New gene therapy possibility for DR, glaucoma

A US study has found that an enzyme, genetically reactivated, showed promise for preventing vision loss from serious retinal injury, including optic nerve damage, and from retinal diseases such as diabetic retinopathy (DR) and glaucoma by protecting retinal ganglion cells.


Using mouse models, Mount Sinai investigators used an FDA-approved adeno-associated viral vector to deliver a variant of CaMKII (calcium/calmodulin-dependent protein kinase II) whose activity had been boosted with a mutated amino acid. Lead investigator Associate Professor Bo Chen said CaMKII could be a therapeutic target for vision preservation in conditions that damage retinal ganglion cells and slowed disease progression in both the study’s glaucoma models.


A 2005 study published by the University of Tokushima, Japan, also linked misregulation of CaMKII to Alzheimer’s disease, Angelman syndrome and heart arrhythmia.


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