Splice-modulating antisense oligonucleotides as therapeutics for inherited metabolic diseases

S Chen, SN Heendeniya, BT Le, K Rahimizadeh… - BioDrugs, 2024 - Springer
Abstract The last decade (2013–2023) has seen unprecedented successes in the clinical
translation of therapeutic antisense oligonucleotides (ASOs). Eight such molecules have …

Autophagy‐associated non‐coding RNAs: Unraveling their impact on Parkinson's disease pathogenesis

MS Hussain, E Moglad, M Afzal… - CNS Neuroscience …, 2024 - Wiley Online Library
Background Parkinson's disease (PD) is a degenerative neurological condition marked by
the gradual loss of dopaminergic neurons in the substantia nigra pars compacta. The …

Potential Implications of miRNAs in the Pathogenesis, Diagnosis, and Therapeutics of Alzheimer's Disease

L Wang, X Shui, Y Diao, D Chen, Y Zhou… - International Journal of …, 2023 - mdpi.com
Alzheimer's disease (AD) is a complex multifactorial disorder that poses a substantial
burden on patients, caregivers, and society. Considering the increased aging population …

Alzheimer's disease: Review of current nanotechnological therapeutic strategies

E Baranowska-Wójcik, D Szwajgier - Expert review of …, 2020 - Taylor & Francis
ABSTRACT Introduction: Alzheimer's Disease (AD) is a progressive neurodegenerative
pathology characterized by the presence of neuritic plaques and neurofibrillary tangles. The …

Neurodegenerative diseases: a hotbed for splicing defects and the potential therapies

D Li, CS McIntosh, FL Mastaglia, SD Wilton… - Translational …, 2021 - Springer
Precursor messenger RNA (pre-mRNA) splicing is a fundamental step in eukaryotic gene
expression that systematically removes non-coding regions (introns) and ligates coding …

Delivery systems for theranostics in neurodegenerative diseases

Y Li, R Liu, W Ji, Y Li, L Liu, X Zhang - Nano Research, 2018 - Springer
Neurodegenerative diseases are characterized by progressive nervous system dysfunction,
which affects over 90,000 people every year. Although numerous contrast agents and …

Targeting amyloid-β precursor protein, APP, splicing with antisense oligonucleotides reduces toxic amyloid-β production

JL Chang, AJ Hinrich, B Roman, M Norrbom, F Rigo… - Molecular Therapy, 2018 - cell.com
Alterations in amyloid beta precursor protein (APP) have been implicated in cognitive
decline in Alzheimer's disease (AD), which is accelerated in Down syndrome/Trisomy 21 …

New RNA-based breakthroughs in Alzheimer's disease diagnosis and therapeutics

M Riscado, B Baptista, F Sousa - Pharmaceutics, 2021 - mdpi.com
Dementia is described as the fifth leading cause of death worldwide and Alzheimer's
disease (AD) is recognized as the most common, causing a huge impact on health costs and …

Magnetic nanoparticles associated PEG/PLGA block copolymer targeted with anti-transferrin receptor antibodies for Alzheimer's disease

N Cui, H Lu, M Li - Journal of Biomedical Nanotechnology, 2018 - ingentaconnect.com
A facile brain drug delivery system for Alzheimer's disease, the Magnetic iron oxide (Fe3O4)
nanoparticles loaded with polyethylene glycol-polylactide-polyglycolide co-polymer …

Alzheimer's Disease: Status of Low‐Dimensional Nanotherapeutic Materials

D Huang, W Liao, J Li, T Chen, X Wang… - Advanced Functional …, 2024 - Wiley Online Library
Alzheimer's disease (AD) is a chronic progressive neurodegenerative disease that
constitutes the third most common cause of death among elderly individuals. AD patients …