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) ...

Abstract Summary Pharmacological enhancement of endoplasmic reticulum (ER) proteostasis is an attractive strategy to mitigate pathology linked to etiologically-diverse protein misfolding diseases.

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 ...