Polyglutamine Disease Modeling: Epitope Based Screen for Homologous Recombination using CRISPR/Cas9 System.
We have previously reported the genetic correction of Huntington's disease (HD) patient-derived induced pluripotent stem cells using traditional homologous recombination (HR) approaches. To extend this work, we have adopted a CRISPR-based genome editing approach to improve the efficiency of recombination in order to generate allelic isogenic HD models in human cells. Incorporation of a rapid antibody-based screening approach to measure recombination provides a powerful method to determine relative efficiency of genome editing for modeling polyglutamine diseases or understanding factors that modulate CRISPR/Cas9 HR.
The Buck Institute for Research on Aging, Novato, California, USA.
Mahru C
MC
An
The Buck Institute for Research on Aging, Novato, California, USA.
Robert N
RN
O'Brien
The Buck Institute for Research on Aging, Novato, California, USA.
Ningzhe
N
Zhang
School of Applied Life Sciences, Keck Graduate Institute, Claremont, California, USA.
Biranchi N
BN
Patra
School of Applied Life Sciences, Keck Graduate Institute, Claremont, California, USA.
Michael
M
De La Cruz
School of Applied Life Sciences, Keck Graduate Institute, Claremont, California, USA.
Animesh
A
Ray
The Buck Institute for Research on Aging, Novato, California, USA.
Lisa M
LM
Ellerby