Nucleic acid drugs: recent progress and future perspectives

X Sun, S Setrerrahmane, C Li, J Hu, H Xu - Signal Transduction and …, 2024‏ - nature.com
High efficacy, selectivity and cellular targeting of therapeutic agents has been an active area
of investigation for decades. Currently, most clinically approved therapeutics are small …

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 …

Thiomorpholino oligonucleotides as a robust class of next generation platforms for alternate mRNA splicing

BT Le, S Paul, K Jastrzebska, H Langer… - Proceedings of the …, 2022‏ - pnas.org
Recent advances in drug development have seen numerous successful clinical translations
using synthetic antisense oligonucleotides (ASOs). However, major obstacles, such as …

Targeting the liver with nucleic acid therapeutics for the treatment of systemic diseases of liver origin

A Gogate, J Belcourt, M Shah, AZ Wang… - Pharmacological …, 2024‏ - Elsevier
Systemic diseases of liver origin (SDLO) are complex diseases in multiple organ systems,
such as cardiovascular, musculoskeletal, endocrine, renal, respiratory, and sensory organ …

The potential of RNA-based therapy for kidney diseases

T Bondue, L van den Heuvel, E Levtchenko… - Pediatric Nephrology, 2023‏ - Springer
Inherited kidney diseases (IKDs) are a large group of disorders affecting different nephron
segments, many of which progress towards kidney failure due to the absence of curative …

Challenges of Assessing Exon 53 Skip** of the Human DMD Transcript with Locked Nucleic Acid-Modified Antisense Oligonucleotides in a Mouse Model for …

S Engelbeen, D O'Reilly, D Van De Vijver… - nucleic acid …, 2023‏ - liebertpub.com
Antisense oligonucleotide (AON)-mediated exon skip** is a promising therapeutic
approach for Duchenne muscular dystrophy (DMD) patients to restore dystrophin expression …

Next steps for the optimization of exon therapy for Duchenne muscular dystrophy

G Filonova, A Aartsma-Rus - Expert Opinion on Biological Therapy, 2023‏ - Taylor & Francis
Introduction It is established that the exon-skip** approach can restore dystrophin in
Duchenne muscular dystrophy (DMD) patients. However, dystrophin restoration levels are …

[HTML][HTML] Oligonucleotide-based therapies for chronic HBV infection: a primer on biochemistry, mechanisms and antiviral effects

A Vaillant - Viruses, 2022‏ - mdpi.com
Three types of oligonucleotide-based medicines are under clinical development for the
treatment of chronic HBV infection. Antisense oligonucleotides (ASOs) and synthetic …

[HTML][HTML] Therapeutically significant microRNAs in primary and metastatic brain malignancies

AA Balachandran, LM Larcher, S Chen, RN Veedu - Cancers, 2020‏ - mdpi.com
Simple Summary The overall survival of brain cancer patients remains grim, with
conventional therapies such as chemotherapy and radiotherapy only providing marginal …

Antisense oligonucleotide-mediated splice switching: potential therapeutic approach for cancer mitigation

P Raguraman, AA Balachandran, S Chen… - Cancers, 2021‏ - mdpi.com
Simple Summary Splicing is an important mechanism by which precursor mRNA is modified
into mature mRNA. This splicing plays a major role in the generation of different proteins …