Trionfini, Piera

CRISPR-Cas9-Mediated Correction of the G189R-PAX2 Mutation in Induced Pluripotent Stem Cells from a Patient with Focal Segmental Glomerulosclerosis. [electronic resource] - The CRISPR journal 04 2019 - 108-120 p. digital

Publication Type: Journal Article; Research Support, Non-U.S. Gov't

2573-1602

10.1089/crispr.2018.0048 doi


Adult
CRISPR-Associated Protein 9--genetics
CRISPR-Cas Systems--genetics
Cell Differentiation
Clustered Regularly Interspaced Short Palindromic Repeats--genetics
Genetic Engineering--methods
Germ-Line Mutation--genetics
Glomerulosclerosis, Focal Segmental--genetics
Humans
Induced Pluripotent Stem Cells--metabolism
Kidney Glomerulus--metabolism
Mutation--genetics
PAX2 Transcription Factor--analysis
Podocytes--chemistry
Polymorphism, Single Nucleotide--genetics