Manipulating the Prion Protein Gene Sequence and Expression Levels with CRISPR/Cas9.

ABSTRACT: The mammalian prion protein (PrP, encoded by Prnp) is most infamous for its central role in prion diseases, invariably fatal neurodegenerative diseases affecting humans, food animals, and animals in the wild. However, PrP is also hypothesized to be an important receptor for toxic protein conformers in Alzheimer's disease, and is associated with other clinically relevant processes such as cancer and stroke. Thus, key insights into important clinical areas, as well as into understanding PrP functions in normal physiology, can be obtained from studying transgenic mouse models and cell culture systems. However, the Prnp locus is difficult to manipulate by homologous recombination, making modifications of the endogenous locus rarely attempted. Fortunately in recent years genome engineering tech

SUBMITTER: Kaczmarczyk L 

PROVIDER: S-EPMC4851410 | biostudies-literature | 2016

REPOSITORIES: biostudies-literature