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Home » Keywords » ASOs

Items Tagged with 'ASOs'

ARTICLES

Illustration of DNA, magnifying glass
Neurology/psychiatric

Quiver Bioscience collaborates with Dup15q Alliance to advance ASOs for Dup15q syndrome

March 20, 2025
Quiver Bioscience Inc. is collaborating with the Dup15q Alliance to advance an antisense oligonucleotide (ASO) therapeutic program for chromosome 15q duplication (Dup15q) syndrome.
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DNA double helix illustration with section being removed in red
Dermatologic

Genetic approaches presented at ESGCT provide hope for genodermatoses

Oct. 28, 2024
By Mar de Miguel
Some rare skin diseases not only reduce the quality of life of patients, but also can be devastating conditions, leading to amputations or death. At the 31st annual congress of the European Society of Gene and Cell Therapy (ESGCT), held last week in Rome, different laboratories showcased their approaches to editing mutations related to this group of diseases.
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3D illustration of a ribosome constructing messenger RNA molecules
Drug Design, Drug Delivery & Technologies

Even personalized drugs could benefit multiple patients

July 18, 2023
By Mar de Miguel
Using whole genome sequencing, scientists at Boston Children’s Hospital have studied the genes and mutations of ataxia-telangiectasia (A-T) that would respond to treatments with splice-switching antisense oligonucleotides (ASOs). Their work, published on July 12, 2023, in Nature, determined the appropriate individualized genetic therapy for these patients and identified a new drug.
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Hands holding test tubes at laptop
Drug Design, Drug Delivery & Technologies

Vanda and Olipass collaborate to develop antisense oligonucleotide therapeutics

Sep. 30, 2022
Vanda Pharmaceuticals Inc. and Olipass Corp. have entered into a research and development collaboration agreement to jointly develop a set of antisense oligonucleotide (ASO) molecules based on Olipass' proprietary modified peptide nucleic acids.
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RNA

Small molecule targets same repeat expansion in distinct diseases

Nov. 11, 2020
By Subhasree Nag
"RNA was long thought to be an 'undruggable' target for small molecules, because most cellular RNAs have extensive secondary structure, but only limited tertiary structure," Matthew Disney told BioWorld Science.
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Dopaminergic neurons

ISSCR 2020: In-brain direct reprogramming creates neurons, alleviates Parkinson’s

June 30, 2020
By Anette Breindl
Two separate groups have recently shown that in mouse models, inactivation of a single gene was enough to directly convert other cell types in the brain into neurons.
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