Contents
Research summary 4
What is Parkinson's disease? 4
Collaboration summary 5
Methods 7
GeneArt
®
Precision TALs technology overview 7
Workflow for genome editing of iPSCs 10
iPSC colony screening methods 12
GeneArt
®
Genomic Cleavage Detection assay 12
TaqMan
®
SNP Genotyping Assay 13
Ion PGM
™
sequencing 14
Exome sequencing for off-target modifications 16
Results 19
Generation of α-synuclein knockout lines in the MSA line 19
Screening for off-target modifications
in the edited MSA lines 25
Correction of LRRK2 G2019S mutation in the PD-3 line 26
Correction of GBA N370S mutation in the PD-3 line 28
Summary, conclusions, and next steps 30
References 31
Ordering information 32
Find out more at lifetechnologies.com/parkinsons
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