G. Karp

发表

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

F. Metzger, E. Pinard, W. Muster, 2016, Journal of medicinal chemistry.

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

S. Peltz, X. Tong, Z. Gu, 2013, Antimicrobial Agents and Chemotherapy.

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.

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

F. Metzger, S. Paushkin, Chunyi Zhou, 2016, Human molecular genetics.

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

S. Erdin, Yong Yu, M. Talkowski, 2020, bioRxiv.

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

Z. Gu, A. Nomeir, F. Lahser, 2016, Bioorganic & medicinal chemistry letters.

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.

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

J. Narasimhan, A. Branstrom, Josephine Sheedy, 2017, Bioorganic & medicinal chemistry letters.

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.

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

Z. Gu, H. Ren, A. Nomeir, 2015, Bioorganic & medicinal chemistry letters.

PREPARATION AND REACTIONS OF INDOLIN-2(3H)-ONES. A REVIEW

G. Karp, 1993 .

Preparation and reactions of [2,2-bis(carbethoxy)propyl]cyclopentadienyldicarbonyliron

M. Kaufman, G. Karp, R. Kerber, 1982 .

Synthesis and herbicidal activity of 1H-1,4-benzodiazepine-2,5-diones

G. Karp, Gary M. Karp, Mark C. Manfredi, 1997 .

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.

Ramesh Kakarla, Guangming Chen, Song Huang, 2014, Journal of medicinal chemistry.

SYNTHESIS AND ALKYLATION OF 3,4-DIHYDRO-1H-1,3,4-BENZOTRIAZEPINE-2,5-DIONES AND RELATED SYSTEMS

G. Karp, 1996 .

PTC124 targets genetic disorders caused by nonsense mutations

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

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

G. Karp, 1992 .

Drugging Pre-mRNA Splicing

G. Karp, N. Naryshkin, M. Woll, 2017 .

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

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

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

S. Erdin, Yong Yu, M. Talkowski, 2021, Nature Communications.

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

F. Metzger, S. Paushkin, L. Pellizzoni, 2016, Human molecular genetics.

Identification of benzazole compounds that induce HIV-1 transcription

Z. Gu, J. Colacino, Jason D Graci, 2017, PloS one.

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

Z. Gu, H. Ren, J. Colacino, 2013, Bioorganic & medicinal chemistry letters.

Ribonucleotide reductase, a novel target for gonorrhea

J. Stubbe, J. Narasimhan, A. Branstrom, 2020, bioRxiv.

Small molecule splicing modifiers with systemic HTT-lowering activity

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