A biological classification of Huntington's disease: the Integrated Staging System

SJ Tabrizi, S Schobel, EC Gantman… - The Lancet …, 2022 - thelancet.com
The current research paradigm for Huntington's disease is based on participants with overt
clinical phenotypes and does not address its pathophysiology nor the biomarker changes …

Prevalence and incidence of Huntington's disease: an updated systematic review and meta‐analysis

A Medina, Y Mahjoub, L Shaver… - Movement …, 2022 - Wiley Online Library
The incidence and prevalence of Huntington's disease (HD) based on a systematic review
and meta‐analysis of 20 studies published from 1985 to 2010 was estimated at 0.38 per …

Emerging role of non‐coding RNA in health and disease

GK Bhatti, N Khullar, IS Sidhu, US Navik… - Metabolic brain …, 2021 - Springer
Human diseases have always been a significant turf of concern since the origin of mankind.
It is cardinal to know the cause, treatment, and cure for every disease condition. With the …

The powerful world of antisense oligonucleotides: From bench to bedside

AM Quemener, L Bachelot, A Forestier… - Wiley …, 2020 - Wiley Online Library
Antisense oligonucleotides (ASOs) represent a new and highly promising class of drugs for
personalized medicine. In the last decade, major chemical developments and improvements …

Metabolic control of cell death

DR Green, L Galluzzi, G Kroemer - Science, 2014 - science.org
Background For several decades, intermediate metabolism and signal transduction have
been considered two independent entities. On one side stood the catabolic and anabolic …

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 …

Sex differences in movement disorders

S Meoni, A Macerollo, E Moro - Nature Reviews Neurology, 2020 - nature.com
In a range of neurological conditions, including movement disorders, sex-related differences
are emerging not only in brain anatomy and function, but also in pathogenesis, clinical …

Permanent inactivation of Huntington's disease mutation by personalized allele-specific CRISPR/Cas9

JW Shin, KH Kim, MJ Chao, RS Atwal… - Human molecular …, 2016 - academic.oup.com
A comprehensive genetics-based precision medicine strategy to selectively and
permanently inactivate only mutant, not normal allele, could benefit many dominantly …

Abundant contribution of short tandem repeats to gene expression variation in humans

M Gymrek, T Willems, A Guilmatre, H Zeng, B Markus… - Nature …, 2016 - nature.com
The contribution of repetitive elements to quantitative human traits is largely unknown. Here
we report a genome-wide survey of the contribution of short tandem repeats (STRs), which …

Huntington disease

GP Bates, R Dorsey, JF Gusella, MR Hayden… - Nature reviews Disease …, 2015 - nature.com
Huntington disease is devastating to patients and their families—with autosomal dominant
inheritance, onset typically in the prime of adult life, progressive course, and a combination …