Biblio
Constitutive IL-1RA production by modified immune cells protects against IL-1-mediated inflammatory disorders. Sci Transl Med. 2023;15(698):eade3856.
CRISPR-based gene disruption and integration of high-avidity, WT1-specific T cell receptors improve antitumor T cell function. Sci Transl Med. 2022;14(631):eabg8027.
Disrupting N-glycan expression on tumor cells boosts chimeric antigen receptor T cell efficacy against solid malignancies. Sci Transl Med. 2022;14(628):eabg3072.
Generation of Human Induced Pluripotent Stem Cell-Derived Bona Fide Neural Stem Cells for Ex Vivo Gene Therapy of Metachromatic Leukodystrophy. Stem Cells Transl Med. 2016.
Hematopoietic Tumors in a Mouse Model of X-linked Chronic Granulomatous Disease after Lentiviral Vector-Mediated Gene Therapy. Mol Ther. 2020.
Human iPSC-based neurodevelopmental models of globoid cell leukodystrophy uncover patient- and cell type-specific disease phenotypes. Stem Cell Reports. 2021.
Scalable GMP-compliant gene correction of CD4+ T cells with IDLV template functionally validated and . Mol Ther Methods Clin Dev. 2023;30:546-557.