Výsledky vyhledávání - "Alicia J. Angelbello"

A blood-brain penetrant RNA-targeted small molecule triggers elimination of r(G4C2)exp in c9ALS/FTD via the nuclear RNA exosome

Autor: Jessica A. Bush, Samantha M. Meyer, Rita Fuerst, Yuquan Tong, Yue Li, Raphael I. Benhamou, Haruo Aikawa, Patrick R. A. Zanon, Quentin M. R. Gibaut, Alicia J. Angelbello, Tania F. Gendron, Yong-Jie Zhang, Leonard Petrucelli, Torben Heick Jensen, Jessica L. Childs-Disney, Matthew D. Disney

Publikováno v: Bush, J A, Meyer, S M, Fuerst, R, Tong, Y, Li, Y, Benhamou, R I, Aikawa, H, Zanon, P R A, Gibaut, Q M R, Angelbello, A J, Gendron, T F, Zhang, Y J, Petrucelli, L, Heick Jensen, T, Childs-Disney, J L & Disney, M D 2022, ' 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 ', Proceedings of the National Academy of Sciences of the United States of America, vol. 119, no. 48, e2210532119 . https://doi.org/10.1073/pnas.2210532119

A hexanucleotide repeat expansion in intron 1 of the C9orf72 gene is the most common genetic cause of amyotrophic lateral sclerosis and frontotemporal dementia, or c9ALS/FTD. The RNA transcribed from the expansion, r(G 4 C 2 ) exp , causes various pa

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::d99afbe6d3582820279ae53936a1dca4
https://pure.au.dk/portal/da/publications/a-bloodbrain-penetrant-rnatargeted-small-molecule-triggers-elimination-of-rg4c2exp-in-c9alsftd-via-the-nuclear-rna-exosome(c64b1cee-2f4b-4d36-8329-562cc33c1c4c).html

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A Toxic RNA Templates the Synthesis of Its Own Fluorogenic Inhibitor by Using a Bio-orthogonal Tetrazine Ligation in Cells and Tissues

Autor: Matthew D. Disney, Alicia J. Angelbello

Publikováno v: ACS Chem Biol

Expanded RNA repeats cause >30 incurable diseases. One approach to mitigate their toxicity is by using small molecules that assemble into potent, oligomeric species upon binding to the disease-causing RNA in cells. Herein, we show that the expanded r

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::5522b8eef31c5228843bd1c230a9af86
https://doi.org/10.1021/acschembio.0c00417

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Precise Targeted Cleavage of a r(CUG) Repeat Expansion in Cells by Using a Small-Molecule–Deglycobleomycin Conjugate

Autor: Christopher M. Glinkerman, Matthew D. Disney, Mary E. DeFeo, Dale L. Boger, Alicia J. Angelbello

Publikováno v: ACS Chem Biol

RNA repeat expansions cause more than 30 neurological and neuromuscular diseases with no known cures. Since repeat expansions operate via diverse pathomechanisms, one potential therapeutic strategy is to rid them from disease-affected cells, using bi

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::eeff6862276631d91c1466508d9682bb
https://doi.org/10.1021/acschembio.0c00036

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Design of small molecules targeting RNA structure from sequence

Autor: Samantha M. Meyer, Collin A. O’Leary, Jessica L. Childs-Disney, Andrei Ursu, Alicia J. Angelbello, Walter N. Moss, Matthew D. Disney, Ryan J. Andrews

Publikováno v: Chem Soc Rev

The design and discovery of small molecule medicines has largely been focused on a small number of druggable protein families. A new paradigm is emerging, however, in which small molecules exert a biological effect by interacting with RNA, both to st

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::3f3a566f2e73b2c6a3f73383bd4b0fa5
https://doi.org/10.1039/d0cs00455c

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A Druglike Small Molecule that Targets r(CCUG) Repeats in Myotonic Dystrophy Type 2 Facilitates Degradation by RNA Quality Control Pathways

Autor: Jessica L. Childs-Disney, Alicia J. Angelbello, Matthew D. Disney, Sarah Wagner-Griffin, Raphael I. Benhamou, Jonathan L. Chen, Kamalakannan Vishnu, Masahito Abe

Publikováno v: J Med Chem

Myotonic dystrophy type 2 (DM2) is one of >40 microsatellite disorders caused by RNA repeat expansions. The DM2 repeat expansion, r(CCUG)(exp) (where “exp” denotes expanded repeating nucleotides), is harbored in intron 1 of the CCHC-type zinc fin

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::aed25d53022adad4fbd3528dffe48185
https://pubmed.ncbi.nlm.nih.gov/34101465

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Small molecule targeting of RNA structures in neurological disorders

Autor: Matthew D. Disney, Alicia J. Angelbello, Jonathan L. Chen

Publikováno v: Ann N Y Acad Sci

Aberrant RNA structure and function operate in neurological disease progression and severity. As RNA contributes to disease pathology in a complex fashion, that is, via various mechanisms, it has become an attractive therapeutic target for small mole

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::a6535c8599d827c5a71546f27f75485a
https://doi.org/10.1111/nyas.14051

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

Autor: Shruti Choudhary, Ilyas Yildirim, Matthew D. Disney, Kye Won Wang, Simon Vezina-Dawod, Alicia J. Angelbello

Publikováno v: ACS Med Chem Lett

[Image: see text] RNA contributes to disease pathobiology and is an important therapeutic target. The downstream biology of disease-causing RNAs can be short-circuited with small molecules that recognize structured regions. The discovery and optimiza

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::7d67eb2034d02c612aaf7aacab1d5352
https://pubmed.ncbi.nlm.nih.gov/34141068

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Gini Coefficients as a Single Value Metric to Define Chemical Probe Selectivity

Autor: Samantha M. Meyer, Andrei Ursu, Jessica L. Childs-Disney, Alicia J. Angelbello, Matthew G. Costales, Matthew D. Disney

Publikováno v: ACS Chem Biol

Selectivity is a key requirement of high-quality chemical probes and lead medicines; however, methods to quantify and compare the selectivity of small molecules have not been standardized across the field. Herein, we discuss the origins and use of a

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::f82feba561082da86373ec916b032584
https://pubmed.ncbi.nlm.nih.gov/32568503

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A small molecule that binds an RNA repeat expansion stimulates its decay via the exosome complex

Autor: Alicia J. Angelbello, Albert S. Jun, Zhen Zhi Tang, Madhuparna Roy, Shruti Choudhary, Matthew D. Disney, Kye Won Wang, Suzanne G. Rzuczek, Raphael I. Benhamou, Ilyas Yildirim, Charles A. Thornton, Jonathan L. Chen

We describe the design of a small molecule that binds the structure of a r(CUG) repeat expansion [r(CUG)exp] and reverses molecular defects in two diseases mediated by the RNA - myotonic dystrophy type 1 (DM1) and Fuchs endothelial corneal dystrophy

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::9eb6b43264c553b3bd8ddbd47b62d7f2
https://doi.org/10.1101/2020.05.11.088427

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