Single-cell dissection of the human brain vasculature

FJ Garcia, N Sun, H Lee, B Godlewski, H Mathys… - Nature, 2022 - nature.com
Despite the importance of the cerebrovasculature in maintaining normal brain physiology
and in understanding neurodegeneration and drug delivery to the central nervous system …

The potential of gene editing for Huntington's disease

W Duan, E Urani, MP Mattson - Trends in neurosciences, 2023 - cell.com
Huntington's disease (HD) is a dominantly inherited neurodegenerative disorder caused by
a trinucleotide repeat expansion in the huntingtin gene resulting in long stretches of …

From recognition to remedy: The significance of biomarkers in neurodegenerative disease pathology

C Toader, N Dobrin, FM Brehar, C Popa… - International journal of …, 2023 - mdpi.com
With the inexorable aging of the global populace, neurodegenerative diseases (NDs) like
Alzheimer's disease (AD), Parkinson's disease (PD), and amyotrophic lateral sclerosis (ALS) …

An RNA-targeting CRISPR–Cas13d system alleviates disease-related phenotypes in Huntington's disease models

KH Morelli, Q Wu, ML Gosztyla, H Liu, M Yao… - Nature …, 2023 - nature.com
Huntington's disease (HD) is a fatal, dominantly inherited neurodegenerative disorder
caused by CAG trinucleotide expansion in exon 1 of the huntingtin (HTT) gene. Since the …

[HTML][HTML] Non-contrast assessment of blood-brain barrier permeability to water in mice: An arterial spin labeling study at cerebral veins

Z Wei, H Liu, Z Lin, M Yao, R Li, C Liu, Y Li, J Xu… - Neuroimage, 2023 - Elsevier
Blood-brain barrier (BBB) plays a critical role in protecting the brain from toxins and
pathogens. However, in vivo tools to assess BBB permeability are scarce and often require …

[HTML][HTML] Allele-specific silencing of the gain-of-function mutation in Huntington's disease using CRISPR/Cas9

JW Shin, EP Hong, SS Park, DE Choi, IS Seong… - JCI insight, 2022 - ncbi.nlm.nih.gov
Dominant gain-of-function mechanisms in Huntington's disease (HD) suggest that selective
silencing of mutant HTT produces robust therapeutic benefits. Here, capitalizing on exonic …

Multiscale imaging informs translational mouse modeling of neurological disease

Y Wang, JM LeDue, TH Murphy - Neuron, 2022 - cell.com
Multiscale neurophysiology reveals that simple motor actions are associated with changes
in neuronal firing in virtually every brain region studied. Accordingly, the assessment of focal …

Huntington's disease: complex pathogenesis and therapeutic strategies

H Tong, T Yang, S Xu, X Li, L Liu, G Zhou… - International Journal of …, 2024 - mdpi.com
Huntington's disease (HD) arises from the abnormal expansion of CAG repeats in the
huntingtin gene (HTT), resulting in the production of the mutant huntingtin protein (mHTT) …

Water-reaching platform for longitudinal assessment of cortical activity and fine motor coordination defects in a Huntington disease mouse model

Y Wang, MD Sepers, D Xiao, LA Raymond, TH Murphy - eneuro, 2023 - eneuro.org
Huntington disease (HD), caused by dominantly inherited expansions of a CAG repeat
results in characteristic motor dysfunction. Although gross motor defects have been …

PAM-altering SNP-based allele-specific CRISPR-Cas9 therapeutic strategies for Huntington's disease

JW Shin, EP Hong, SS Park, DE Choi, S Zeng… - … Therapy-Methods & …, 2022 - cell.com
Huntington's disease (HD) is caused by an expanded CAG repeat in huntingtin (HTT). Since
HD is dominant and loss of HTT leads to neurological abnormalities, safe therapeutic …