therapy patients reveals a diversity of human hematop...
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2020Journal (source)Haematologica
A gain-of-function RAC2 mutation is associated with bone-marrow hypoplasia an...
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2020Journal (source)Blood
Clonal tracking in gene therapy patients reveals a diversity of human hematop...
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2020Journal (source)J Immunol
Seletalisib for Activated PI3Kδ Syndromes: Open-Label Phase 1b and Extension ...
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2018Journal (source)Front Immunol
Disease Evolution and Response to Rapamycin in Activated Phosphoinositide 3-K...
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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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2018Journal (source)Front Immunol
Disease Evolution and Response to Rapamycin in Activated Phosphoinositide 3-K...
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2019Journal (source)Blood Adv
Baboon envelope LVs efficiently transduced human adult, fetal, and progenitor...
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2021Journal (source)Blood
A combination of cyclophosphamide and interleukin-2 allows CD4+ T cells conve...
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2017Journal (source)Blood
Reticular dysgenesis: international survey on clinical presentation, transpla...
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2015Journal (source)J Allergy Clin Immunol
An in vivo genetic reversion highlights the crucial role of Myb-Like, SWIRM, ...
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2014Journal (source)J Allergy Clin Immunol
The BLNK adaptor protein has a nonredundant role in human B-cell differentiat...
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2014Journal (source)J Allergy Clin Immunol
Successful RAG1-SCID gene therapy depends on the level of RAG1 expression.
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2014Journal (source)J Allergy Clin Immunol
Recombination-activating gene 1 (Rag1)-deficient mice with severe combined im...
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2019Journal (source)Bone Marrow Transplant
Ex vivo generated human T-lymphoid progenitors as a tool to accelerate immune...
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2017Journal (source)Stem Cells Dev
Gene Therapy with Hematopoietic Stem Cells: The Diseased Bone Marrow's Point ...
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2015Journal (source)JAMA
Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndr...
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2020Journal (source)Am J Transplant
Donor-targeted serotherapy as a rescue therapy for steroid-resistant acute GV...
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2021Journal (source)Nat Immunol
Single-cell analysis of FOXP3 deficiencies in humans and mice unmasks intrins...
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2021Journal (source)Cell Mol Immunol
A DL-4- and TNFα-based culture system to generate high numbers of nonmodified...
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2021Journal (source)Nat Commun
Transient mTOR inhibition rescues 4-1BB CAR-Tregs from tonic signal-induced d...
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2022Journal (source)Nat Med
Long-term outcomes of lentiviral gene therapy for the β-hemoglobinopathies: t...
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2022Journal (source)Nat Commun
Base-editing-mediated dissection of a γ-globin cis-regulatory element for the...
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2023Journal (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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2023Journal (source)Blood
Adenine base editor-mediated correction of the common and severe IVS1-110 (G>...
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2022Journal (source)Mol Ther
Combination of lentiviral and genome editing technologies for the treatment o...