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Molecular Therapy : the Journal of the American Society of Gene Therapy|April 1, 2010
Lymphomagenesis in SCID-X1 mice following lentivirus-mediated phenotype correction independent of insertional mutagenesis and gammac overexpressionSamantha L Ginn, Sophia H Y Liao, Allison P Dane, et al.Science Translational Medicine|August 26, 2011
Long-term persistence of a polyclonal T cell repertoire after gene therapy for X-linked severe combined immunodeficiencyH Bobby Gaspar, Samantha Cooray, Kimberly C Gilmour, et al.Nature Communications|March 13, 2021
Long-term lymphoid progenitors independently sustain naïve T and NK cell production in humansNatalia Izotova, Christine Rivat, Cristina Baricordi, et al.Molecular Therapy. Methods & Clinical Development|October 10, 2022
Preclinical model for phenotypic correction of dystrophic epidermolysis bullosa by in vivo CRISPR-Cas9 delivery using adenoviral vectorsMarta García, Jose Bonafont, Jesús Martínez-Palacios, et al.Frontiers in Immunology|March 6, 2018
Dried Blood Spots, an Affordable Tool to Collect, Ship, and Sequence gDNA from Patients with an X-Linked Agammaglobulinemia Phenotype Residing in a Developing CountryGesmar R S Segundo, Anh T V Nguyen, Huyen T Thuc, et al.The Journal of Allergy and Clinical Immunology|September 13, 2011
Effect of rituximab on human in vivo antibody immune responsesMark D Pescovitz, Troy R Torgerson, Hans D Ochs, et al.Blood|November 5, 2008
Relevance of biallelic versus monoallelic TNFRSF13B mutations in distinguishing disease-causing from risk-increasing TNFRSF13B variants in antibody deficiency syndromesUlrich Salzer, Chiara Bacchelli, Sylvie Buckridge, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|June 14, 2012
Correction of murine Rag2 severe combined immunodeficiency by lentiviral gene therapy using a codon-optimized RAG2 therapeutic transgeneNiek P van Til, Helen de Boer, Nomusa Mashamba, et al.Genes & Diseases|March 17, 2025
Preclinical ex vivo IL2RG gene therapy using autologous hematopoietic stem cells as an effective and safe treatment for X-linked severe combined immunodeficiency diseaseMingfeng Hu, Qiling Xu, Fang Zhang, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|April 2, 2009
A novel model of SCID-X1 reconstitution reveals predisposition to retrovirus-induced lymphoma but no evidence of gammaC gene oncogenicityLinda Scobie, Ralph D Hector, Louise Grant, et al.Pageof 51