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 …

RNAi-based drug design: considerations and future directions

Q Tang, A Khvorova - Nature Reviews Drug Discovery, 2024 - nature.com
More than 25 years after its discovery, the post-transcriptional gene regulation mechanism
termed RNAi is now transforming pharmaceutical development, proved by the recent FDA …

A versatile CRISPR-Cas13d platform for multiplexed transcriptomic regulation and metabolic engineering in primary human T cells

V Tieu, E Sotillo, JR Bjelajac, C Chen, M Malipatlolla… - Cell, 2024 - cell.com
CRISPR technologies have begun to revolutionize T cell therapies; however, conventional
CRISPR-Cas9 genome-editing tools are limited in their safety, efficacy, and scope. To …

[HTML][HTML] Nano-based drug delivery systems: Conventional drug delivery routes, recent developments and future prospects

A Sultana, M Zare, V Thomas, TSS Kumar… - Medicine in Drug …, 2022 - Elsevier
Drug delivery can be defined as the group of approaches a drug or pharmacologically active
agent can be transported to the target cell to treat disease or health issue. Conventional …

A comprehensive review of small interfering RNAs (siRNAs): mechanism, therapeutic targets, and delivery strategies for cancer therapy

J Zhang, B Chen, C Gan, H Sun, J Zhang… - International journal of …, 2023 - Taylor & Francis
Small interfering RNA (siRNA) delivery by nanocarriers has been identified as a promising
strategy in the study and treatment of cancer. Short nucleotide sequences are synthesized …

RNA-based therapeutics: from antisense oligonucleotides to miRNAs

S Bajan, G Hutvagner - Cells, 2020 - mdpi.com
The first therapeutic nucleic acid, a DNA oligonucleotide, was approved for clinical use in
1998. Twenty years later, in 2018, the first therapeutic RNA-based oligonucleotide was …

Induced protein degradation: an emerging drug discovery paradigm

AC Lai, CM Crews - Nature reviews Drug discovery, 2017 - nature.com
Small-molecule drug discovery has traditionally focused on occupancy of a binding site that
directly affects protein function, and this approach typically precludes targeting proteins that …

[HTML][HTML] Nano-scale delivery systems for siRNA delivery in cancer therapy: New era of gene therapy empowered by nanotechnology

N Ebrahimi, MS Manavi, A Nazari, A Momayezi… - Environmental …, 2023 - Elsevier
RNA interference (RNAi) is a unique treatment approach used to decrease a disease's
excessive gene expression, including cancer. SiRNAs may find and destroy homologous …

Therapeutic siRNA: state-of-the-art and future perspectives

M Friedrich, A Aigner - BioDrugs, 2022 - Springer
The highly specific induction of RNA interference-mediated gene knockdown, based on the
direct application of small interfering RNAs (siRNAs), opens novel avenues towards …

Small interfering RNAs (siRNAs) in cancer therapy: a nano-based approach

G Mahmoodi Chalbatani, H Dana… - International journal …, 2019 - Taylor & Francis
Cancer is one of the most complex diseases that has resulted in multiple genetic disorders
and cellular abnormalities. Globally, cancer is the most common health concern disease that …