A Single “All-in-One” Helper-Dependent Adenovirus to Deliver Donor DNA and CRISPR/Cas9 for Efficient Homology-Directed Repair

Ohbayashi, 2005, Correction of chromosomal mutation and random integration in embryonic stem cells with helper-dependent adenoviral vectors, Proc. Natl. Acad. Sci. USA, 102, 13628, 10.1073/pnas.0506598102

Suzuki, 2008, Highly efficient transient gene expression and gene targeting in primate embryonic stem cells with helper-dependent adenoviral vectors, Proc. Natl. Acad. Sci. USA, 105, 13781, 10.1073/pnas.0806976105

Li, 2011, Efficient correction of hemoglobinopathy-causing mutations by homologous recombination in integration-free patient iPSCs, Cell Res., 21, 1740, 10.1038/cr.2011.186

Liu, 2011, Targeted gene correction of laminopathy-associated LMNA mutations in patient-specific iPSCs, Cell Stem Cell, 8, 688, 10.1016/j.stem.2011.04.019

Liu, 2012, Progressive degeneration of human neural stem cells caused by pathogenic LRRK2, Nature, 491, 603, 10.1038/nature11557

Aizawa, 2012, Efficient and accurate homologous recombination in hESCs and hiPSCs using helper-dependent adenoviral vectors, Mol. Ther., 20, 424, 10.1038/mt.2011.266

Umeda, 2013, Albumin gene targeting in human embryonic stem cells and induced pluripotent stem cells with helper-dependent adenoviral vector to monitor hepatic differentiation, Stem Cell Res. (Amst.), 10, 179, 10.1016/j.scr.2012.11.003

Liu, 2014, Modelling Fanconi anemia pathogenesis and therapeutics using integration-free patient-derived iPSCs, Nat. Commun., 5, 4330, 10.1038/ncomms5330

Suzuki, 2014, Targeted gene correction minimally impacts whole-genome mutational load in human-disease-specific induced pluripotent stem cell clones, Cell Stem Cell, 15, 31, 10.1016/j.stem.2014.06.016

Yoshida, 2014, The use of induced pluripotent stem cells to reveal pathogenic gene mutations and explore treatments for retinitis pigmentosa, Mol. Brain, 7, 45, 10.1186/1756-6606-7-45

Zhang, 2015, Aging stem cells. A Werner syndrome stem cell model unveils heterochromatin alterations as a driver of human aging, Science, 348, 1160, 10.1126/science.aaa1356

Yamamoto, 2016, BTK gene targeting by homologous recombination using a helper-dependent adenovirus/adeno-associated virus hybrid vector, Gene Ther., 23, 205, 10.1038/gt.2015.91

Palmer, 2016, Homology requirements for efficient, footprintless gene editing at the CFTR locus in human iPSCs with helper-dependent adenoviral vectors, Mol. Ther. Nucleic Acids, 5, e372, 10.1038/mtna.2016.83

Palmer, 2017, Gene Editing with Helper-Dependent Adenovirus Can Efficiently Introduce Multiple Changes Simultaneously over a Large Genomic Region, Mol. Ther. Nucleic Acids, 8, 101, 10.1016/j.omtn.2017.06.001

Palmer, 2019, Bi-allelic homology-directed repair with helper-dependent adenoviruses, Mol. Ther. Methods Clin. Dev., 15, 285, 10.1016/j.omtm.2019.10.003

Crane, 2015, Targeted correction and restored function of the CFTR gene in cystic fibrosis induced pluripotent stem cells, Stem Cell Reports, 4, 569, 10.1016/j.stemcr.2015.02.005

Palmer, 2019, Production of CRISPR/Cas9-Mediated Self-Cleaving Helper-Dependent Adenoviruses, Mol. Ther. Methods Clin. Dev., 13, 432, 10.1016/j.omtm.2019.04.003

Xia, 2018, Overcoming the Undesirable CRISPR-Cas9 Expression in Gene Correction, Mol. Ther. Nucleic Acids, 13, 699, 10.1016/j.omtn.2018.10.015