Zobrazeno 1 - 10
of 23
pro vyhledávání: '"Nenad Svrzikapa"'
Autor:
Britt Hanson, Sofia Stenler, Nina Ahlskog, Katarzyna Chwalenia, Nenad Svrzikapa, Anna M.L. Coenen-Stass, Marc S. Weinberg, Matthew J.A. Wood, Thomas C. Roberts
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 30, Iss , Pp 379-397 (2022)
Duchenne muscular dystrophy (DMD) is the most prevalent inherited myopathy affecting children, caused by genetic loss of the gene encoding the dystrophin protein. Here we have investigated the use of the Staphylococcus aureus CRISPR-Cas9 system and a
Externí odkaz:
https://doaj.org/article/6f00b10bd1c34b468b19eea787f859a7
Autor:
Nenad Svrzikapa, Kenneth A. Longo, Nripesh Prasad, Ramakrishna Boyanapalli, Jeffrey M. Brown, Daniel Dorset, Scott Yourstone, Jason Powers, Shawn E. Levy, Aaron J. Morris, Chandra Vargeese, Jaya Goyal
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 19, Iss , Pp 162-173 (2020)
Novel treatments for Huntington’s disease (HD), a progressive neurodegenerative disorder, include selective targeting of the mutant allele of the huntingtin gene (mHTT) carrying the abnormally expanded disease-causing cytosine-adenine-guanine (CAG)
Externí odkaz:
https://doaj.org/article/65d1f0e1bbb64c3b9d640e8783a84fb1
Autor:
Howard A. Burris, Jared A. Gollob, Dinah W.Y. Sah, Akshay K. Vaishnaw, Christina Gamba-Vitalo, Saraswathy V. Nochur, Jeffrey Cehelsky, Valerie A. Clausen, Renta M. Hutabarat, Nenad Svrzikapa, Gregory Hinkle, Rachel E. Meyers, David Bumcrot, Iva Toudjarska, Amy C. Seila White, Jamie Harrop, Rick Falzone, Mrinal M. Gounder, Maria Alsina, Jeffrey R. Infante, Daniel C. Cho, Luis Paz-Ares, Glen J. Weiss, Gary K. Schwartz, Andres Cervantes, Patricia M. LoRusso, Geoffrey I. Shapiro, Josep Tabernero
Supplementary Figures PDF file - 114K, Includes sequence information on KSP and VEGF siRNAs, murine Hep3B tumor model data with ALN-VSP, ALN-VSP Phase I study design, KSP and VEGF mRNA levels in tumor cell lines and normal liver, and comparison of AL
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::798921fa41c39c58506772199a018c15
https://doi.org/10.1158/2159-8290.22530521
https://doi.org/10.1158/2159-8290.22530521
Autor:
Howard A. Burris, Jared A. Gollob, Dinah W.Y. Sah, Akshay K. Vaishnaw, Christina Gamba-Vitalo, Saraswathy V. Nochur, Jeffrey Cehelsky, Valerie A. Clausen, Renta M. Hutabarat, Nenad Svrzikapa, Gregory Hinkle, Rachel E. Meyers, David Bumcrot, Iva Toudjarska, Amy C. Seila White, Jamie Harrop, Rick Falzone, Mrinal M. Gounder, Maria Alsina, Jeffrey R. Infante, Daniel C. Cho, Luis Paz-Ares, Glen J. Weiss, Gary K. Schwartz, Andres Cervantes, Patricia M. LoRusso, Geoffrey I. Shapiro, Josep Tabernero
Supplementary Methods and Legends PDF file - 72K, Includes detailed methods for 5' RACE assay and DCE-MRI scans, as well as figure legends for the six supplementary figures
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::b60fded2d3b851bda5dc56f8308997f4
https://doi.org/10.1158/2159-8290.22530518
https://doi.org/10.1158/2159-8290.22530518
Autor:
Howard A. Burris, Jared A. Gollob, Dinah W.Y. Sah, Akshay K. Vaishnaw, Christina Gamba-Vitalo, Saraswathy V. Nochur, Jeffrey Cehelsky, Valerie A. Clausen, Renta M. Hutabarat, Nenad Svrzikapa, Gregory Hinkle, Rachel E. Meyers, David Bumcrot, Iva Toudjarska, Amy C. Seila White, Jamie Harrop, Rick Falzone, Mrinal M. Gounder, Maria Alsina, Jeffrey R. Infante, Daniel C. Cho, Luis Paz-Ares, Glen J. Weiss, Gary K. Schwartz, Andres Cervantes, Patricia M. LoRusso, Geoffrey I. Shapiro, Josep Tabernero
