Urbinati, Fabrizia

Selection-free genome editing of the sickle mutation in human adult hematopoietic stem/progenitor cells

DeWitt, M. A., Magis, W., Bray, N. L., Wang, T., Berman, J. R., Urbinati, F., Heo, S.-J., Mitros, T., Muñoz, D. P., Boffelli, D., et al. (2016) · Science Translational Medicine

CRISPR and Genetic EngineeringHemoglobinopathies and Related Disorders

Cited by 477

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

CRISPR/Cas9-Mediated Correction of the Sickle Mutation in Human CD34+ cells

Hoban, M. D., Lumaquin, D., Kuo, C. Y., Romero, Z., Long, J., Ho, M., Young, C. S., Mojadidi, M., Fitz-Gibbon, S., Cooper, A. R., et al. (2016) · Molecular Therapy

CRISPR and Genetic EngineeringHemoglobinopathies and Related Disorders

Cited by 220

The woodchuck hepatitis virus post-transcriptional regulatory element reduces readthrough transcription from retroviral vectors

Higashimoto, T., Urbinati, F., Perumbeti, A., Jiang, G., Zarzuela, A., Chang, L.-J., Kohn, D. B., & Malik, P. (2007) · Gene Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 101

Zinc Finger Nucleases Targeting The β-Globin Locus Drive Efficient Correction Of The Sickle Mutation In CD34+ Cells

Hoban, M. D., Joglekar, A. V., Gray, D., Kaufman, M. L., Urbinati, F., Senadheera, S., Cost, G., Reik, A., Holmes, M. C., Gregory, P. D., et al. (2013) · Blood

Hemoglobinopathies and Related DisordersCRISPR and Genetic Engineering

Cited by 2