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Small molecule splicing modifiers with systemic HTT-lowering activity

Nature communications, 2021-12, Vol.12 (1), p.7299-7299, Article 7299

2021

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

Science (American Association for the Advancement of Science), 2014-08, Vol.345 (6197), p.688-693

2014

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Specific Correction of Alternative Survival Motor Neuron 2 Splicing by Small Molecules: Discovery of a Potential Novel Medicine To Treat Spinal Muscular Atrophy

Journal of medicinal chemistry, 2016-07, Vol.59 (13), p.6086-6100

2016

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Identification of benzazole compounds that induce HIV-1 transcription

PloS one, 2017-06, Vol.12 (6), p.e0179100

2017

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A deep learning approach to identify gene targets of a therapeutic for human splicing disorders

Nature communications, 2021-06, Vol.12 (1), p.3332-3332, Article 3332

2021

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

Journal of medicinal chemistry, 2016-07, Vol.59 (13), p.6070-6085

2016

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

Bioorganic & medicinal chemistry letters, 2013-07, Vol.23 (13), p.3947-3953

2013

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Discovery of N-(4′-(indol-2-yl)phenyl)sulfonamides as novel inhibitors of HCV replication

Bioorganic & medicinal chemistry letters, 2013-07, Vol.23 (13), p.3942-3946

2013

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Discovery and Optimization of Indolyl-Containing 4‑Hydroxy-2-Pyridone Type II DNA Topoisomerase Inhibitors Active against Multidrug Resistant Gram-negative Bacteria

Journal of medicinal chemistry, 2018-05, Vol.61 (10), p.4456-4475

2018

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Ribonucleotide reductase, a novel drug target for gonorrhea

eLife, 2022-02, Vol.11

2022

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4-Hydroxy-2-pyridones: Discovery and evaluation of a novel class of antibacterial agents targeting DNA synthesis

Bioorganic & medicinal chemistry letters, 2017-11, Vol.27 (22), p.5014-5021

2017

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Correction to Discovery and Optimization of Indolyl-Containing 4‑Hydroxy-2-pyridone Type II DNA Topoisomerase Inhibitors Active against Multidrug Resistant Gram-Negative Bacteria

Journal of medicinal chemistry, 2018-10, Vol.61 (20), p.9394-9394

2018

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Discovery of 2-(4-sulfonamidophenyl)-indole 3-carboxamides as potent and selective inhibitors with broad hepatitis C virus genotype activity targeting HCV NS4B

Bioorganic & medicinal chemistry letters, 2016-01, Vol.26 (2), p.594-601

2016

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6-(Azaindol-2-yl)pyridine-3-sulfonamides as potent and selective inhibitors targeting hepatitis C virus NS4B

Bioorganic & medicinal chemistry letters, 2015-02, Vol.25 (4), p.781-786

2015

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Identification of PTC725, an Orally Bioavailable Small Molecule That Selectively Targets the Hepatitis C Virus NS4B Protein

Antimicrobial Agents and Chemotherapy, 2013-07, Vol.57 (7), p.3250-3261

2013

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Corrigendum to: Pharmacologically-induced mouse model of adult spinal muscular atrophy to evaluate effectiveness of therapeutics after disease onset

Human molecular genetics, 2021-05, Vol.30 (9), p.843-843

2021

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Palladium(II)-mediated coupling reactions of bromo- and iodo-1H-1,4-benzodiazepine-2,5-diones. A route to functionalized benzodiazepinediones

Journal of organic chemistry, 1995-09, Vol.60 (18), p.5814-5819

Karp, Gary M

1995

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Pharmacokinetics, pharmacodynamics, and efficacy of a small-molecule SMN2 splicing modifier in mouse models of spinal muscular atrophy

Human molecular genetics, 2016-05, Vol.25 (10), p.1885-1899

2016

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Synthesis and Herbicidal Activity of 1H-1,4-Benzodiazepine-2,5-diones

