A blood-brain penetrant RNA-targeted small molecule triggers elimination of r(G 4 C 2 ) ^exp in c9ALS/FTD via the nuclear RNA exosome.

Autor: Bush JA; Department of Chemistry, The Scripps Research Institute and UF Scripps Biomedical Research, University of Florida, Jupiter, FL 33458., Meyer SM; Department of Chemistry, The Scripps Research Institute and UF Scripps Biomedical Research, University of Florida, Jupiter, FL 33458., Fuerst R; Department of Chemistry, The Scripps Research Institute and UF Scripps Biomedical Research, University of Florida, Jupiter, FL 33458., Tong Y; Department of Chemistry, The Scripps Research Institute and UF Scripps Biomedical Research, University of Florida, Jupiter, FL 33458., Li Y; Department of Chemistry, The Scripps Research Institute and UF Scripps Biomedical Research, University of Florida, Jupiter, FL 33458., Benhamou RI; Department of Chemistry, The Scripps Research Institute and UF Scripps Biomedical Research, University of Florida, Jupiter, FL 33458., Aikawa H; Department of Chemistry, The Scripps Research Institute and UF Scripps Biomedical Research, University of Florida, Jupiter, FL 33458., Zanon PRA; Department of Chemistry, The Scripps Research Institute and UF Scripps Biomedical Research, University of Florida, Jupiter, FL 33458., Gibaut QMR; Department of Chemistry, The Scripps Research Institute and UF Scripps Biomedical Research, University of Florida, Jupiter, FL 33458., Angelbello AJ; Department of Chemistry, The Scripps Research Institute and UF Scripps Biomedical Research, University of Florida, Jupiter, FL 33458., Gendron TF; Department of Neuroscience, Mayo Clinic, Jacksonville, FL 32224., Zhang YJ; Department of Neuroscience, Mayo Clinic, Jacksonville, FL 32224., Petrucelli L; Department of Neuroscience, Mayo Clinic, Jacksonville, FL 32224., Heick Jensen T; Department of Molecular Biology and Genetics, Aarhus University, Aarhus C DK-8000, Denmark., Childs-Disney JL; Department of Chemistry, The Scripps Research Institute and UF Scripps Biomedical Research, University of Florida, Jupiter, FL 33458., Disney MD; Department of Chemistry, The Scripps Research Institute and UF Scripps Biomedical Research, University of Florida, Jupiter, FL 33458.; Department of Neuroscience, The Scripps Research Institute and UF Scripps Biomedical Research, University of Florida, Jupiter, FL 33458.

Publikováno v: Proceedings of the National Academy of Sciences of the United States of America [Proc Natl Acad Sci U S A] 2022 Nov 29; Vol. 119 (48), pp. e2210532119. Date of Electronic Publication: 2022 Nov 21.

Massively Parallel Optimization of the Linker Domain in Small Molecule Dimers Targeting a Toxic r(CUG) Repeat Expansion.

Autor: Vezina-Dawod S; Department of Chemistry, The Scripps Research Institute, 130 Scripps Way, Jupiter, Florida 33458, United States., Angelbello AJ; Department of Chemistry, The Scripps Research Institute, 130 Scripps Way, Jupiter, Florida 33458, United States., Choudhary S; Department of Chemistry, The Scripps Research Institute, 130 Scripps Way, Jupiter, Florida 33458, United States., Wang KW; Department of Chemistry, Florida Atlantic University, Jupiter, Florida 33458, United States., Yildirim I; Department of Chemistry, Florida Atlantic University, Jupiter, Florida 33458, United States., Disney MD; Department of Chemistry, The Scripps Research Institute, 130 Scripps Way, Jupiter, Florida 33458, United States.

Publikováno v: ACS medicinal chemistry letters [ACS Med Chem Lett] 2021 Mar 02; Vol. 12 (6), pp. 907-914. Date of Electronic Publication: 2021 Mar 02 (Print Publication: 2021).

Design of small molecules targeting RNA structure from sequence.

Autor: Ursu A; Department of Chemistry, The Scripps Research Institute, 130 Scripps Way, Jupiter, FL 33458, USA. disney@scripps.edu., Childs-Disney JL; Department of Chemistry, The Scripps Research Institute, 130 Scripps Way, Jupiter, FL 33458, USA. disney@scripps.edu., Andrews RJ; Roy J. Carver Department of Biochemistry, Biophysics & Molecular Biology, Iowa State University, Ames, Iowa, USA. wmoss@iastate.edu., O'Leary CA; Roy J. Carver Department of Biochemistry, Biophysics & Molecular Biology, Iowa State University, Ames, Iowa, USA. wmoss@iastate.edu., Meyer SM; Department of Chemistry, The Scripps Research Institute, 130 Scripps Way, Jupiter, FL 33458, USA. disney@scripps.edu., Angelbello AJ; Department of Chemistry, The Scripps Research Institute, 130 Scripps Way, Jupiter, FL 33458, USA. disney@scripps.edu., Moss WN; Roy J. Carver Department of Biochemistry, Biophysics & Molecular Biology, Iowa State University, Ames, Iowa, USA. wmoss@iastate.edu., Disney MD; Department of Chemistry, The Scripps Research Institute, 130 Scripps Way, Jupiter, FL 33458, USA. disney@scripps.edu.

Publikováno v: Chemical Society reviews [Chem Soc Rev] 2020 Oct 19; Vol. 49 (20), pp. 7252-7270.

Optimization of the Linker Domain in a Dimeric Compound that Degrades an r(CUG) Repeat Expansion in Cells.

Autor: Benhamou RI; Department of Chemistry, The Scripps Research Institute, 130 Scripps Way, Jupiter, Florida 33458, United States., Abe M; Department of Chemistry, The Scripps Research Institute, 130 Scripps Way, Jupiter, Florida 33458, United States., Choudhary S; Department of Chemistry, The Scripps Research Institute, 130 Scripps Way, Jupiter, Florida 33458, United States., Meyer SM; Department of Chemistry, The Scripps Research Institute, 130 Scripps Way, Jupiter, Florida 33458, United States., Angelbello AJ; Department of Chemistry, The Scripps Research Institute, 130 Scripps Way, Jupiter, Florida 33458, United States., Disney MD; Department of Chemistry, The Scripps Research Institute, 130 Scripps Way, Jupiter, Florida 33458, United States.

Publikováno v: Journal of medicinal chemistry [J Med Chem] 2020 Jul 23; Vol. 63 (14), pp. 7827-7839. Date of Electronic Publication: 2020 Jul 13.

Small molecule targeting of RNA structures in neurological disorders.

Autor: Angelbello AJ; Department of Chemistry, The Scripps Research Institute, Jupiter, Florida., Chen JL; Department of Chemistry, The Scripps Research Institute, Jupiter, Florida., Disney MD; Department of Chemistry, The Scripps Research Institute, Jupiter, Florida.

Publikováno v: Annals of the New York Academy of Sciences [Ann N Y Acad Sci] 2020 Jul; Vol. 1471 (1), pp. 57-71. Date of Electronic Publication: 2019 Apr 09.