Dual-targeting CRISPR-CasRx reduces C9orf72 ALS/FTD sense and antisense repeat RNAs in vitro and in...
Dual-targeting CRISPR-CasRx reduces C9orf72 ALS/FTD sense and antisense repeat RNAs in vitro and in vivo
About this item
Full title
Author / Creator
Kempthorne, Liam , Vaizoglu, Deniz , Cammack, Alexander J. , Carcolé, Mireia , Roberts, Martha J. , Mikheenko, Alla , Fisher, Alessia , Suklai, Pacharaporn , Muralidharan, Bhavana , Kroll, François , Moens, Thomas G. , Yshii, Lidia , Verschoren, Stijn , Hölbling, Benedikt V. , Moreira, Francisco C. , Katona, Eszter , Coneys, Rachel , de Oliveira, Paula , Zhang, Yong-Jie , Jansen, Karen , Daughrity, Lillian M. , McGown, Alexander , Ramesh, Tennore M. , Van Den Bosch, Ludo , Lignani, Gabriele , Rahim, Ahad A. , Coyne, Alyssa N. , Petrucelli, Leonard , Rihel, Jason and Isaacs, Adrian M.
Publisher
London: Nature Publishing Group UK
Journal title
Language
English
Formats
Publication information
Publisher
London: Nature Publishing Group UK
Subjects
More information
Scope and Contents
Contents
The most common genetic cause of frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS) is an intronic G
4
C
2
repeat expansion in
C9orf72
. The repeats undergo bidirectional transcription to produce sense and antisense repeat RNA species, which are translated into dipeptide repeat proteins (DPRs). As toxicity has b...
Alternative Titles
Full title
Dual-targeting CRISPR-CasRx reduces C9orf72 ALS/FTD sense and antisense repeat RNAs in vitro and in vivo
Authors, Artists and Contributors
Author / Creator
Vaizoglu, Deniz
Cammack, Alexander J.
Carcolé, Mireia
Roberts, Martha J.
Mikheenko, Alla
Fisher, Alessia
Suklai, Pacharaporn
Muralidharan, Bhavana
Kroll, François
Moens, Thomas G.
Yshii, Lidia
Verschoren, Stijn
Hölbling, Benedikt V.
Moreira, Francisco C.
Katona, Eszter
Coneys, Rachel
de Oliveira, Paula
Zhang, Yong-Jie
Jansen, Karen
Daughrity, Lillian M.
McGown, Alexander
Ramesh, Tennore M.
Van Den Bosch, Ludo
Lignani, Gabriele
Rahim, Ahad A.
Coyne, Alyssa N.
Petrucelli, Leonard
Rihel, Jason
Isaacs, Adrian M.
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_doaj_primary_oai_doaj_org_article_01e4420112ec452d903fa41f98416f89
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_01e4420112ec452d903fa41f98416f89
Other Identifiers
ISSN
2041-1723
E-ISSN
2041-1723
DOI
10.1038/s41467-024-55550-x
