A base-editing approach optimized to target the retina shows high editing rates in a mouse model of Stargardt disease, as well as in nonhuman primates and ex vivo human retinal explants, paving the wa...

Small heat shock proteins (sHSPs), including HSPB1, are essential regulators of cellular proteostasis that interact with unfolded and partially folded proteins to prevent aberrant misfolding and aggre...

Adeno-associated virus (AAV)-mediated gene therapy is widely applied to treat numerous hereditary diseases in animal models and humans. The specific expression of AAV-delivered transgenes driven by ce...

imageimageThe split intein-mediated protein ligation (SIMPL) system for detecting protein-protein interactions has been enhanced by incorporating tri-part Nanoluciferase. This updated system, called S...

DNA-encoded libraries hold great potential for discovering small, cyclized peptides with drug potential. Split-intein circular ligation of peptides and proteins (SICLOPPS) is a well-established method...

Exon skipping technologies remain hindered by aberrant splicing and low efficacy. Here, Miskalis et.al. developed SPLICER, a Cas9 base editor toolbox which enhances overall exon skipping efficiency an...

Adeno-associated virus (AAV)-based gene therapy has become one of the key directions of modern translational medicine geared towards treatment of hereditary ...