Journal of agricultural and food chemistry, 1997-02, Vol.45 (2), p.493-500

1997

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Regioselective alkylation of phenoxy-substituted 3-(methylthio)indolin-2(3H)-ones. Preparation of 3-, 1,3-, and 1,3,3-substituted indolin-2(3H)-ones

Journal of organic chemistry, 1992-08, Vol.57 (17), p.4765-4772

Karp, Gary M

1992

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Drugging Pre-mRNA Splicing

RNA Therapeutics, 2017, Vol.27, p.135-176

Garner, Amanda L

2017

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Motor neuron disease. SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy

Science (American Association for the Advancement of Science), 2014-08, Vol.345 (6197), p.688-693

2014

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Synthesis and alkylation of 3,4‐dihydro‐1 H ‐1,3‐4‐benzotriazepine‐2,5‐diones and related systems

Journal of heterocyclic chemistry, 1996-07, Vol.33 (4), p.1131-1135

Karp, Gary M.

1996

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Small molecule splicing modifiers with systemic HTT-lowering activity

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

Specific Correction of Alternative Survival Motor Neuron 2 Splicing by Small Molecules: Discovery of a Potential Novel Medicine To Treat Spinal Muscular Atrophy

Identification of benzazole compounds that induce HIV-1 transcription

A deep learning approach to identify gene targets of a therapeutic for human splicing disorders

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

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

Structure–Activity Relationship (SAR) Optimization of 6‑(Indol-2-yl)pyridine-3-sulfonamides: Identification of Potent, Selective, and Orally Bioavailable Small Molecules Targeting Hepatitis C (HCV) NS4B

Discovery of N-(4′-(indol-2-yl)phenyl)sulfonamides as novel inhibitors of HCV replication

Discovery and Optimization of Indolyl-Containing 4‑Hydroxy-2-Pyridone Type II DNA Topoisomerase Inhibitors Active against Multidrug Resistant Gram-negative Bacteria

Ribonucleotide reductase, a novel drug target for gonorrhea

4-Hydroxy-2-pyridones: Discovery and evaluation of a novel class of antibacterial agents targeting DNA synthesis

Correction to Discovery and Optimization of Indolyl-Containing 4‑Hydroxy-2-pyridone Type II DNA Topoisomerase Inhibitors Active against Multidrug Resistant Gram-Negative Bacteria

Discovery of 2-(4-sulfonamidophenyl)-indole 3-carboxamides as potent and selective inhibitors with broad hepatitis C virus genotype activity targeting HCV NS4B

6-(Azaindol-2-yl)pyridine-3-sulfonamides as potent and selective inhibitors targeting hepatitis C virus NS4B

Identification of PTC725, an Orally Bioavailable Small Molecule That Selectively Targets the Hepatitis C Virus NS4B Protein

Corrigendum to: Pharmacologically-induced mouse model of adult spinal muscular atrophy to evaluate effectiveness of therapeutics after disease onset

PTC124 targets genetic disorders caused by nonsense mutations

Palladium(II)-mediated coupling reactions of bromo- and iodo-1H-1,4-benzodiazepine-2,5-diones. A route to functionalized benzodiazepinediones

Pharmacokinetics, pharmacodynamics, and efficacy of a small-molecule SMN2 splicing modifier in mouse models of spinal muscular atrophy

Synthesis and Herbicidal Activity of 1H-1,4-Benzodiazepine-2,5-diones

Regioselective alkylation of phenoxy-substituted 3-(methylthio)indolin-2(3H)-ones. Preparation of 3-, 1,3-, and 1,3,3-substituted indolin-2(3H)-ones

Drugging Pre-mRNA Splicing

Motor neuron disease. SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy

Synthesis and alkylation of 3,4‐dihydro‐1 H ‐1,3‐4‐benzotriazepine‐2,5‐diones and related systems

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