Antisense oligonucleotides: A primer

DR Scoles, EV Minikel, SM Pulst - Neurology: Genetics, 2019 - AAN Enterprises
There are few disease-modifying therapeutics for neurodegenerative diseases, but
successes on the development of antisense oligonucleotide (ASO) therapeutics for spinal …

RNA splicing and disease: animal models to therapies

M Montes, BL Sanford, DF Comiskey, DS Chandler - Trends in Genetics, 2019 - cell.com
Alternative splicing of pre-mRNA increases genetic diversity, and recent studies estimate
that most human multiexon genes are alternatively spliced. If this process is not highly …

[HTML][HTML] Disease-modifying therapies for Alzheimer's disease: more questions than answers

TE Golde - Neurotherapeutics, 2023 - Elsevier
Scientific advances over the last four decades have steadily infused the Alzheimer's disease
(AD) field with great optimism that therapies targeting Aβ, amyloid, tau, and innate immune …

Antisense oligonucleotides for Alzheimer's disease therapy: from the mRNA to miRNA paradigm

W Grabowska-Pyrzewicz, A Want, J Leszek… - EBioMedicine, 2021 - thelancet.com
Alzheimer's disease (AD) represents a particular therapeutic challenge because its
aetiology is very complex, with dynamic progression from preclinical to clinical stages …

RNA-based therapeutics for neurological diseases

K Anthony - RNA biology, 2022 - Taylor & Francis
ABSTRACT RNA-based therapeutics have entered the mainstream with seemingly limitless
possibilities to treat all categories of neurological disease. Here, common RNA-based drug …

Tip60's novel RNA-binding function modulates alternative splicing of pre-mRNA targets implicated in Alzheimer's disease

A Bhatnagar, K Krick, BC Karisetty… - Journal of …, 2023 - Soc Neuroscience
The severity of Alzheimer9s disease (AD) progression involves a complex interplay of
genetics, age, and environmental factors orchestrated by histone acetyltransferase (HAT) …

Gene-based therapies for neurodegenerative diseases

J Sun, S Roy - Nature neuroscience, 2021 - nature.com
Gene therapy is making a comeback. With its twin promise of targeting disease etiology and
'long-term correction', gene-based therapies (defined here as all forms of genome …

[HTML][HTML] Contribution of A-to-I RNA editing, M6A RNA Methylation, and Alternative Splicing to physiological brain aging and neurodegenerative diseases

V Tassinari, P La Rosa, E Guida, A Colopi… - Mechanisms of ageing …, 2023 - Elsevier
Aging is a physiological and progressive phenomenon in all organisms' life cycle,
characterized by the accumulation of degenerative processes triggered by several …

Towards personalized allele-specific antisense oligonucleotide therapies for toxic gain-of-function neurodegenerative diseases

J Helm, L Schöls, S Hauser - Pharmaceutics, 2022 - mdpi.com
Antisense oligonucleotides (ASOs) are single-stranded nucleic acid strings that can be used
to selectively modify protein synthesis by binding complementary (pre-) mRNA sequences …

Alternative splicing in Alzheimer's disease

G Biamonti, A Amato, E Belloni, A Di Matteo… - Aging clinical and …, 2021 - Springer
Alzheimer's disease (AD) is the most frequent neurodegenerative disorder in the elderly,
occurring in approximately 20% of people older than 80. The molecular causes of AD are …