Article

Overcoming the Blood-Brain Barrier for Gene Therapy via Systemic Administration of GSH-Responsive Silica Nanocapsules

2022-10-28

Abstract excerpt

CRISPR genome editing has demonstrated great potential to treat the root causes of many genetic diseases, including central nervous system (CNS) disorders. However, the promise of brain-targeted therapeutic genome editing relies on the efficient delivery of biologics bypassing the blood-brain barrier (BBB), which represents a substantial challenge in the development of CRISPR therapeutics. In this study, we create...

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Literature Corpus work: 7d560404-cf06-5778-bd3d-6c4bdc84fd85
DOI: 10.1101/2022.10.27.513950

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