In Vivo Gene Editing Of Tau Locus Via Liponanoparticle Delivery

Tech ID: 33310 / UC Case 2024-026-0

Patent Status

Patent Pending

Brief Description

Delivery technologies such as lipid nanoparticles (LNP) offer significant advantages over the delivery of free RNA for various RNA therapeutic, vaccine, and basic science applications. 

UC Berkeley researchers developed a new class of lipid nanoparticle (LNP) which is effective in delivering various types of nuclei acids in different tissues.  The LNP was successfully tested in in-vivo mouse models and therefore poses a significant promise in the gene editing field. The lipid formulation was packaged together with CRISPR Cas9 and a gRNA targeting the endogenous Tau locus. Tau dysrregulation is a pathological feature of Alzheimers disease, thus the invention provides a means to intervene in the development of pathological states associated with Tau aggregate formation. 

Suggested uses

  • therapeutic applications, particularly ones targeting the Tau locus (e.g., Alzheimers disease)

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  • Doudna, Jennifer A.

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