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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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The DHODH inhibitor PTC299 arrests SARS-CoV-2 replication and suppresses induction of inflammatory cytokines

Virus research, 2021-01, Vol.292, p.198246-198246, Article 198246

2021

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

PloS one, 2018-10, Vol.13 (10), p.e0206158

2018

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Preclinical and Early Clinical Development of PTC596, a Novel Small-Molecule Tubulin-Binding Agent

Molecular cancer therapeutics, 2021-10, Vol.20 (10), p.1846-1857

2021

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

PloS one, 2016-12, Vol.11 (12), p.e0168366

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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Emvododstat, a Potent Dihydroorotate Dehydrogenase Inhibitor, Is Effective in Preclinical Models of Acute Myeloid Leukemia

Frontiers in oncology, 2022-02, Vol.12, p.832816-832816

2022

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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

Journal of medicinal chemistry, 2014-03, Vol.57 (5), p.2121-2135

2014

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In vitro cytochrome P450‐ and transporter‐mediated drug interaction potential of 6β‐hydroxy‐21‐desacetyl deflazacort—A major human metabolite of deflazacort

Pharmacology research & perspectives, 2021-04, Vol.9 (2), p.e00748-n/a

2021

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In vitro metabolism, reaction phenotyping, enzyme kinetics, CYP inhibition and induction potential of ataluren

Pharmacology research & perspectives, 2020-04, Vol.8 (2), p.e00576-n/a

2020

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

Molecular cancer therapeutics, 2019-01, Vol.18 (1), p.3-16

2019

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Oligomerization and Cooperative RNA Synthesis Activity of Hepatitis C Virus RNA-Dependent RNA Polymerase

Journal of Virology, 2002-04, Vol.76 (8), p.3865-3872

2002

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The nucleoside analog clitocine is a potent and efficacious readthrough agent

RNA (Cambridge), 2017-04, Vol.23 (4), p.567-577

2017

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PTC725, an NS4B-Targeting Compound, Inhibits a Hepatitis C Virus Genotype 3 Replicon, as Predicted by Genome Sequence Analysis and Determined Experimentally

Antimicrobial agents and chemotherapy, 2016-12, Vol.60 (12), p.7060-7066

2016

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Toxicity of Antiviral Nucleoside Analogs and the Human Mitochondrial DNA Polymerase

The Journal of biological chemistry, 2001-11, Vol.276 (44), p.40847-40857

2001

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Professor De Clercq and 25 Years of International Collaboration on Antiviral Research

Antiviral chemistry & chemotherapy, 2013-04, Vol.23 (2), p.57-58

Colacino, Joseph M

2013

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High-throughput Screening Uncovers a Compound That Activates Latent HIV-1 and Acts Cooperatively with a Histone Deacetylase (HDAC) Inhibitor

The Journal of biological chemistry, 2011-06, Vol.286 (24), p.21083-21091

2011

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The identification and development of antiviral agents for the treatment of chronic hepatitis B virus infection

Progress in drug research, 1998, Vol.50, p.259

1998

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Antiviral activity and toxicity of fialuridine in the woodchuck model of hepatitis B virus infection

Hepatology (Baltimore, Md.), 1998-07, Vol.28 (1), p.179-191

1998

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Molecular Basis for the Resistance of Influenza Viruses to 4-Guanidino-Neu5Ac2en

Virology (New York, N.Y.), 1995-12, Vol.214 (2), p.642-646

1995

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HIV Type-1 Latency: Targeted Induction of Proviral Reservoirs

Antiviral Chemistry and Chemotherapy, 2009-04, Vol.19 (5), p.177-187

2009

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Mammalian DNA Polymerases .alpha., .beta., .gamma., .delta., and .epsilon. Incorporate Fialuridine (FIAU) Monophosphate into DNA and Are Inhibited Competitively by FIAU Triphosphate

Biochemistry (Easton), 1994-12, Vol.33 (48), p.14620-14624

1994

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Small molecule splicing modifiers with systemic HTT-lowering activity

The DHODH inhibitor PTC299 arrests SARS-CoV-2 replication and suppresses induction of inflammatory cytokines

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

Preclinical and Early Clinical Development of PTC596, a Novel Small-Molecule Tubulin-Binding Agent

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

Identification of benzazole compounds that induce HIV-1 transcription

Emvododstat, a Potent Dihydroorotate Dehydrogenase Inhibitor, Is Effective in Preclinical Models of Acute Myeloid Leukemia

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

In vitro cytochrome P450‐ and transporter‐mediated drug interaction potential of 6β‐hydroxy‐21‐desacetyl deflazacort—A major human metabolite of deflazacort

In vitro metabolism, reaction phenotyping, enzyme kinetics, CYP inhibition and induction potential of ataluren

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

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

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

Oligomerization and Cooperative RNA Synthesis Activity of Hepatitis C Virus RNA-Dependent RNA Polymerase

PTC124 targets genetic disorders caused by nonsense mutations

The nucleoside analog clitocine is a potent and efficacious readthrough agent

PTC725, an NS4B-Targeting Compound, Inhibits a Hepatitis C Virus Genotype 3 Replicon, as Predicted by Genome Sequence Analysis and Determined Experimentally

Toxicity of Antiviral Nucleoside Analogs and the Human Mitochondrial DNA Polymerase

Professor De Clercq and 25 Years of International Collaboration on Antiviral Research

High-throughput Screening Uncovers a Compound That Activates Latent HIV-1 and Acts Cooperatively with a Histone Deacetylase (HDAC) Inhibitor

The identification and development of antiviral agents for the treatment of chronic hepatitis B virus infection

Antiviral activity and toxicity of fialuridine in the woodchuck model of hepatitis B virus infection

Molecular Basis for the Resistance of Influenza Viruses to 4-Guanidino-Neu5Ac2en

HIV Type-1 Latency: Targeted Induction of Proviral Reservoirs

Mammalian DNA Polymerases .alpha., .beta., .gamma., .delta., and .epsilon. Incorporate Fialuridine (FIAU) Monophosphate into DNA and Are Inhibited Competitively by FIAU Triphosphate

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