Lentiviral vector mediated gene therapy for type I Dent disease ameliorates Dent disease-like phenotypes for three months in ClC-5 null mice

por Yadav, Manish Kumar, Yoo, Kyung Whan, Atala, Anthony, Lu, Baisong
Publicado 2022

Sensitive and reliable evaluation of single-cut sgRNAs to restore dystrophin by a GFP-reporter assay

por Lyu, Pin, Yoo, Kyung Whan, Yadav, Manish Kumar, Atala, Anthony, Aartsma-Rus, Annemieke, van Putten, Maaike, Duan, Dongsheng, Lu, Baisong
Publicado 2020

Delivering SaCas9 mRNA by lentivirus-like bionanoparticles for transient expression and efficient genome editing

por Lu, Baisong, Javidi-Parsijani, Parisa, Makani, Vishruti, Mehraein-Ghomi, Farideh, Sarhan, Walaa Mohamed, Sun, Dongjun, Yoo, Kyung Whan, Atala, Zachary P, Lyu, Pin, Atala, Anthony
Publicado 2019