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9 in total

1. Lentiviral Gene Therapy for Familial Hemophagocytic Lymphohistiocytosis Type 3, Caused by UNC13D Genetic Defects.

Authors: Sarah E Takushi; Na Yoon Paik; Andrew Fedanov; Chengyu Prince; Christopher B Doering; H Trent Spencer; Shanmuganathan Chandrakasan
Journal: Hum Gene Ther Date: 2020-06 Impact factor: 5.695

2. Gene transfer into hematopoietic stem cells reduces HLH manifestations in a murine model of Munc13-4 deficiency.

Authors: Tayebeh Soheili; Amandine Durand; Fernando E Sepulveda; Julie Rivière; Chantal Lagresle-Peyrou; Hanem Sadek; Geneviève de Saint Basile; Samia Martin; Fulvio Mavilio; Marina Cavazzana; Isabelle André-Schmutz
Journal: Blood Adv Date: 2017-12-21

3. Alternative UNC13D Promoter Encodes a Functional Munc13-4 Isoform Predominantly Expressed in Lymphocytes and Platelets.

Authors: Donatella Galgano; Tayebeh Soheili; Matthias Voss; Lamberto Torralba-Raga; Bianca Tesi; Frank Cichocki; Isabelle Andre; Jens Rettig; Marina Cavazzana; Yenan Bryceson
Journal: Front Immunol Date: 2020-06-09 Impact factor: 7.561

4. A Nontoxic Transduction Enhancer Enables Highly Efficient Lentiviral Transduction of Primary Murine T Cells and Hematopoietic Stem Cells.

Authors: Marianne Delville; Tayebeh Soheili; Florence Bellier; Amandine Durand; Adeline Denis; Chantal Lagresle-Peyrou; Marina Cavazzana; Isabelle Andre-Schmutz; Emmanuelle Six
Journal: Mol Ther Methods Clin Dev Date: 2018-08-08 Impact factor: 6.698

Gene-corrected human Munc13-4-deficient CD8+ T cells can efficiently restrict EBV-driven lymphoproliferation in immunodeficient mice.

1. Lentiviral Gene Therapy for Familial Hemophagocytic Lymphohistiocytosis Type 3, Caused by UNC13D Genetic Defects.

2. Gene transfer into hematopoietic stem cells reduces HLH manifestations in a murine model of Munc13-4 deficiency.

3. Alternative UNC13D Promoter Encodes a Functional Munc13-4 Isoform Predominantly Expressed in Lymphocytes and Platelets.

4. A Nontoxic Transduction Enhancer Enables Highly Efficient Lentiviral Transduction of Primary Murine T Cells and Hematopoietic Stem Cells.

Review 5. Haematopoietic Stem Cell Transplantation for Primary Haemophagocytic Lymphohistiocytosis.

Review 6. Gene Therapy for Primary Immunodeficiency.

Review 7. Gene Edited T Cell Therapies for Inborn Errors of Immunity.

8. Gene therapy for severe combined immunodeficiencies and beyond.

Review 9. Recent advances in primary immunodeficiency: from molecular diagnosis to treatment.