Detalhe da pesquisa
1.
A blood-brain penetrant RNA-targeted small molecule triggers elimination of r(G4C2)exp in c9ALS/FTD via the nuclear RNA exosome.
Proc Natl Acad Sci U S A
; 119(48): e2210532119, 2022 11 29.
Artigo
em Inglês
| MEDLINE | ID: mdl-36409902
2.
Study of an RNA-Focused DNA-Encoded Library Informs Design of a Degrader of a r(CUG) Repeat Expansion.
J Am Chem Soc
; 144(48): 21972-21979, 2022 12 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-36399603
3.
Transcriptome-Wide Studies of RNA-Targeted Small Molecules Provide a Simple and Selective r(CUG)exp Degrader in Myotonic Dystrophy.
ACS Cent Sci
; 9(7): 1342-1353, 2023 Jul 26.
Artigo
em Inglês
| MEDLINE | ID: mdl-37521782
4.
Systematically Studying the Effect of Small Molecules Interacting with RNA in Cellular and Preclinical Models.
ACS Chem Biol
; 16(7): 1111-1127, 2021 07 16.
Artigo
em Inglês
| MEDLINE | ID: mdl-34166593
5.
A Small Molecule Exploits Hidden Structural Features within the RNA Repeat Expansion That Causes c9ALS/FTD and Rescues Pathological Hallmarks.
ACS Chem Neurosci
; 12(21): 4076-4089, 2021 11 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-34677935
6.
Ribonuclease recruitment using a small molecule reduced c9ALS/FTD r(G4C2) repeat expansion in vitro and in vivo ALS models.
Sci Transl Med
; 13(617): eabd5991, 2021 Oct 27.
Artigo
em Inglês
| MEDLINE | ID: mdl-34705518
7.
Structural Features of Small Molecules Targeting the RNA Repeat Expansion That Causes Genetically Defined ALS/FTD.
ACS Chem Biol
; 15(12): 3112-3123, 2020 12 18.
Artigo
em Inglês
| MEDLINE | ID: mdl-33196168