[HTML][HTML] Advances in stem cell and other therapies for Huntington's disease: An update
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 …
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) …
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 …
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 …
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 …
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 …
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 …
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 …
multiple cellular pathways which exert effects in an array of neuronal and glial cell-types …
Research progress of PROTACs for neurodegenerative diseases therapy
Neurodegenerative diseases (NDD) are characterized by the gradual deterioration of
neuronal function and integrity, resulting in an overall decline in brain function. The existing …
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 …
socioeconomic burden on patients and their caretakers. The need for effective therapies and …