Literature DB >> 35075291

New antisense oligonucleotide therapies reach first base in ALS.

Elia R Lopez1, William F Borschel1, Bryan J Traynor2,3.   

Abstract

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Year:  2022        PMID： 35075291     DOI： 10.1038/s41591-021-01629-7

Source DB:  PubMed          Journal:  Nat Med        ISSN： 1078-8956            Impact factor:   53.440


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  2 in total

1.  Cholesterol-functionalized DNA/RNA heteroduplexes cross the blood-brain barrier and knock down genes in the rodent CNS.

Authors:  Tetsuya Nagata; Chrissa A Dwyer; Kie Yoshida-Tanaka; Kensuke Ihara; Masaki Ohyagi; Hidetoshi Kaburagi; Haruka Miyata; Satoe Ebihara; Kotaro Yoshioka; Takashi Ishii; Kanjiro Miyata; Kenichi Miyata; Berit Powers; Tomoko Igari; Syunsuke Yamamoto; Naoto Arimura; Hideki Hirabayashi; Toshiki Uchihara; Rintaro Iwata Hara; Takeshi Wada; C Frank Bennett; Punit P Seth; Frank Rigo; Takanori Yokota
Journal:  Nat Biotechnol       Date:  2021-08-12       Impact factor: 68.164

2.  Suppression of mutant C9orf72 expression by a potent mixed backbone antisense oligonucleotide.

Authors:  Hélène Tran; Michael P Moazami; Huiya Yang; Diane McKenna-Yasek; Catherine L Douthwright; Courtney Pinto; Jake Metterville; Minwook Shin; Nitasha Sanil; Craig Dooley; Ajit Puri; Alexandra Weiss; Nicholas Wightman; Heather Gray-Edwards; Miklos Marosfoi; Robert M King; Thomas Kenderdine; Daniele Fabris; Robert Bowser; Jonathan K Watts; Robert H Brown
Journal:  Nat Med       Date:  2021-12-23       Impact factor: 87.241
  2 in total

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