New avenues for the treatment of Huntington's disease

A Kim, K Lalonde, A Truesdell, P Gomes Welter… - International journal of …, 2021 - mdpi.com
Huntington's disease (HD) is a neurodegenerative disorder caused by a CAG expansion in
the HD gene. The disease is characterized by neurodegeneration, particularly in the striatum …

Current and possible future therapeutic options for Huntington's disease

MW Ferguson, CJ Kennedy… - Journal of central …, 2022 - journals.sagepub.com
Huntington's disease (HD) is an autosomal neurodegenerative disease that is characterized
by an excessive number of CAG trinucleotide repeats within the huntingtin gene (HTT). HD …

Antisense oligonucleotide therapy: from design to the Huntington disease clinic

ME Rook, AL Southwell - BioDrugs, 2022 - Springer
Huntington disease (HD) is a fatal progressive neurodegenerative disorder caused by an
inherited mutation in the huntingtin (HTT) gene, which encodes mutant HTT protein. Though …

Small molecule splicing modifiers with systemic HTT-lowering activity

A Bhattacharyya, CR Trotta, J Narasimhan… - Nature …, 2021 - nature.com
Huntington's disease (HD) is a hereditary neurodegenerative disorder caused by expansion
of cytosine-adenine-guanine (CAG) trinucleotide repeats in the huntingtin (HTT) gene …

MicroRNA alteration, application as biomarkers, and therapeutic approaches in neurodegenerative diseases

TPN Nguyen, M Kumar, E Fedele, G Bonanno… - International journal of …, 2022 - mdpi.com
MicroRNAs (miRNAs) are essential post-transcriptional gene regulators involved in various
neuronal and non-neuronal cell functions and play a key role in pathological conditions …

Current status of microRNA-based therapeutic approaches in neurodegenerative disorders

S Paul, LA Bravo Vázquez, S Pérez Uribe… - Cells, 2020 - mdpi.com
MicroRNAs (miRNAs) are a key gene regulator and play essential roles in several biological
and pathological mechanisms in the human system. In recent years, plenty of miRNAs have …

Molecular strategies to target protein aggregation in Huntington's disease

OD Jarosińska, SGD Rüdiger - Frontiers in molecular biosciences, 2021 - frontiersin.org
Huntington's disease (HD) is a neurodegenerative disorder caused by the aggregation of
the mutant huntingtin (mHTT) protein in nerve cells. mHTT self-aggregates to form soluble …

Impact of guanidine-containing backbone linkages on stereopure antisense oligonucleotides in the CNS

P Kandasamy, Y Liu, V Aduda, S Akare… - Nucleic acids …, 2022 - academic.oup.com
Attaining sufficient tissue exposure at the site of action to achieve the desired
pharmacodynamic effect on a target is an important determinant for any drug discovery …

Interrupting sequence variants and age of onset in Huntington's disease: clinical implications and emerging therapies

GEB Wright, HF Black, JA Collins… - The Lancet …, 2020 - thelancet.com
Background Huntington's disease is a fatal neurodegenerative disorder that is caused by
CAG-CAA repeat expansion, encoding polyglutamine, in the huntingtin (HTT) gene. Current …

Toward the development of epigenome editing-based therapeutics: potentials and challenges

J Ueda, T Yamazaki, H Funakoshi - International Journal of Molecular …, 2023 - mdpi.com
The advancement in epigenetics research over the past several decades has led to the
potential application of epigenome-editing technologies for the treatment of various …