High-efficiency base editing in the retina in primates and human tissues - Nature Medicine 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...

www.nature.com/articles/s41...

Capturing the Conformational Heterogeneity of HSPB1 Chaperone Oligomers at Atomic Resolution 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...

www.biorxiv.org/content/10.1...

Engineering of the AAV-Compatible Hair Cell-Specific Small-Size Myo15 Promoter for Gene Therapy in the Inner Ear | Research 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...

spj.science.org/doi/10.34133...

A split intein and split luciferase-coupled system for detecting protein-protein interactions | Molecular Systems Biology 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...

www.embopress.org/doi/full/10....

Optimized Construction of a Yeast SICLOPPS Library for Unbiased In Vivo Selection of Cyclic Peptides 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...

pubs.acs.org/doi/10.1021/...

SPLICER: a highly efficient base editing toolbox that enables in vivo therapeutic exon skipping - Nature Communications 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...

www.nature.com/articles/s41...

Frontiers | Protein trans-splicing: optimization of intein-mediated GFP assembly as a model for the development of gene therapy Adeno-associated virus (AAV)-based gene therapy has become one of the key directions of modern translational medicine geared towards treatment of hereditary ...

www.frontiersin.org/journals/bio...