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Gene Therapy|March 30, 2026
First-in-human nuclease-free homologous recombination-dependent gene editing in pediatric patients with methylmalonic acidemia: results of a phase 1/2 studyJirair K Bedoyan, Thomas Morgan, Angela Sun, et al.
Nature|January 7, 2014
Selection and evaluation of clinically relevant AAV variants in a xenograft liver modelLeszek Lisowski, Allison P Dane, Kirk Chu, et al.
Biomaterials|November 22, 2023
AAV vectors displaying bispecific DARPins enable dual-control targeted gene deliverySamuel A Theuerkauf, Elena Herrera-Carrillo, Fabian John, et al.
Nucleic Acids Research|August 12, 2025
A versatile anti-CRISPR platform for opto- and chemogenetic control of CRISPR-Cas9 and Cas12 across a wide range of orthologsLuca Brenker, Sabine Aschenbrenner, Felix Bubeck, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|April 23, 2025
Identification of a robust promoter in mouse and human hepatocytes by in vivo biopanning of a barcoded AAV libraryJonas Becker, Claire Domenger, Pervinder Choksi, et al.
JCI Insight|June 19, 2019
Coupling AAV-mediated promoterless gene targeting to SaCas9 nuclease to efficiently correct liver metabolic diseasesAlessia De Caneva, Fabiola Porro, Giulia Bortolussi, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|September 6, 2014
AAV8-mediated in vivo overexpression of miR-155 enhances the protective capacity of genetically attenuated malarial parasitesFranziska Hentzschel, Christiane Hammerschmidt-Kamper, Kathleen Börner, et al.
EMBO Reports|September 4, 2024
An hepatitis B and D virus infection model using human pluripotent stem cell-derived hepatocytesHuanting Chi, Bingqian Qu, Angga Prawira, et al.
BMC Cancer|June 20, 2018
MAP3K7 is recurrently deleted in pediatric T-lymphoblastic leukemia and affects cell proliferation independently of NF-κBDavid M Cordas Dos Santos, Juliane Eilers, Alfonso Sosa Vizcaino, et al.
Nature Genetics|December 12, 2018
Immune genes are primed for robust transcription by proximal long noncoding RNAs located in nuclear compartmentsStephanie Fanucchi, Ezio T Fok, Emiliano Dalla, et al.
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