Jeffrey Cehelsky

CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis

Julian D. Gillmore, Ed Gane, Jörg Täubel et al. · New England Journal of Medicine · 2021 · 1.5K citations · Full text

Safety and Efficacy of RNAi Therapy for Transthyretin Amyloidosis

Teresa Coelho, David Adams, Ana Silva et al. · New England Journal of Medicine · 2013 · 947 citations · Full text

First-in-Humans Trial of an RNA Interference Therapeutic Targeting VEGF and KSP in Cancer Patients with Liver Involvement

Josep Tabernero, Geoffrey I. Shapiro, Patricia LoRusso et al. · Cancer Discovery · 2013 · 691 citations

Effect of an RNA interference drug on the synthesis of proprotein convertase subtilisin/kexin type 9 (PCSK9) and the concentration of serum LDL cholesterol in healthy volunteers: a randomised, single-blind, placebo-controlled, phase 1 trial

Kevin Fitzgerald, Maria Frank-Kamenetsky, Svetlana Shulga‐Morskaya et al. · The Lancet · 2013 · 571 citations · Full text

Metabolic Syndrome, Oxysterol, Biochemistry +9

A randomized, double-blind, placebo-controlled study of an RNAi-based therapy directed against respiratory syncytial virus

John P. DeVincenzo, Rob Lambkin‐Williams, Tom Wilkinson et al. · Proceedings of the National Academy of Sciences · 2010 · 434 citations · Full text

Rsv Antibody, Respiratory Syncytial Virus, Rnai-based Therapy +14