45 results on '"Naryshkin, Nikolai"'
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2. A deep learning approach to identify gene targets of a therapeutic for human splicing disorders
3. Small molecule splicing modifiers with systemic HTT-lowering activity
4. Age-dependent SMN expression in disease-relevant tissue and implications for SMA treatment
5. Drugging Pre-mRNA Splicing
6. Development of a novel oral treatment that rescues gait ataxia and retinal degeneration in a phenotypic mouse model of familial dysautonomia
7. SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy
8. Drugging Pre-mRNA Splicing
9. SMN protein is required throughout life to prevent spinal muscular atrophy disease progression
10. MOTOR NEURON DISEASE: SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy
11. Impaired prenatal motor axon development necessitates early therapeutic intervention in severe SMA
12. The DHODH inhibitor PTC299 arrests SARS-CoV-2 replication and suppresses induction of inflammatory cytokines
13. Corrigendum to: Pharmacologically-induced mouse model of adult spinal muscular atrophy to evaluate effectiveness of therapeutics after disease onset
14. SMN protein is required throughout life to prevent spinal muscular atrophy disease progression.
15. Static and Kinetic Site-Specific Protein-DNA Photocrosslinking: Analysis of Bacterial Transcription Initiation Complexes
16. The DHODH Inhibitor PTC299 Arrests SARS-CoV-2 Replication and Suppresses Induction of Inflammatory Cytokines
17. A deep learning approach to identify new gene targets of a novel therapeutic for human splicing disorders
18. 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
19. Detection of Glycosylase, Endonuclease and Methyltransferase Enzyme Activities Using Immobilized Oligonucleotides
20. Synthetic RNA Modification and Cross-linking Approaches towards the Structure of the Hairpin Ribozyme and the HIV-1 Tat Protein Interaction with TAR RNA
21. ELP1 Splicing Correction Reverses Proprioceptive Sensory Loss in Familial Dysautonomia
22. Risdiplam distributes and increases SMN protein in both the central nervous system and peripheral organs
23. Discovery of Risdiplam, a Selective Survival of Motor Neuron-2 (SMN2) Gene Splicing Modifier for the Treatment of Spinal Muscular Atrophy (SMA)
24. Relationship Between Central and Peripheral SMN Protein Increase Upon Treatment with RO7034067 (RG7916) (S46.007)
25. Discovery of a Novel Class of Survival Motor Neuron 2 Splicing Modifiers for the Treatment of Spinal Muscular Atrophy
26. Specific Correction of Alternative Survival Motor Neuron 2 Splicing by Small Molecules: Discovery of a Potential Novel Medicine To Treat Spinal Muscular Atrophy
27. Discovery and Optimization of Small Molecule Splicing Modifiers of Survival Motor Neuron 2 as a Treatment for Spinal Muscular Atrophy
28. Pharmacokinetics, pharmacodynamics, and efficacy of a small-moleculeSMN2splicing modifier in mouse models of spinal muscular atrophy
29. Pharmacologically induced mouse model of adult spinal muscular atrophy to evaluate effectiveness of therapeutics after disease onset
30. Site-Specific Protein-DNA Photocrosslinking: Analysis of Bacterial Transcription Initiation Complexes
31. Assays for the Identification and Prioritization of Drug Candidates for Spinal Muscular Atrophy
32. Pharmacokinetics, pharmacodynamics, and efficacy of a small-molecule SMN2 splicing modifier in mouse models of spinal muscular atrophy.
33. Pharmacologically induced mouse model of adult spinal muscular atrophy to evaluate effectiveness of therapeutics after disease onset.
34. Utility of Survival Motor Neuron ELISA for Spinal Muscular Atrophy Clinical and Preclinical Analyses
35. Nonsense suppression activity of PTC124 (ataluren)
36. Upstream promoter sequences and αCTD mediate stable DNA wrapping within the RNA polymerase–promoter open complex
37. Transcription Factor B Contacts Promoter DNA Near the Transcription Start Site of the Archaeal Transcription Initiation Complex
38. Site-Specific Protein-DNA Photocrosslinking.
39. Structural Organization of the RNA Polymerase-Promoter Open Complex
40. Corrigendum to: Pharmacologically-induced mouse model of adult spinal muscular atrophy to evaluate effectiveness of therapeutics after disease onset.
41. 2 - Synthetic RNA Modification and Cross-linking Approaches towards the Structure of the Hairpin Ribozyme and the HIV-1 Tat Protein Interaction with TAR RNA
42. The DHODH Inhibitor PTC299 Arrests SARS-CoV-2 Replication and Suppresses Induction of Inflammatory Cytokines.
43. Pharmacokinetics, pharmacodynamics, and efficacy of a small-molecule SMN2 splicing modifier in mouse models of spinal muscular atrophy.
44. Upstream promoter sequences and alphaCTD mediate stable DNA wrapping within the RNA polymerase-promoter open complex.
45. Site-specific protein-DNA photo-cross-linking: analysis of structural organization of protein-DNA and multiprotein-DNA complexes.
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