Presentation
Resources & publications
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Journal (source)Mol Ther Methods Clin DevA 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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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)Haematologica
A gain-of-function RAC2 mutation is associated with bone-marrow hypoplasia an...
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2021Journal (source)J Allergy Clin Immunol
Improving the diagnostic efficiency of primary immunodeficiencies with target...
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Journal (source)Mol Ther Methods Clin Dev
Successful Preclinical Development of Gene Therapy for Recombinase-Activating...
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2020Journal (source)Hum Mol Genet
NHP2 deficiency impairs rRNA biogenesis and causes pulmonary fibrosis and Høy...
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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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2019Journal (source)Front Pediatr
Hematopoietic Stem Cell Transplant for the Treatment of X-MAID.
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2019Journal (source)Blood Adv
Baboon envelope LVs efficiently transduced human adult, fetal, and progenitor...
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2021Journal (source)J Allergy Clin Immunol
Improving the diagnostic efficiency of primary immunodeficiencies with target...
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2021Journal (source)Blood Adv
Adenylate kinase 2 expression and addiction in T-ALL.
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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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2017Journal (source)Stem Cells Dev
Gene Therapy with Hematopoietic Stem Cells: The Diseased Bone Marrow's Point ...
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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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2024Journal (source)Cancer Res
Comprehensive Genetic Profiling Reveals Frequent Alterations of Driver Genes ...