N. Naryshkin

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.

Structural Organization of the RNA Polymerase-Promoter Open Complex

Younggyu Kim, R. Ebright, A. Revyakin, 2000, Cell.

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.

Risdiplam distributes and increases SMN protein in both the central nervous system and peripheral organs

S. Bassett, S. Paushkin, J. Alsenz, 2018, Pharmacology research & perspectives.

Static and kinetic site-specific protein-DNA photocrosslinking: analysis of bacterial transcription initiation complexes.

R. Ebright, V. Mekler, S. Druzhinin, 2009, Methods in molecular biology.

Site-specific protein-DNA photocrosslinking. Analysis of bacterial transcription initiation complexes.

R. Ebright, N. Naryshkin, Q. Dong, 2001, Methods in molecular biology.

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.

Development of an oral treatment that rescues gait ataxia and retinal degeneration in a phenotypic mouse model of familial dysautonomia

S. Slaugenhaupt, J. Narasimhan, A. Mollin, 2023, American journal of human genetics.

Nonsense suppression activity of PTC124 (ataluren)

S. Peltz, H. Sweeney, A. Jacobson, 2009, Proceedings of the National Academy of Sciences.

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

S. Peltz, J. Luban, C. Strambio-De-Castillia, 2020, Virus Research.

The DHODH Inhibitor PTC299 Arrests SARS-CoV-2 Replication and Suppresses Induction of Inflammatory Cytokines

S. Peltz, J. Luban, C. Strambio-De-Castillia, 2020, bioRxiv.

Upstream promoter sequences and αCTD mediate stable DNA wrapping within the RNA polymerase–promoter open complex

R. Ebright, C. Rivetti, Ekaterine Kortkhonjia, 2007, EMBO reports.

Transcription Factor B Contacts Promoter DNA Near the Transcription Start Site of the Archaeal Transcription Initiation Complex*

R. Ebright, R. A. Scott, N. Naryshkin, 2004, Journal of Biological Chemistry.

Synthetic RNA Modification and Cross-linking Approaches towards the Structure of the Hairpin Ribozyme and the HIV-1 Tat Protein Interaction with TAR RNA

N. Naryshkin, M. Gait, M. Farrow, 1998 .

Chemical cross-linking of the human immunodeficiency virus type 1 Tat protein to synthetic models of the RNA recognition sequence TAR containing site-specific trisubstituted pyrophosphate analogues.

N. Naryshkin, M. Gait, Z. Shabarova, 1997, Biochemistry.

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.

Discovery of Risdiplam, a Selective Survival of Motor Neuron-2 ( SMN2) Gene Splicing Modifier for the Treatment of Spinal Muscular Atrophy (SMA).

Michael Reutlinger, Andreas Zeller, Stefan Kustermann, 2018, Journal of medicinal chemistry.

Impaired prenatal motor axon development necessitates early therapeutic intervention in severe SMA

T. Crawford, G. Mentis, C. Lutz, 2021, Science Translational Medicine.

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.

RNA recognition and regulation of HIV-1 gene expression by viral factor Tat.

N. Naryshkin, M. Gait, M. Ivanovskaya, 1998, Biochemistry. Biokhimiia.

ELP1 Splicing Correction Reverses Proprioceptive Sensory Loss in Familial Dysautonomia.

M. Talkowski, S. Slaugenhaupt, P. Dietrich, 2019, American journal of human genetics.

Small molecule splicing modifiers with systemic HTT-lowering activity

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

Detection of Glycosylase, Endonuclease and Methyltransferase Enzyme Activities Using Immobilized Oligonucleotides

T. Oretskaya, E. Kubareva, A. Sudina, 2004, IUBMB life.

Discovery of a Novel Class of Survival Motor Neuron 2 Splicing Modifiers for the Treatment of Spinal Muscular Atrophy.

F. Metzger, E. Pinard, S. Paushkin, 2017, Journal of medicinal chemistry.