Jean Hedrick

PTC124 targets genetic disorders caused by nonsense mutations

Ellen Welch, Elisabeth R. Barton, Jin Zhuo et al. · Nature · 2007 · 1.1K citations

Life Sciences, Genetics, Molecular Genetics +8

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

Liangxian Cao, Marla Weetall, Christopher R. Trotta et al. · Molecular Cancer Therapeutics · 2018 · 85 citations

Medicine, Oncology, Pharmacology +14

ELP1 Splicing Correction Reverses Proprioceptive Sensory Loss in Familial Dysautonomia

Elisabetta Morini, Dadi Gao, Connor M. Montgomery et al. · The American Journal of Human Genetics · 2019 · 50 citations · Full text

Life Sciences, Genetics, Molecular Genetics +6

The nucleoside analog clitocine is a potent and efficacious readthrough agent

Westley J. Friesen, Christopher R. Trotta, Yuki Tomizawa et al. · RNA · 2017 · 48 citations · Full text

Medicine, Genetics, Oncology +12

The minor gentamicin complex component, X2, is a potent premature stop codon readthrough molecule with therapeutic potential

Westley J. Friesen, Briana Johnson, Jairo Sierra et al. · PLoS ONE · 2018 · 38 citations · Full text

Medicine, Pharmacology, Pharmacotherapy +14