Supplementary Tables PDF file - 67K, Includes data showing effect of ALN-VSP on spleen in non-human primates, tumor response data from the Phase I trial, and safety data (including adverse events and dose-limiting toxicities) from the Phase I trial
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::2abf232c1a30a2513228f151bdf1bb65
https://doi.org/10.1158/2159-8290.22530515
https://doi.org/10.1158/2159-8290.22530515
Publikováno v:
Methods in Molecular Biology ISBN: 9781071628188
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::4501be382191c8794f91c5b8facf59cc
https://doi.org/10.1007/978-1-0716-2819-5_3
https://doi.org/10.1007/978-1-0716-2819-5_3
Publikováno v:
Methods in molecular biology (Clifton, N.J.). 2590
Haplotyping individual full-length transcripts can be important in diagnosis and treatment of certain genetic diseases. One set of diseases, repeat expansions of simple tandem repeat sequences are the cause of over 40 neurological disorders. In many
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=pmid________::6b57f9a822087e8977e0af286aeeb4e9
https://pubmed.ncbi.nlm.nih.gov/36335491
https://pubmed.ncbi.nlm.nih.gov/36335491
Autor:
Daniel Dorset, Ramakrishna Boyanapalli, Kenneth Longo, Jeffrey M. Brown, Aaron J. Morris, Nripesh Prasad, Scott Yourstone, Shawn Levy, Chandra Vargeese, Nenad Svrzikapa, Jaya Goyal, Jason Powers
Publikováno v:
Molecular Therapy. Methods & Clinical Development
Molecular Therapy: Methods & Clinical Development, Vol 19, Iss, Pp 162-173 (2020)
Molecular Therapy: Methods & Clinical Development, Vol 19, Iss, Pp 162-173 (2020)
Novel treatments for Huntington’s disease (HD), a progressive neurodegenerative disorder, include selective targeting of the mutant allele of the huntingtin gene (mHTT) carrying the abnormally expanded disease-causing cytosine-adenine-guanine (CAG)
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::b6a876e8e2835d79e2bba28b9a1adca6
http://europepmc.org/articles/PMC7648085
http://europepmc.org/articles/PMC7648085
Autor:
Mary Edmondson, Mark S. LeDoux, H. Diana Rosas, Vicki L. Wheelock, Michael A. Panzara, E. Ray Dorsey, Nenad Svrzikapa, Kenneth Longo, Jaya Goyal, Sandra K. Kostyk, Francis O. Walker, Jody Corey-Bloom, Serena Hung, Ralf Reilmann, Daniel O. Claassen
Publikováno v:
Neurology: Genetics
Neurology. Genetics, vol 6, iss 3
Neurology. Genetics, vol 6, iss 3
BackgroundThe huntingtin gene (HTT) pathogenic cytosine-adenine-guanine (CAG) repeat expansion responsible for Huntington disease (HD) is phased with single nucleotide polymorphisms (SNPs), providing targets for allele-selective treatments.ObjectiveT
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::f7f5f3f8d8167346914b9ab104beb78c
https://doi.org/10.1212/nxg.0000000000000430
https://doi.org/10.1212/nxg.0000000000000430
Autor:
Kenneth Longo, Serena Hung, Nenad Svrzikapa, Mary Edmondson, Ralf Reilmann, Michael Panzara, Jaya Goyal, Daniel O. Claassen
Publikováno v:
Clinical therapeutics.
Background Expansion of a cytosine-adenine-guanine (CAG) triplet repeat in the Huntingtin (HTT) gene causes HD through production and accumulation of mutant HTT (mHTT) protein, leading to progressive loss of neurons in the brain. The pathogenic CAG r
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::5364d1c6c992f9f4dcfaffd67fc20f34
https://doi.org/10.1136/jnnp-2018-ehdn.264
https://doi.org/10.1136/jnnp-2018-ehdn.264