Krzysztof Palczewski

Engineered mRNA-packaging virus-like particles enable efficient and precise mitochondrial base editing in vitro and in vivo

This study develops mtBE-VLPs for transient mitochondrial base editing, achieving high efficiency and reduced off-target effects in vitro and in vivo.

A cargo receptor entrapment complex is a therapeutic node for genetically and clinically distinct proteinopathies

This study identifies a therapeutic target, the entrapment complex, that clears toxic protein accumulation in distinct proteinopathies by disrupting a cargo receptor complex.

Impaired axonal transport at the optic nerve head contributes to neurodegeneration in a novel Cre-inducible mouse model of myocilin glaucoma

This study develops a novel Cre-inducible mouse model of myocilin glaucoma that replicates key human disease features, showing impaired axonal transport precedes retinal ganglion cell loss.

Homeostatic plasticity triggered by rod photoreceptor degenerative disease is associated with maintenance of sensitive night vision

The paper shows that homeostatic plasticity in the retina maintains sensitive night vision despite rod photoreceptor degeneration in a mouse model of Retinitis Pigmentosa.

Melanopsin Carboxy-terminus Phosphorylation Plasticity and Bulk Negative Charge, not Strict Site Specificity, Achieves Phototransduction Deactivation