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Molecular Therapy : the Journal of the American Society of Gene Therapy|July 14, 2011
Correction of murine SCID-X1 by lentiviral gene therapy using a codon-optimized IL2RG gene and minimal pretransplant conditioningMarshall W Huston, Niek P van Til, Trudi P Visser, et al.
The Journal of Experimental Medicine|December 21, 2016
Autoinflammatory periodic fever, immunodeficiency, and thrombocytopenia (PFIT) caused by mutation in actin-regulatory gene WDR1Ariane S I Standing, Dessislava Malinova, Ying Hong, et al.
Human Gene Therapy|February 1, 2020
High-Throughput In Vitro, Ex Vivo, and In Vivo Screen of Adeno-Associated Virus Vectors Based on Physical and Functional TransductionAdrian Westhaus, Marti Cabanes-Creus, Arkadiusz Rybicki, et al.
Molecular Therapy. Methods & Clinical Development|June 2, 2015
Coherence analysis discriminates between retroviral integration patterns in CD34(+) cells transduced under differing clinical trial conditionsClaus V Hallwirth, Gagan Garg, Timothy J Peters, et al.
The Journal of Allergy and Clinical Immunology|March 13, 2024
Hematopoietic stem cell gene editing rescues B-cell development in X-linked agammaglobulinemiaSameer Bahal, Marta Zinicola, Shefta E Moula, et al.
Nature Medicine|February 24, 2006
Effective gene therapy with nonintegrating lentiviral vectorsRafael J Yáñez-Muñoz, Kamaljit S Balaggan, Angus MacNeil, et al.
Blood|December 3, 2011
AAV-mediated gene transfer in the perinatal period results in expression of FVII at levels that protect against fatal spontaneous hemorrhageChristopher Binny, Jenny McIntosh, Marco Della Peruta, et al.
Lancet (London, England)|November 11, 2024
Betibeglogene autotemcel gene therapy in patients with transfusion-dependent, severe genotype β-thalassaemia (HGB-212): a non-randomised, multicentre, single-arm, open-label, single-dose, phase 3 trialJanet L Kwiatkowski, Mark C Walters, Suradej Hongeng, et al.
Plos Neglected Tropical Diseases|August 5, 2021
The clinical spectrum and immunopathological mechanisms underlying ZIKV-induced neurological manifestationsIgor Salerno Filgueiras, Amanda Torrentes de Carvalho, Daniela Prado Cunha, et al.
The Journal of Allergy and Clinical Immunology|July 8, 2008
Novel signal transducer and activator of transcription 3 (STAT3) mutations, reduced T(H)17 cell numbers, and variably defective STAT3 phosphorylation in hyper-IgE syndromeEllen D Renner, Stacey Rylaarsdam, Stephanie Anover-Sombke, et al.
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