Chemistry, structure and function of approved oligonucleotide therapeutics

M Egli, M Manoharan - Nucleic Acids Research, 2023 - academic.oup.com
Eighteen nucleic acid therapeutics have been approved for treatment of various diseases in
the last 25 years. Their modes of action include antisense oligonucleotides (ASOs), splice …

[HTML][HTML] Emerging concepts of miRNA therapeutics: from cells to clinic

C Diener, A Keller, E Meese - Trends in Genetics, 2022 - cell.com
MicroRNAs (miRNAs) are very powerful genetic regulators, as evidenced by the fact that a
single miRNA can direct entire cellular pathways via interacting with a broad spectrum of …

[HTML][HTML] Drug delivery systems for RNA therapeutics

K Paunovska, D Loughrey, JE Dahlman - Nature Reviews Genetics, 2022 - nature.com
RNA-based gene therapy requires therapeutic RNA to function inside target cells without
eliciting unwanted immune responses. RNA can be ferried into cells using non-viral drug …

Emerging new therapeutic antibody derivatives for cancer treatment

S Jin, Y Sun, X Liang, X Gu, J Ning, Y Xu… - Signal transduction and …, 2022 - nature.com
Monoclonal antibodies constitute a promising class of targeted anticancer agents that
enhance natural immune system functions to suppress cancer cell activity and eliminate …

Targeting non-coding RNAs to overcome cancer therapy resistance

BQ Chen, MP Dragomir, C Yang, Q Li, D Horst… - Signal transduction and …, 2022 - nature.com
It is now well known that non-coding RNAs (ncRNAs), rather than protein-coding transcripts,
are the preponderant RNA transcripts. NcRNAs, particularly microRNAs (miRNAs), long non …

The current landscape of nucleic acid therapeutics

JA Kulkarni, D Witzigmann, SB Thomson… - Nature …, 2021 - nature.com
The increasing number of approved nucleic acid therapeutics demonstrates the potential to
treat diseases by targeting their genetic blueprints in vivo. Conventional treatments …

Lipids and lipid derivatives for RNA delivery

Y Zhang, C Sun, C Wang, KE Jankovic… - Chemical reviews, 2021 - ACS Publications
RNA-based therapeutics have shown great promise in treating a broad spectrum of
diseases through various mechanisms including knockdown of pathological genes …

Preclinical development and phase 1 trial of a novel siRNA targeting lipoprotein (a)

MJ Koren, PM Moriarty, SJ Baum, J Neutel… - Nature medicine, 2022 - nature.com
Compelling evidence supports a causal role for lipoprotein (a)(Lp (a)) in cardiovascular
disease. No pharmacotherapies directly targeting Lp (a) are currently available for clinical …

Wound healing: from passive to smart dressings

M Farahani, A Shafiee - Advanced Healthcare Materials, 2021 - Wiley Online Library
The universal increase in the number of patients with nonhealing skin wounds imposes a
huge social and economic burden on the patients and healthcare systems. Although, the …

Amyotrophic lateral sclerosis: translating genetic discoveries into therapies

F Akçimen, ER Lopez, JE Landers, A Nath… - Nature Reviews …, 2023 - nature.com
Recent advances in sequencing technologies and collaborative efforts have led to
substantial progress in identifying the genetic causes of amyotrophic lateral sclerosis (ALS) …