Josephine Sheedy

<i>SMN2</i> splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy

Nikolai A. Naryshkin, Marla Weetall, Amal Dakka et al. · Science · 2014 · 526 citations

Life Sciences, Molecular Biology, Neuroscience +17

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

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

Matthew G. Woll, Hongyan Qi, Anthony Turpoff et al. · Journal of Medicinal Chemistry · 2016 · 61 citations

Life Sciences, Medicinal Chemistry, Neuroscience +13

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

BMI1 is a therapeutic target in recurrent medulloblastoma

David Bakhshinyan, Chitra Venugopal, Ashley Adile et al. · Oncogene · 2018 · 44 citations

Medicine, Cancer Biology, Oncology +12