The use of miR122 and its target sequence in adeno-associated virus-mediated trichosanthin gene therapy

Relief of microRNA-Mediated Translational Repression in Human Cells Subjected to Stress

Suvendra N. Bhattacharyya, Regula Habermacher, Ursula Martiné et al. · Cell · 2006 · 1.3K citations · Full text

Life Sciences, Cell Biology, Molecular Biology +14

Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant

Lindsey A. George, Spencer K. Sullivan, Adam Giermasz et al. · New England Journal of Medicine · 2017 · 759 citations · Full text

Medicine, Hematology, Molecular Medicine +15

Endogenous microRNA can be broadly exploited to regulate transgene expression according to tissue, lineage and differentiation state

Brian D. Brown, Bernhard Gentner, Alessio Cantore et al. · Nature Biotechnology · 2007 · 558 citations

Life Sciences, Molecular Biology, Molecular Genetics +16

A long-term study of AAV gene therapy in dogs with hemophilia A identifies clonal expansions of transduced liver cells

Giang N. Nguyen, J.K. Everett, Samita Kafle et al. · Nature Biotechnology · 2020 · 445 citations · Full text

Medicine, Hematology, Molecular Medicine +8

MicroRNA-mediated inhibition of transgene expression reduces dorsal root ganglion toxicity by AAV vectors in primates

Juliette Hordeaux, Elizabeth L. Buza, Brianne A Jeffrey et al. · Science Translational Medicine · 2020 · 170 citations

Life Sciences, Biotechnology, Neuroscience +17