SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy
SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy
About this item
Full title
Author / Creator
Naryshkin, Nikolai A. , Weetall, Marla , Dakka, Amal , Narasimhan, Jana , Zhao, Xin , Feng, Zhihua , Ling, Karen K. Y. , Karp, Gary M. , Qi, Hongyan , Woll, Matthew G. , Chen, Guangming , Zhang, Nanjing , Gabbeta, Vijayalakshmi , Vazirani, Priya , Bhattacharyya, Anuradha , Furia, Bansri , Risher, Nicole , Sheedy, Josephine , Kong, Ronald , Ma, Jiyuan , Turpoff, Anthony , Lee, Chang-Sun , Zhang, Xiaoyan , Moon, Young-Choon , Trifillis, Panayiota , Welch, Ellen M. , Colacino, Joseph M. , Babiak, John , Almstead, Neil G. , Peltz, Stuart W. , Eng, Loren A. , Chen, Karen S. , Mull, Jesse L. , Lynes, Maureen S. , Rubin, Lee L. , Fontoura, Paulo , Santarelli, Luca , Haehnke, Daniel , McCarthy, Kathleen D. , Schmucki, Roland , Ebeling, Martin , Sivaramakrishnan, Manaswini , Ko, Chien-Ping , Paushkin, Sergey V. , Ratni, Hasane , Gerlach, Irene , Ghosh, Anirvan and Metzger, Friedrich
Publisher
Washington: American Association for the Advancement of Science
Journal title
Language
English
Formats
Publication information
Publisher
Washington: American Association for the Advancement of Science
Subjects
More information
Scope and Contents
Contents
Spinal muscular atrophy (SMA) is a genetic disease caused by mutation or deletion of the survival of motor neuron 1 (SMN1) gene. A paralogous gene in humans, SMN2, produces low, insufficient levels of functional SMN protein due to alternative splicing that truncates the transcript. The decreased levels of SMN protein lead to progressive neuromuscul...
Alternative Titles
Full title
SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy
Authors, Artists and Contributors
Author / Creator
Weetall, Marla
Dakka, Amal
Narasimhan, Jana
Zhao, Xin
Feng, Zhihua
Ling, Karen K. Y.
Karp, Gary M.
Qi, Hongyan
Woll, Matthew G.
Chen, Guangming
Zhang, Nanjing
Gabbeta, Vijayalakshmi
Vazirani, Priya
Bhattacharyya, Anuradha
Furia, Bansri
Risher, Nicole
Sheedy, Josephine
Kong, Ronald
Ma, Jiyuan
Turpoff, Anthony
Lee, Chang-Sun
Zhang, Xiaoyan
Moon, Young-Choon
Trifillis, Panayiota
Welch, Ellen M.
Colacino, Joseph M.
Babiak, John
Almstead, Neil G.
Peltz, Stuart W.
Eng, Loren A.
Chen, Karen S.
Mull, Jesse L.
Lynes, Maureen S.
Rubin, Lee L.
Fontoura, Paulo
Santarelli, Luca
Haehnke, Daniel
McCarthy, Kathleen D.
Schmucki, Roland
Ebeling, Martin
Sivaramakrishnan, Manaswini
Ko, Chien-Ping
Paushkin, Sergey V.
Ratni, Hasane
Gerlach, Irene
Ghosh, Anirvan
Metzger, Friedrich
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_proquest_miscellaneous_1904238838
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_miscellaneous_1904238838
Other Identifiers
ISSN
0036-8075
E-ISSN
1095-9203
DOI
10.1126/science.1250127
