This article highlights the current state of macromolecular gene delivery systems, their applications, and the ongoing ...

Gene therapy has emerged as a revolutionary approach to treating genetic disorders by directly addressing underlying genetic abnormalities. However ...

This study reports the important development and characterization of next-generation analogs of the molecule AA263, which was previously identified for its ability to promote adaptive ER proteostasis ...

CRISPR construct to genetically ablate the GABA transporter GAT3 in the mouse visual cortex, with effects on population-level neuronal activity. This work is important, as it sheds light on how GAT3 ...

Antibody–siRNA conjugates (ARCs) are a type of promising drug modalities for cancer therapy. However, initial reports of ARCs present the gene knockdown effect only in limited tissues (e.g., muscles) ...