[HTML][HTML] Advances in stem cell and other therapies for Huntington's disease: An update

LT Conner, B Srinageshwar, JL Bakke, GL Dunbar… - Brain Research …, 2023 - Elsevier
Huntington's disease (HD) is a neurodegenerative disorder caused by an autosomal
dominant mutation leading to an abnormal CAG repeat expansion. The result is the …

[HTML][HTML] Induced pluripotent stem cell-derived and directly reprogrammed neurons to study neurodegenerative diseases: The impact of aging signatures

S Aversano, C Caiazza, M Caiazzo - Frontiers in Aging Neuroscience, 2022 - frontiersin.org
Several diseases of the central nervous system are age-associated and do not directly result
from genetic mutations. These include late-onset neurodegenerative diseases (NDDs) …

[HTML][HTML] Efficient correction of ABCA4 variants by CRISPR-Cas9 in hiPSCs derived from Stargardt disease patients

L Siles, S Ruiz-Nogales, A Navinés-Ferrer… - … Therapy-Nucleic Acids, 2023 - cell.com
Inherited retinal dystrophies comprise a broad group of genetic eye diseases without
effective treatment. Among them, Stargardt disease is the second most prevalent pathology …

[HTML][HTML] Development of CRISPR Cas9, spin-off technologies and their application in model construction and potential therapeutic methods of Parkinson's disease

J Qu, N Liu, L Gao, J Hu, M Sun, D Yu - Frontiers in Neuroscience, 2023 - frontiersin.org
Parkinson's disease (PD) is one of the most common degenerative diseases. It is most
typically characterized by neuronal death following the accumulation of Lewis inclusions in …

[HTML][HTML] The Multifaceted role of WNT signaling in Alzheimer's disease onset and age-related progression

WW Kostes, DA Brafman - Cells, 2023 - mdpi.com
The evolutionary conserved WNT signaling pathway orchestrates numerous complex
biological processes during development and is critical to the maintenance of tissue integrity …

[HTML][HTML] CRISPR/Cas9: implication for modeling and therapy of amyotrophic lateral sclerosis

Y Shi, Y Zhao, L Lu, Q Gao, D Yu, M Sun - Frontiers in Neuroscience, 2023 - frontiersin.org
Amyotrophic lateral sclerosis (ALS) is a deadly neurological disease with a complicated and
variable pathophysiology yet to be fully understood. There is currently no effective treatment …

[HTML][HTML] Thalamic alterations in motor neuron diseases: a systematic review of MRI findings

S Mohammadi, S Ghaderi, M Mohammadi… - Journal of Integrative …, 2024 - imrpress.com
Background: Motor neuron diseases (MNDs) are progressive neurodegenerative disorders
characterized by motor impairment and non-motor symptoms. The involvement of the …

[HTML][HTML] CRISPRi: a way to integrate iPSC-derived neuronal models

SNJ Franks, R Heon-Roberts… - Biochemical Society …, 2024 - portlandpress.com
The genetic landscape of neurodegenerative diseases encompasses genes affecting
multiple cellular pathways which exert effects in an array of neuronal and glial cell-types …

Research progress of PROTACs for neurodegenerative diseases therapy

Z Cai, Z Yang, H Li, Y Fang - Bioorganic Chemistry, 2024 - Elsevier
Neurodegenerative diseases (NDD) are characterized by the gradual deterioration of
neuronal function and integrity, resulting in an overall decline in brain function. The existing …

Induced pluripotent stem cell models as a tool to investigate and test fluid biomarkers in Alzheimer's disease and frontotemporal dementia

JJ McInvale, P Canoll, G Hargus - Brain Pathology, 2024 - Wiley Online Library
Neurodegenerative diseases are increasing in prevalence and comprise a large
socioeconomic burden on patients and their caretakers. The need for effective therapies and …