Ongoing clinical trials of nonviral siRNA therapeutics
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 …
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 …
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 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 …
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 …
immunogenicity of antigen and inducing long‐lasting immunity. However, there are only a …
Gene editing with CRISPR-Cas12a guides possessing ribose-modified pseudoknot handles
CRISPR-Cas12a is a leading technology for development of model organisms, therapeutics,
and diagnostics. These applications could benefit from chemical modifications that stabilize …
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
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 …
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 …
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
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 …
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 …
and specific gene silencing through activation of the RNA interference (RNAi) pathway. The …