Zobrazeno 1 - 10
of 11
pro vyhledávání: '"C. Frank Bennet"'
Autor:
Moira A. McMahon, Meghdad Rahdar, Swagatam Mukhopadhyay, Huynh-Hoa Bui, Christopher Hart, Sagar Damle, Margo Courtney, Michael W. Baughn, Don W. Cleveland, C. Frank Bennett
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 32, Iss , Pp 289-301 (2023)
Antisense oligonucleotides (ASOs) are short synthetic nucleic acids that recognize and bind to complementary RNA to modulate gene expression. It is well established that single-stranded, phosphorothioate-modified ASOs enter cells independent of carri
Externí odkaz:
https://doaj.org/article/b4603aa807a3456aa6ae0c476b922870
Autor:
Crystal N. Doty, Eric E. Swayze, Michael R. Hayden, Nicholas S. Caron, Holly Kordasiewicz, Susan M. Freier, C. Frank Bennet, Yuanyun Xie, Amber L. Southwell, Niels H. Skotte, Boguslaw Felczak, Punit P. Seth, Erika B. Villanueva, Michael Oestergaard
Publikováno v:
Molecular Therapy. 23
Huntington disease (HD) is a dominant, genetic neurodegenerative disease characterized by progressive loss of voluntary motor control, psychiatric disturbance, and cognitive decline, for which there is currently no disease-modifying therapy. HD is ca
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::d3ca6d18226ecf67b3ce7e1af720b41d
Autor:
Kung-Chi Chang, Sarah D. Diermeier, Allen T. Yu, Lily D. Brine, Suzanne Russo, Sonam Bhatia, Habeeb Alsudani, Karen Kostroff, Tawfiqul Bhuiya, Edi Brogi, Darryl J. Pappin, C. Frank Bennett, Frank Rigo, David L. Spector
Publikováno v:
Nature Communications, Vol 11, Iss 1, Pp 1-19 (2020)
A group of long non-coding RNAs (lncRNAs), Mammary Tumor Associated RNA s 1-30 (MaTARs 1-30), are differentially expressed between mammary tumor cells and normal mammary epithelial cells. Here the authors report that MaTAR25 plays a role in breast ca
Externí odkaz:
https://doaj.org/article/6dafc61ca8e54b14af09f6dca2979a49
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 16, Iss , Pp 313-325 (2019)
Splice-switching antisense oligonucleotides (ASOs) are promising therapeutic tools to target various genetic diseases, including cancer. However, in vivo delivery of ASOs to orthotopic tumors in cancer mouse models or to certain target tissues remain
Externí odkaz:
https://doaj.org/article/e178c55d3a444d6099f64f44b7a50fce
Autor:
Ningyan Hu, Layal Antoury, Timothy M. Baran, Soumya Mitra, C. Frank Bennett, Frank Rigo, Thomas H. Foster, Thurman M. Wheeler
Publikováno v:
Nature Communications, Vol 9, Iss 1, Pp 1-15 (2018)
Myotonic dystrophy type 1 (DM1) is associated with aberrant transcript splicing. Here, the authors develop a transgenic mouse model expressing a bi-chromatic reporter system that allows non-invasive monitoring of splicing of a transcript altered in D
Externí odkaz:
https://doaj.org/article/b1565fb81d1d487aac6b620c6b16e1b3
Autor:
Tracy A. Cole, Hien Zhao, Timothy J. Collier, Ivette Sandoval, Caryl E. Sortwell, Kathy Steece-Collier, Brian F. Daley, Alix Booms, Jack Lipton, Mackenzie Welch, Melissa Berman, Luke Jandreski, Danielle Graham, Andreas Weihofen, Stephanie Celano, Emily Schulz, Allyson Cole-Strauss, Esteban Luna, Duc Quach, Apoorva Mohan, C. Frank Bennett, Eric E. Swayze, Holly B. Kordasiewicz, Kelvin C. Luk, Katrina L. Paumier
Publikováno v:
JCI Insight, Vol 6, Iss 5 (2021)
Parkinson’s disease (PD) is a prevalent neurodegenerative disease with no approved disease-modifying therapies. Multiplications, mutations, and single nucleotide polymorphisms in the SNCA gene, encoding α-synuclein (aSyn) protein, either cause or
Externí odkaz:
https://doaj.org/article/d06ea6f3c76f4b97a20be199ac938389
Autor:
Dominic Jauvin, Jessina Chrétien, Sanjay K. Pandey, Laurie Martineau, Lucille Revillod, Guillaume Bassez, Aline Lachon, A. Robert McLeod, Geneviève Gourdon, Thurman M. Wheeler, Charles A. Thornton, C. Frank Bennett, Jack Puymirat
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 7, Iss C, Pp 465-474 (2017)
Myotonic dystrophy type 1 (DM1), a dominant hereditary muscular dystrophy, is caused by an abnormal expansion of a (CTG)n trinucleotide repeat in the 3′ UTR of the human dystrophia myotonica protein kinase (DMPK) gene. As a consequence, mutant tran
Externí odkaz:
https://doaj.org/article/b019e0b86069472cbd72368b12dffd00
Autor:
Sarah D. Diermeier, Kung-Chi Chang, Susan M. Freier, Junyan Song, Osama El Demerdash, Alexander Krasnitz, Frank Rigo, C. Frank Bennett, David L. Spector
Publikováno v:
Cell Reports, Vol 17, Iss 1, Pp 261-274 (2016)
Long non-coding RNAs (lncRNAs) represent the largest and most diverse class of non-coding RNAs, comprising almost 16,000 currently annotated transcripts in human and 10,000 in mouse. Here, we investigated the role of lncRNAs in mammary tumors by perf
Externí odkaz:
https://doaj.org/article/c2fa708b91c44e2c9a56c1372141076a
Autor:
Andrew P. Lieberman, Zhigang Yu, Sue Murray, Raechel Peralta, Audrey Low, Shuling Guo, Xing Xian Yu, Constanza J. Cortes, C. Frank Bennett, Brett P. Monia, Albert R. La Spada, Gene Hung
Publikováno v:
Cell Reports, Vol 7, Iss 3, Pp 774-784 (2014)
Spinal and bulbar muscular atrophy (SBMA) is caused by the polyglutamine androgen receptor (polyQ-AR), a protein expressed by both lower motor neurons and skeletal muscle. Although viewed as a motor neuronopathy, data from patients and mouse models s
Externí odkaz:
https://doaj.org/article/362a686dbe4b43e48c2719a1d0ab43eb
Autor:
Kentaro Sahashi, Karen K. Y. Ling, Yimin Hua, John Erby Wilkinson, Tomoki Nomakuchi, Frank Rigo, Gene Hung, David Xu, Ya‐Ping Jiang, Richard Z. Lin, Chien‐Ping Ko, C. Frank Bennett, Adrian R. Krainer
Publikováno v:
EMBO Molecular Medicine, Vol 5, Iss 10, Pp 1586-1601 (2013)
Abstract Loss‐of‐function mutations in SMN1 cause spinal muscular atrophy (SMA), a leading genetic cause of infant mortality. The related SMN2 gene expresses suboptimal levels of functional SMN protein, due to a splicing defect. Many SMA patients
Externí odkaz:
https://doaj.org/article/02f20b7d6ba64acfbfeeba4de2cef7d3