N. Risher

发表

P3.12 Small molecule compounds that correct alternative splicing of the SMN2 gene and restore SMN protein expression and function

S. Peltz, D. Kobayashi, S. Paushkin, 2011, Neuromuscular Disorders.

Discovery of novel HCV inhibitors: synthesis and biological activity of 6-(indol-2-yl)pyridine-3-sulfonamides targeting hepatitis C virus NS4B.

Z. Gu, C. Espiritu, H. Ren, 2013, Bioorganic & Medicinal Chemistry Letters.

Targeting of Hematologic Malignancies with PTC299, A Novel Potent Inhibitor of Dihydroorotate Dehydrogenase with Favorable Pharmaceutical Properties

S. Peltz, A. Jacobson, T. Davis, 2018, Molecular Cancer Therapeutics.

SMN Protein Can Be Reliably Measured in Whole Blood with an Electrochemiluminescence (ECL) Immunoassay: Implications for Clinical Trials

S. Paushkin, T. Prior, K. Swoboda, 2016, PloS one.

Discovery and Optimization of Small Molecule Splicing Modifiers of Survival Motor Neuron 2 as a Treatment for Spinal Muscular Atrophy.

J. Narasimhan, Song Huang, Josephine Sheedy, 2016, Journal of medicinal chemistry.

Ataluren metabolism: Ataluren-O-1β-acyl glucuronide is a stable circulating metabolite in mouse, rat, dog and human.

J. Colacino, Ronald Kong, M. Weetall, 2021, Drug metabolism and pharmacokinetics.

Immunologic Pathways in a Quantitative Model of Immunosuppression Based on Rejection of an Allogeneic or Xenogeneic Tumor Graft

P. Lake, M. Weetall, Jin Zhang, 2005, Transplantation.

Discovery of Novel Small Molecule Inhibitors of VEGF Expression in Tumor Cells Using a Cell-Based High Throughput Screening Platform

Jean Hedrick, S. Peltz, T. Davis, 2016, PloS one.

PTC124 targets genetic disorders caused by nonsense mutations

Meenal Patel, Feng He, Masataka Kawana, 2007, Nature.

SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy

Anirvan Ghosh, Roland Schmucki, Hasane Ratni, 2014, Science.

SMN protein is required throughout life to prevent spinal muscular atrophy disease progression.

F. Metzger, J. Narasimhan, Josephine Sheedy, 2021, Human molecular genetics.

Small molecule splicing modifiers with systemic HTT-lowering activity

S. Peltz, Kerstin A. Effenberger, J. Narasimhan, 2021, Nature Communications.

Abstract 5517: PTC596-induced Bmi1 hyper-phosphorylation via Cdk1/2 activation resulting in tumor stem cell depletion

T. Davis, J. Colacino, Josephine Sheedy, 2014 .