[HTML][HTML] RNA editing: Expanding the potential of RNA therapeutics

BJ Booth, S Nourreddine, D Katrekar, Y Savva, D Bose… - Molecular Therapy, 2023 - cell.com
RNA therapeutics have had a tremendous impact on medicine, recently exemplified by the
rapid development and deployment of mRNA vaccines to combat the COVID-19 pandemic …

Amplifying gene expression with RNA-targeted therapeutics

O Khorkova, J Stahl, A Joji, CH Volmar… - Nature Reviews Drug …, 2023 - nature.com
Many diseases are caused by insufficient expression of mutated genes and would benefit
from increased expression of the corresponding protein. However, in drug development, it …

Programmable RNA base editing via targeted modifications

J Song, Y Zhuang, C Yi - Nature Chemical Biology, 2024 - nature.com
Clustered regularly interspaced short palindromic repeats (CRISPR)-based genome editors
are powerful tools in biology and hold great promise for the treatment of human diseases …

Programmable RNA sensing for cell monitoring and manipulation

Y Qian, J Li, S Zhao, EA Matthews, M Adoff, W Zhong… - Nature, 2022 - nature.com
RNA is a central and universal mediator of genetic information underlying the diversity of cell
types and cell states, which together shape tissue organization and organismal function …

Precision RNA base editing with engineered and endogenous effectors

LS Pfeiffer, T Stafforst - Nature Biotechnology, 2023 - nature.com
RNA base editing refers to the rewriting of genetic information within an intact RNA molecule
and serves various functions, such as evasion of the endogenous immune system and …

CRISPR-free, programmable RNA pseudouridylation to suppress premature termination codons

J Song, L Dong, H Sun, N Luo, Q Huang, K Li, X Shen… - Molecular Cell, 2023 - cell.com
Summary Nonsense mutations, accounting for> 20% of disease-associated mutations, lead
to premature translation termination. Replacing uridine with pseudouridine in stop codons …

[PDF][PDF] Precise in vivo RNA base editing with a wobble-enhanced circular CLUSTER guide RNA

P Reautschnig, C Fruhner, N Wahn, CP Wiegand… - Nature …, 2024 - nature.com
Recruiting the endogenous editing enzyme adenosine deaminase acting on RNA (ADAR)
with tailored guide RNAs for adenosine-to-inosine (A-to-I) RNA base editing is promising for …

RNA editing enzymes: structure, biological functions and applications

D Zhang, L Zhu, Y Gao, Y Wang, P Li - Cell & Bioscience, 2024 - Springer
With the advancement of sequencing technologies and bioinformatics, over than 170
different RNA modifications have been identified. However, only a few of these modifications …

Impact of stereopure chimeric backbone chemistries on the potency and durability of gene silencing by RNA interference

W Liu, N Iwamoto, S Marappan, K Luu… - Nucleic Acids …, 2023 - academic.oup.com
Herein, we report the systematic investigation of stereopure phosphorothioate (PS) and
phosphoryl guanidine (PN) linkages on siRNA-mediated silencing. The incorporation of …

Gene therapy strategies targeting aging-related diseases

J Yu, T Li, J Zhu - Aging and disease, 2023 - pmc.ncbi.nlm.nih.gov
Rapid advancements have taken place in gene therapy technology. However, effective
methods for treating aging-or age-related chronic diseases, which are often closely related …