Hemophilia as a blueprint for gene therapy

Enhancement of liver-directed transgene expression at initial and repeat doses of AAV vectors admixed with ImmTOR nanoparticles

(2021) Petr O. Ilyinskii et al.   Science Advances

Factor VIII exhibits chaperone-dependent and glucose-regulated reversible amyloid formation in the endoplasmic reticulum

(2020) Juthakorn Poothong et al.   BLOOD

IgG-cleaving endopeptidase enables in vivo gene therapy in the presence of anti-AAV neutralizing antibodies

(2020) Christian Leborgne et al.   NATURE MEDICINE

CRISPR/Cas9-mediated in vivo gene targeting corrects hemostasis in newborn and adult factor IX knockout mice

(2019) Lili Wang et al.   BLOOD

Subcutaneous concizumab prophylaxis in hemophilia A and hemophilia A/B with inhibitors: Phase 2 trial results

(2019) Amy D. Shapiro et al.   BLOOD

Hemophilia A ameliorated in mice by CRISPR-based in vivo genome editing of human Factor VIII

(2019) Hainan Chen et al.   Scientific Reports

CIRCLE-seq: a highly sensitive in vitro screen for genome-wide CRISPR–Cas9 nuclease off-targets

(2017) Shengdar Q Tsai et al.   NATURE METHODS

Targeting of Antithrombin in Hemophilia A or B with RNAi Therapy

(2017) K. John Pasi et al.   NEW ENGLAND JOURNAL OF MEDICINE

Emicizumab Prophylaxis in Hemophilia A with Inhibitors

(2017) Johannes Oldenburg et al.   NEW ENGLAND JOURNAL OF MEDICINE

AAV5–Factor VIII Gene Transfer in Severe Hemophilia A

(2017) Savita Rangarajan et al.   NEW ENGLAND JOURNAL OF MEDICINE

Murine coagulation factor VIII is synthesized in endothelial cells

(2014) L. A. Everett et al.   BLOOD