Multiplex CRISPR/Cas9-based genome engineering from a single lentiviral vector

por Kabadi, Ami M., Ousterout, David G., Hilton, Isaac B., Gersbach, Charles A.
Publicado 2014

A CRISPR/Cas9-Based System for Reprogramming Cell Lineage Specification

por Chakraborty, Syandan, Ji, HaYeun, Kabadi, Ami M., Gersbach, Charles A., Christoforou, Nicolas, Leong, Kam W.
Publicado 2014

Multiplex CRISPR/Cas9-Based Genome Editing for Correction of Dystrophin Mutations that Cause Duchenne Muscular Dystrophy

por Ousterout, David G., Kabadi, Ami M., Thakore, Pratiksha I., Majoros, William H., Reddy, Timothy E., Gersbach, Charles A.
Publicado 2015

Correction of Dystrophin Expression in Cells From Duchenne Muscular Dystrophy Patients Through Genomic Excision of Exon 51 by Zinc Finger Nucleases

por Ousterout, David G, Kabadi, Ami M, Thakore, Pratiksha I, Perez-Pinera, Pablo, Brown, Matthew T, Majoros, William H, Reddy, Timothy E, Gersbach, Charles A
Publicado 2015

Enhanced MyoD-Induced Transdifferentiation to a Myogenic Lineage by Fusion to a Potent Transactivation Domain

por Kabadi, Ami M., Thakore, Pratiksha I., Vockley, Christopher M., Ousterout, David G., Gibson, Tyler M., Guilak, Farshid, Reddy, Timothy E., Gersbach, Charles A.
Publicado 2014

Reading Frame Correction by Targeted Genome Editing Restores Dystrophin Expression in Cells From Duchenne Muscular Dystrophy Patients

por Ousterout, David G, Perez-Pinera, Pablo, Thakore, Pratiksha I, Kabadi, Ami M, Brown, Matthew T, Qin, Xiaoxia, Fedrigo, Olivier, Mouly, Vincent, Tremblay, Jacques P, Gersbach, Charles A
Publicado 2013

Highly Specific Epigenome Editing by CRISPR/Cas9 Repressors for Silencing of Distal Regulatory Elements

por Thakore, Pratiksha I., D’Ippolito, Anthony M, Song, Lingyun, Safi, Alexias, Shivakumar, Nishkala K., Kabadi, Ami M., Reddy, Timothy E., Crawford, Gregory E., Gersbach, Charles A.
Publicado 2015

RNA-guided gene activation by CRISPR-Cas9-based transcription factors

por Perez-Pinera, Pablo, Kocak, D. Dewran, Vockley, Christopher M., Adler, Andrew F., Kabadi, Ami M., Polstein, Lauren R., Thakore, Pratiksha I., Glass, Katherine A., Ousterout, David G., Leong, Kam W., Guilak, Farshid, Crawford, Gregory E., Reddy, Timothy E., Gersbach, Charles A.
Publicado 2013