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Efficacy and safety of an investigational single-course CRISPR base-editing therapy targeting PCSK9 in nonhuman primate and mouse models

They are chemically and functionally identical to FDA-approved prescription drugs that are widely prescribed for diabetes and weight management

Excessive production of RAGE on brain microvascular endothelial cells increases A transport into the brain tissue and decreases its removal, leading to the accumulation of A in the brain tissue, which in turn triggers a series of neurodegenerative pathological changes, such as the formation of amyloid plaques, the formation of neurofibrillary tangles, and the loss of neurons (23)

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