Gene therapy for ultrarare diseases: a geneticist's perspective
WL Hwu - Journal of Biomedical Science, 2024 - Springer
Gene therapy has made considerable strides in recent years. More than 4000 protein-
coding genes have been implicated in more than 6000 genetic diseases; next-generation …
coding genes have been implicated in more than 6000 genetic diseases; next-generation …
Current Small Molecule–Based Medicinal Chemistry Approaches for Neurodegeneration Therapeutics
Abstract Neurodegenerative diseases (NDDs) like Alzheimer's disease (AD), Parkinson's
disease (PD), and Amyotrophic lateral sclerosis (ALS) possess multifactorial aetiologies. In …
disease (PD), and Amyotrophic lateral sclerosis (ALS) possess multifactorial aetiologies. In …
Application of antisense oligonucleotide drugs in amyotrophic lateral sclerosis and Huntington's disease
K Ou, Q Jia, D Li, S Li, XJ Li, P Yin - Translational Neurodegeneration, 2025 - Springer
Amyotrophic lateral sclerosis (ALS) and Huntington's disease (HD) are diverse in clinical
presentation and are caused by complex and multiple factors, including genetic mutations …
presentation and are caused by complex and multiple factors, including genetic mutations …
Elevated plasma and CSF neurofilament light chain concentrations are stabilized in response to mutant huntingtin lowering in the brains of Huntington's disease mice
Background Therapeutic approaches aimed at lowering toxic mutant huntingtin (mHTT)
levels in the brain can reverse disease phenotypes in animal models of Huntington's …
levels in the brain can reverse disease phenotypes in animal models of Huntington's …
Advancing CNS Therapeutics: Enhancing Neurological Disorders with Nanoparticle-Based Gene and Enzyme Replacement Therapies
S Liu, H Li, S **, Y Zhang, T Sun - International Journal of …, 2025 - Taylor & Francis
Given the complexity of the central nervous system (CNS) and the diversity of neurological
conditions, the increasing prevalence of neurological disorders poses a significant …
conditions, the increasing prevalence of neurological disorders poses a significant …
Plasma and CSF neurofilament light chain are stabilized in response to mutant huntingtin lowering in the brain of Huntington disease mice
Background Therapeutic approaches aimed at lowering levels of toxic mutant huntingtin
(mHTT) in the brain can reverse disease phenotypes in animal models of Huntington …
(mHTT) in the brain can reverse disease phenotypes in animal models of Huntington …