Paschon, David E

A TALE nuclease architecture for efficient genome editing

Miller, J. C., Tan, S., Qiao, G., Barlow, K. A., Wang, J., Xia, D. F., Meng, X., Paschon, D. E., Leung, E., Hinkley, S. J., et al. (2011) · Nature Biotechnology

CRISPR and Genetic EngineeringRNA regulation and disease

Cited by 2,098

In vivo genome editing restores haemostasis in a mouse model of haemophilia

Li, H., Haurigot, V., Doyon, Y., Li, T., Wong, S. Y., Bhagwat, A. S., Malani, N., Anguela, X. M., Sharma, R., Ivanciu, L., et al. (2011) · Nature

Hemophilia Treatment and ResearchCRISPR and Genetic Engineering

Cited by 548

Editing T cell specificity towards leukemia by zinc finger nucleases and lentiviral gene transfer

Provasi, E., Genovese, P., Lombardo, A., Magnani, Z., Liu, P.-Q., Reik, A., Chu, V., Paschon, D. E., Zhang, L., Kuball, J., et al. (2012) · Nature Medicine

CAR-T cell therapy researchCRISPR and Genetic Engineering

Cited by 448

Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells

Hoban, M. D., Cost, G. J., Mendel, M. C., Romero, Z., Kaufman, M. L., Joglekar, A. V., Ho, M., Lumaquin, D., Gray, D., Lill, G. R., et al. (2015) · Blood

Hemoglobinopathies and Related DisordersCRISPR and Genetic Engineering

Cited by 320

In vivo genome editing of the albumin locus as a platform for protein replacement therapy

Sharma, R., Anguela, X. M., Doyon, Y., Wechsler, T., DeKelver, R. C., Sproul, S., Paschon, D. E., Miller, J. C., Davidson, R. J., Shivak, D., et al. (2015) · Blood

Hemophilia Treatment and ResearchVirus-based gene therapy research

Cited by 297

Targeted gene addition in human CD34+ hematopoietic cells for correction of X-linked chronic granulomatous disease

De Ravin, S. S., Reik, A., Liu, P.-Q., Li, L., Wu, X., Su, L., Raley, C., Theobald, N., Choi, U., Song, A. H., et al. (2016) · Nature Biotechnology

Virus-based gene therapy researchCRISPR and Genetic Engineering

Cited by 195

Functional footprinting of regulatory DNA

Vierstra, J., Reik, A., Chang, K.-H., Stehling-Sun, S., Zhou, Y., Hinkley, S. J., Paschon, D. E., Zhang, L., Psatha, N., Bendana, Y. R., et al. (2015) · Nature Methods

Genomics and Chromatin DynamicsCRISPR and Genetic Engineering

Cited by 133

Non-viral Delivery of Zinc Finger Nuclease mRNA Enables Highly Efficient In Vivo Genome Editing of Multiple Therapeutic Gene Targets

Conway, A., Mendel, M., Kim, K., McGovern, K., Boyko, A., Zhang, L., Miller, J. C., DeKelver, R. C., Paschon, D. E., Mui, B. L., et al. (2019) · Molecular Therapy

RNA Interference and Gene DeliveryCRISPR and Genetic Engineering

Cited by 95