Gene Therapy for SCID-X1: Focus on Clinical Data
Author:
Publisher
Elsevier BV
Subject
Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine
Reference14 articles.
1. Long-term persistence of a polyclonal T cell repertoire after gene therapy for X-linked severe combined immunodeficiency;Gaspar;Sci Transl Med,2011
2. Efficacy of gene therapy for X-linked severe combined immunodeficiency;Hacein-Bey-Abina;N Engl J Med,2010
3. Long-term survival and transplantation of haemopoietic stem cells for immunodeficiencies: report of the European experience 1968–99;Antoine;Lancet,2003
4. Transplantation of hematopoietic stem cells and long-term survival for primary immunodeficiencies in Europe: entering a new century, do we do better;Gennery;J Allergy Clin Immunol,2010
5. Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients;Howe;J Clin Invest,2008
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1. Gene correction of HAX1 reversed Kostmann disease phenotype in patient-specific induced pluripotent stem cells;Blood Advances;2017-06-02
2. Genomic discovery of potent chromatin insulators for human gene therapy;Nature Biotechnology;2015-01-12
3. Blood Induction and Embryonic Formation;Principles of Developmental Genetics;2015
4. The State of the Art and Future of Gene Medicines;Pharmaceutical Sciences Encyclopedia;2013-11-25
5. Gene therapy for Wiskott–Aldrich syndrome;Expert Opinion on Orphan Drugs;2013-08-13
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