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Risdiplam distributes and increases SMN protein in both the central nervous system and peripheral organs.
Published in:
Pharmacology Research & Perspectives, 2018, v. 6, n. 6, p. 1, doi. 10.1002/prp2.447
By:
- Poirier, Agnès;
- Weetall, Marla;
- Heinig, Katja;
- Bucheli, Franz;
- Schoenlein, Kerstin;
- Alsenz, Jochem;
- Bassett, Simon;
- Ullah, Mohammed;
- Senn, Claudia;
- Ratni, Hasane;
- Naryshkin, Nikolai;
- Paushkin, Sergey;
- Mueller, Lutz
Publication type:
Article
Upstream promoter sequences and αCTD mediate stable DNA wrapping within the RNA polymerase–promoter open complex.
Published in:
EMBO Reports, 2007, v. 8, n. 3, p. 271, doi. 10.1038/sj.embor.7400888
By:
- Cellai, Sara;
- Mangiarotti, Laura;
- Vannini, Nicola;
- Naryshkin, Nikolai;
- Kortkhonjia, Ekaterine;
- Ebright, Richard H;
- Rivetti, Claudio
Publication type:
Article
Small molecule splicing modifiers with systemic HTT-lowering activity.
Published in:
Nature Communications, 2021, v. 12, n. 1, p. 1, doi. 10.1038/s41467-021-27157-z
By:
- Bhattacharyya, Anuradha;
- Trotta, Christopher R.;
- Narasimhan, Jana;
- Wiedinger, Kari J.;
- Li, Wencheng;
- Effenberger, Kerstin A.;
- Woll, Matthew G.;
- Jani, Minakshi B.;
- Risher, Nicole;
- Yeh, Shirley;
- Cheng, Yaofeng;
- Sydorenko, Nadiya;
- Moon, Young-Choon;
- Karp, Gary M.;
- Weetall, Marla;
- Dakka, Amal;
- Gabbeta, Vijayalakshmi;
- Naryshkin, Nikolai A.;
- Graci, Jason D.;
- Tripodi Jr., Thomas
Publication type:
Article
A deep learning approach to identify gene targets of a therapeutic for human splicing disorders.
Published in:
Nature Communications, 2021, v. 12, n. 1, p. 1, doi. 10.1038/s41467-021-23663-2
By:
- Gao, Dadi;
- Morini, Elisabetta;
- Salani, Monica;
- Krauson, Aram J.;
- Chekuri, Anil;
- Sharma, Neeraj;
- Ragavendran, Ashok;
- Erdin, Serkan;
- Logan, Emily M.;
- Li, Wencheng;
- Dakka, Amal;
- Narasimhan, Jana;
- Zhao, Xin;
- Naryshkin, Nikolai;
- Trotta, Christopher R.;
- Effenberger, Kerstin A.;
- Woll, Matthew G.;
- Gabbeta, Vijayalakshmi;
- Karp, Gary;
- Yu, Yong
Publication type:
Article
Utility of Survival Motor Neuron ELISA for Spinal Muscular Atrophy Clinical and Preclinical Analyses.
Published in:
PLoS ONE, 2011, v. 6, n. 8, p. 1, doi. 10.1371/journal.pone.0024269
By:
- Kobayashi, Dione T.;
- Olson, Rory J.;
- Sly, Laurel;
- Swanson, Chad J.;
- Chung, Brett;
- Naryshkin, Nikolai;
- Narasimhan, Jana;
- Bhattacharyya, Anuradha;
- Mullenix, Michael;
- Chen, Karen S.
Publication type:
Article
Age-dependent SMN expression in disease-relevant tissue and implications for SMA treatment.
Published in:
2019
By:
- Ramos, Daniel M.;
- d'Ydewalle, Constantin;
- Gabbeta, Vijayalakshmi;
- Dakka, Amal;
- Klein, Stephanie K.;
- Norris, Daniel A.;
- Matson, John;
- Taylor, Shannon J.;
- Zaworski, Phillip G.;
- Prior, Thomas W.;
- Snyder, Pamela J.;
- Valdivia, David;
- Hatem, Christine L.;
- Waters, Ian;
- Gupte, Nikhil;
- Swoboda, Kathryn J.;
- Rigo, Frank;
- Bennett, C. Frank;
- Naryshkin, Nikolai;
- Paushkin, Sergey
Publication type:
journal article
Assays for the Identification and Prioritization of Drug Candidates for Spinal Muscular Atrophy.
