Authors

Sage Berry

Senior Project Advisor

Jeff Carroll

Document Type

Project

Publication Date

Spring 2023

Keywords

Huntington's disease, Antisense oligonucleotides, Neurodegenerative, Allele-selective

Abstract

Huntington's disease (HD) is a uniformly fatal, neurodegenerative disease originating from a mutation in one allele of the HTT gene. While global HTT reduction has shown some improvement in treating the progression of HD, the wild type HTT allele has necessary functions for survival and cannot be ablated. Antisense oligonucleotides (ASOs) are a way to target pre-MRNA and prevent the mRNA's translation into a protein. In this study, we examined the efficacy of allele selective ASOs in comparison to pan ASOs in ameliorating HD pathology and symptomology.

Department

Behavioral Neuroscience

Subjects - Topical (LCSH)

Huntington's disease; Nervous system--Degeneration; Oligonucleotides

Type

Text

Rights

Copying of this document in whole or in part is allowable only for scholarly purposes. It is understood, however, that any copying or publication of this document for commercial purposes, or for financial gain, shall not be allowed without the author’s written permission.

Language

English

Format

application/pdf

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