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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.
Molecular Therapy : the Journal of the American Society of Gene Therapy|October 22, 2009
Low-level shRNA cytotoxicity can contribute to MYC-induced hepatocellular carcinoma in adult miceShelly Beer, David I Bellovin, Joyce S Lee, et al.
Blood|June 7, 2003
Preclinical in vivo evaluation of pseudotyped adeno-associated virus vectors for liver gene therapyDirk Grimm, Shangzhen Zhou, Hiroyuki Nakai, 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.
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.
EMBO Molecular Medicine|April 9, 2025
Clinically translatable mitochondrial gene therapy in muscle using tandem mtZFN architecturePavel A Nash, Keira M Turner, Christopher A Powell, et al.
Nature Genetics|January 17, 2019
Publisher Correction: Immune genes are primed for robust transcription by proximal long noncoding RNAs located in nuclear compartmentsStephanie Fanucchi, Ezio T Fok, Emiliano Dalla, et al.
Nature Methods|November 1, 2018
Engineered anti-CRISPR proteins for optogenetic control of CRISPR-Cas9Felix Bubeck, Mareike D Hoffmann, Zander Harteveld, et al.
Molecular Therapy. Methods & Clinical Development|February 16, 2019
Novel Chimeric Gene Therapy Vectors Based on Adeno-Associated Virus and Four Different Mammalian BocavirusesJulia Fakhiri, Marc A Schneider, Jens Puschhof, et al.
Science Translational Medicine|December 1, 2017
Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophyLeonela Amoasii, Chengzu Long, Hui Li, et al.
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