Published in:
Assay & Drug Development Technologies, 2014, v. 12, n. 6, p. 315, doi. 10.1089/adt.2014.587
By:
- Cherry, Jonathan J.;
- Kobayashi, Dione T.;
- Lynes, Maureen M.;
- Naryshkin, Nikolai N.;
- Tiziano, Francesco Danilo;
- Zaworski, Phillip G.;
- Rubin, Lee L.;
- Jarecki, Jill
Publication type:
Article
SMN protein is required throughout life to prevent spinal muscular atrophy disease progression.
Published in:
Human Molecular Genetics, 2022, v. 31, n. 1, p. 82, doi. 10.1093/hmg/ddab220
By:
- Zhao, Xin;
- Feng, Zhihua;
- Risher, Nicole;
- Mollin, Anna;
- Sheedy, Josephine;
- Ling, Karen K Y;
- Narasimhan, Jana;
- Dakka, Amal;
- Baird, John D;
- Ratni, Hasane;
- Lutz, Catherine;
- Chen, Karen S;
- Naryshkin, Nikolai A;
- Ko, Chien-Ping;
- Welch, Ellen;
- Metzger, Friedrich;
- Weetall, Marla
Publication type:
Article
Corrigendum to: Pharmacologically-induced mouse model of adult spinal muscular atrophy to evaluate effectiveness of therapeutics after disease onset.
Published in:
Human Molecular Genetics, 2021, v. 30, n. 9, p. 843, doi. 10.1093/hmg/ddaa247
By:
- Feng, Zhihua;
- Ling, Karen K Y;
- Zhao, Xin;
- Zhou, Chunyi;
- Karp, Gary;
- Welch, Ellen M;
- Naryshkin, Nikolai;
- Ratni, Hasane;
- Chen, Karen S;
- Metzger, Friedrich;
- Paushkin, Sergey;
- Weetall, Marla;
- Ko, Chien-Ping
Publication type:
Article
Pharmacokinetics, pharmacodynamics, and efficacy of a small-molecule SMN2 splicing modifier in mouse models of spinal muscular atrophy.
Published in:
Human Molecular Genetics, 2016, v. 25, n. 10, p. 1885, doi. 10.1093/hmg/ddw062
By:
- Xin Zhao;
- Zhihua Feng;
- Ling, Karen K. Y.;
- Mollin, Anna;
- Sheedy, Josephine;
- Yeh, Shirley;
- Petruska, Janet;
- Narasimhan, Jana;
- Dakka, Amal;
- Welch, Ellen M.;
- Karp, Gary;
- Chen, Karen S.;
- Metzger, Friedrich;
- Ratni, Hasane;
- Lotti, Francesco;
- Tisdale, Sarah;
- Naryshkin, Nikolai A.;
- Pellizzoni, Livio;
- Paushkin, Sergey;
- Chien-Ping Ko
Publication type:
Article
Pharmacologically induced mouse model of adult spinal muscular atrophy to evaluate effectiveness of therapeutics after disease onset.
Published in:
Human Molecular Genetics, 2016, v. 25, n. 5, p. 964, doi. 10.1093/hmg/ddv629
By:
- Zhihua Feng;
- Ling, Karen K. Y.;
- Xin Zhao;
- Chunyi Zhou;
- Karp, Gary;
- Welch, Ellen M.;
- Naryshkin, Nikolai;
- Ratni, Hasane;
- Chen, Karen S.;
- Metzger, Friedrich;
- Paushkin, Sergey;
- Weetall, Marla;
- Chien-Ping Ko
Publication type:
Article
Detection of Glycosylase, Endonuclease and Methyltransferase Enzyme Activities Using Immobilized Oligonucleotides.
Published in:
IUBMB Life, 2004, v. 56, n. 3, p. 139, doi. 10.1080/15216540410001671925
By:
- Sudina, Anna;
- Volkov, Eugeny;
- Oretskaya, Tatiana;
- Naryshkin, Nikolai;
- Ivanovskaya, Marina;
- Kubareva, Elena
Publication type:
Article
Impaired prenatal motor axon development necessitates early therapeutic intervention in severe SMA.
Published in:
Science Translational Medicine, 2021, v. 13, n. 578, p. 1, doi. 10.1126/scitranslmed.abb6871
By:
- Kong, Lingling;
- Valdivia, David O.;
- Simon, Christian M.;
- Hassinan, Cera W.;
- Delestrée, Nicolas;
- Ramos, Daniel M.;
- Park, Jae Hong;
- Pilato, Celeste M.;
- Xu, Xixi;
- Crowder, Melissa;
- Grzyb, Chloe C.;
- King, Zachary A.;
- Petrillo, Marco;
- Swoboda, Kathryn J.;
- Davis, Crystal;
- Lutz, Cathleen M.;
- Stephan, Alexander H.;
- Zhao, Xin;
- Weetall, Marla;
- Naryshkin, Nikolai A.
Publication type:
Article

Found: 13