Kohn, Donald B.

Gene therapy comes of age

Dunbar, C. E., High, K. A., Joung, J. K., Kohn, D. B., Ozawa, K., & Sadelain, M. (2018) · Science

Virus-based gene therapy researchCRISPR and Genetic Engineering

Cited by 1,410

Human hematopoietic stem/progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivo

Holt, N., Wang, J., Kim, K., Friedman, G., Wang, X., Taupin, V., Crooks, G. M., Kohn, D. B., Gregory, P. D., Holmes, M. C., et al. (2010) · Nature Biotechnology

HIV Research and TreatmentCRISPR and Genetic Engineering

Cited by 677

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

A Modified γ-Retrovirus Vector for X-Linked Severe Combined Immunodeficiency

Hacein-Bey-Abina, S., Pai, S.-Y., Gaspar, H. B., Armant, M., Berry, C. C., Blanche, S., Bleesing, J., Blondeau, J., de Boer, H., Buckland, K. F., et al. (2014) · New England Journal of Medicine

Virus-based gene therapy researchImmunodeficiency and Autoimmune Disorders

Cited by 412

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

Transient Gene Expression by Nonintegrating Lentiviral Vectors

Nightingale, S. J., Hollis, R. P., Pepper, K. A., Petersen, D., Yu, X.-J., Yang, C., Bahner, I., & Kohn, D. B. (2006) · Molecular Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 192

Frequency and causes of adrenal crises over lifetime in patients with 21-hydroxylase deficiency

Reisch, N., Willige, M., Kohn, D., Schwarz, H.-P., Allolio, B., Reincke, M., Quinkler, M., Hahner, S., & Beuschlein, F. (2012) · European Journal of Endocrinology

Sexual Differentiation and DisordersAdrenal Hormones and Disorders

Cited by 136

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

Differentiation of RPE cells from integration-free iPS cells and their cell biological characterization

Hazim, R. A., Karumbayaram, S., Jiang, M., Dimashkie, A., Lopes, V. S., Li, D., Burgess, B. L., Vijayaraj, P., Alva-Ornelas, J. A., Zack, J. A., et al. (2017) · Stem Cell Research & Therapy

Retinal Development and DisordersPluripotent Stem Cells Research

Cited by 66

Integrase-defective Lentiviral Vectors as a Delivery Platform for Targeted Modification of Adenosine Deaminase Locus

Joglekar, A. V., Hollis, R. P., Kuftinec, G., Senadheera, S., Chan, R., & Kohn, D. B. (2013) · Molecular Therapy

Virus-based gene therapy researchCRISPR and Genetic Engineering

Cited by 65

Gene therapy for blood diseases

Kohn, D. B. (2019) · Current Opinion in Biotechnology

Virus-based gene therapy researchHemophilia Treatment and Research

Cited by 39

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