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Human Lentiviral Gene Therapy Restores the Cellular Phenotype of Autosomal Recessive Complete IFN-γR1 Deficiency.
Mol Ther Methods Clin Dev. 2020 Apr 11;17:785-795. doi: 10.1016/j.omtm.2020.04.002. eCollection 2020 Jun 12.
Mol Ther Methods Clin Dev. 2020.
PMID: 32355867
Free PMC article.
Hematopoietic stem cell gene therapy for IFNγR1 deficiency protects mice from mycobacterial infections.
Hetzel M, Mucci A, Blank P, Nguyen AHH, Schiller J, Halle O, Kühnel MP, Billig S, Meineke R, Brand D, Herder V, Baumgärtner W, Bange FC, Goethe R, Jonigk D, Förster R, Gentner B, Casanova JL, Bustamante J, Schambach A, Kalinke U, Lachmann N.
Hetzel M, et al. Among authors: nguyen ahh.
Blood. 2018 Feb 1;131(5):533-545. doi: 10.1182/blood-2017-10-812859. Epub 2017 Dec 12.
Blood. 2018.
PMID: 29233822
Free article.
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ISG15 deficiency features a complex cellular phenotype that responds to treatment with itaconate and derivatives.
Waqas SF, Sohail A, Nguyen AHH, Usman A, Ludwig T, Wegner A, Malik MNH, Schuchardt S, Geffers R, Winterhoff M, Merkert S, Martin U, Olmer R, Lachmann N, Pessler F.
Waqas SF, et al. Among authors: nguyen ahh.
Clin Transl Med. 2022 Jul;12(7):e931. doi: 10.1002/ctm2.931.
Clin Transl Med. 2022.
PMID: 35842904
Free PMC article.
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Effective hematopoietic stem cell-based gene therapy in a murine model of hereditary pulmonary alveolar proteinosis.
Hetzel M, Lopez-Rodriguez E, Mucci A, Nguyen AHH, Suzuki T, Shima K, Buchegger T, Dettmer S, Rodt T, Bankstahl JP, Malik P, Knudsen L, Schambach A, Hansen G, Trapnell BC, Lachmann N, Moritz T.
Hetzel M, et al. Among authors: nguyen ahh.
Haematologica. 2020 Apr;105(4):1147-1157. doi: 10.3324/haematol.2018.214866. Epub 2019 Jul 9.
Haematologica. 2020.
PMID: 31289207
Free PMC article.
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A 3D iPSC-differentiation model identifies interleukin-3 as a regulator of early human hematopoietic specification.
Ackermann M, Haake K, Kempf H, Kaschutnig P, Weiss AC, Nguyen AHH, Abeln M, Merkert S, Kühnel MP, Hartmann D, Jonigk D, Thum T, Kispert A, Milsom MD, Lachmann N.
Ackermann M, et al. Among authors: nguyen ahh.
Haematologica. 2021 May 1;106(5):1354-1367. doi: 10.3324/haematol.2019.228064.
Haematologica. 2021.
PMID: 32327499
Free PMC article.
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