A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout...
A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system
About this item
Full title
Author / Creator
Alterman, Julia F. , Godinho, Bruno M. D. C. , Hassler, Matthew R. , Ferguson, Chantal M. , Echeverria, Dimas , Sapp, Ellen , Haraszti, Reka A. , Coles, Andrew H. , Conroy, Faith , Miller, Rachael , Roux, Loic , Yan, Paul , Knox, Emily G. , Turanov, Anton A. , King, Robert M. , Gernoux, Gwladys , Mueller, Christian , Gray-Edwards, Heather L. , Moser, Richard P. , Bishop, Nina C. , Jaber, Samer M. , Gounis, Matthew J. , Sena-Esteves, Miguel , Pai, Athma A. , DiFiglia, Marian , Aronin, Neil and Khvorova, Anastasia
Publisher
New York: Nature Publishing Group US
Journal title
Language
English
Formats
Publication information
Publisher
New York: Nature Publishing Group US
Subjects
More information
Scope and Contents
Contents
Sustained silencing of gene expression throughout the brain using small interfering RNAs (siRNAs) has not been achieved. Here we describe an siRNA architecture, divalent siRNA (di-siRNA), that supports potent, sustained gene silencing in the central nervous system (CNS) of mice and nonhuman primates following a single injection into the cerebrospin...
Alternative Titles
Full title
A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system
Authors, Artists and Contributors
Author / Creator
Godinho, Bruno M. D. C.
Hassler, Matthew R.
Ferguson, Chantal M.
Echeverria, Dimas
Sapp, Ellen
Haraszti, Reka A.
Coles, Andrew H.
Conroy, Faith
Miller, Rachael
Roux, Loic
Yan, Paul
Knox, Emily G.
Turanov, Anton A.
King, Robert M.
Gernoux, Gwladys
Mueller, Christian
Gray-Edwards, Heather L.
Moser, Richard P.
Bishop, Nina C.
Jaber, Samer M.
Gounis, Matthew J.
Sena-Esteves, Miguel
Pai, Athma A.
DiFiglia, Marian
Aronin, Neil
Khvorova, Anastasia
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_6879195
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_6879195
Other Identifiers
ISSN
1087-0156
E-ISSN
1546-1696
DOI
10.1038/s41587-019-0205-0
