TDP-43 pathology: from noxious assembly to therapeutic removal

SS Keating, R San Gil, MEV Swanson, EL Scotter… - Progress in …, 2022 - Elsevier
Our understanding of amyotrophic lateral sclerosis and frontotemporal dementia has
advanced dramatically since the discovery of cytoplasmic TAR DNA-binding protein 43 (TDP …

Connecting TDP-43 pathology with neuropathy

JR Klim, G Pintacuda, LA Nash, IG San Juan… - Trends in …, 2021 - cell.com
Transactive response DNA-binding protein 43 kDa (TDP-43), a multifunctional nucleic acid-
binding protein, is a primary component of insoluble aggregates associated with several …

Nanomedicine based strategies for oligonucleotide traversion across the blood–brain barrier

E Amulya, A Sikder, G Vambhurkar, S Shah… - Journal of Controlled …, 2023 - Elsevier
Neurological disorders are considered the most prominent cause of disability worldwide.
The major hurdle in the management of neurological disorders is the existence of the blood …

From basic research to the clinic: innovative therapies for ALS and FTD in the pipeline

RM Liscic, A Alberici, NJ Cairns, M Romano… - Molecular …, 2020 - Springer
Amyotrophic lateral sclerosis (ALS) and Frontotemporal Degeneration (FTD) are
neurodegenerative disorders, related by deterioration of motor and cognitive functions and …

Delivery of genome editing tools: A promising strategy for HPV-related cervical malignancy therapy

S Aghamiri, S Talaei, S Roshanzamiri… - Expert opinion on …, 2020 - Taylor & Francis
Introduction Persistent high-risk human papillomavirus infection is the main cause of various
types of cancer especially cervical cancer. The E6 and E7 oncoproteins of HPV play critical …

General and specific cytotoxicity of chimeric antisense oligonucleotides in bacterial cells and human cell lines

KB Popova, R Penchovsky - Antibiotics, 2024 - mdpi.com
In the last two decades, antisense oligonucleotide technology has emerged as a promising
approach to tackling various healthcare issues and diseases, such as antimicrobial …

A small molecule exploits hidden structural features within the RNA repeat expansion that causes c9ALS/FTD and rescues pathological hallmarks

A Ursu, JT Baisden, JA Bush, A Taghavi… - ACS chemical …, 2021 - ACS Publications
The hexanucleotide repeat expansion GGGGCC [r (G4C2) exp] within intron 1 of C9orf72
causes genetically defined amyotrophic lateral sclerosis and frontotemporal dementia …

Pervasive role of the long noncoding RNA DNM3OS in development and diseases

S Fellah, R Larrue, M Truchi, G Vassaux… - Wiley …, 2023 - Wiley Online Library
Thousands of unique noncoding RNAs (ncRNAs) are expressed in human cells, some are
tissue or cell type specific whereas others are considered as house‐keeping molecules …

Transcriptomics and RNA-based therapeutics as potential approaches to manage SARS-CoV-2 infection

C Arriaga-Canon, L Contreras-Espinosa… - International Journal of …, 2022 - mdpi.com
SARS-CoV-2 is a coronavirus family member that appeared in China in December 2019 and
caused the disease called COVID-19, which was declared a pandemic in 2020 by the World …

Modern approaches in gene therapy of motor neuron diseases

M Zakharova - Medicinal Research Reviews, 2021 - Wiley Online Library
Motor neuron disorders are a group of neurodegenerative diseases characterized by muscle
weakness, loss of ambulation, respiratory insufficiency, leading to an early death. Spinal …