Ongoing clinical trials of nonviral siRNA therapeutics

EA Narasipura, R VanKeulen-Miller, Y Ma… - Bioconjugate …, 2023 - ACS Publications
Short interfering RNAs (siRNA) are a powerful class of genetic medicines whose clinical
translation can be hindered by their suboptimal delivery properties in vivo. Here, we provide …

CRISPR/Cas and argonaute-based biosensors for pathogen detection

Z Yang, S Mao, L Wang, S Fu, Y Dong… - ACS …, 2023 - ACS Publications
Over the past few decades, pathogens have posed a threat to human security, and rapid
identification of pathogens should be one of the ideal methods to prevent major public …

From bench to bedside: Improving the clinical safety of GalNAc–siRNA conjugates using seed-pairing destabilization

MK Schlegel, MM Janas, Y Jiang, JD Barry… - Nucleic acids …, 2022 - academic.oup.com
Preclinical mechanistic studies have pointed towards RNA interference-mediated off-target
effects as a major driver of hepatotoxicity for GalNAc–siRNA conjugates. Here, we …

Effects of univariate stiffness and degradation of DNA hydrogels on the transcriptomics of neural progenitor cells

B Zhou, B Yang, Q Liu, L Jin, Y Shao… - Journal of the …, 2023 - ACS Publications
Mechanical interactions between cells and extracellular matrix (ECM) are critical for stem
cell fate decision. Synthetic models of ECM, such as hydrogels, can be used to precisely …

cGAS‐STING pathway agonists are promising vaccine adjuvants

X Tian, J Ai, X Tian, X Wei - Medicinal Research Reviews, 2024 - Wiley Online Library
Adjuvants are of critical value in vaccine development as they act on enhancing
immunogenicity of antigen and inducing long‐lasting immunity. However, there are only a …

Gene editing with CRISPR-Cas12a guides possessing ribose-modified pseudoknot handles

EA Ageely, R Chilamkurthy, S Jana… - Nature …, 2021 - nature.com
CRISPR-Cas12a is a leading technology for development of model organisms, therapeutics,
and diagnostics. These applications could benefit from chemical modifications that stabilize …

2′-O-Alkyl-N3-Methyluridine Functionalized Passenger Strand Improves RNAi Activity by Modulating the Thermal Stability

A Sahoo, S Gupta, G Das, A Ghosh… - ACS Medicinal …, 2024 - ACS Publications
Herein, we have demonstrated that the siRNA activity could be enhanced by incorporating
the guide strand in the RISC complex through thermodynamic asymmetry caused by m3U …

Site-specific incorporation of 2′, 5′-linked nucleic acids enhances therapeutic profile of antisense oligonucleotides

TP Prakash, J Yu, W Shen, CL De Hoyos… - ACS Medicinal …, 2021 - ACS Publications
Site-specific incorporation of 2′-modifications and neutral linkages in the deoxynucleotide
gap region of toxic phosphorothioate (PS) gapmer ASOs can enhance therapeutic index and …

Enhancing Gene Delivery to Breast Cancer with Highly Efficient siRNA Loading and pH-Responsive Small Extracellular Vesicles

G Kim, R Zhu, S Yu, B Fan, H Jeon, J Leon… - ACS Biomaterials …, 2024 - ACS Publications
Small extracellular vesicles (sEVs) are promising nanocarriers for drug delivery to treat a
wide range of diseases due to their natural origin and innate homing properties. However …

Chemical strategies for strand selection in short-interfering RNAs

AJ Varley, JP Desaulniers - RSC advances, 2021 - pubs.rsc.org
Therapeutic small interfering RNAs (siRNAs) are double stranded RNAs capable of potent
and specific gene silencing through activation of the RNA interference (RNAi) pathway. The …