Donald B Kohn
Affiliation: University of California
Masiuk K, Laborada J, Roncarolo M, Hollis R, Kohn D. Lentiviral Gene Therapy in HSCs Restores Lineage-Specific Foxp3 Expression and Suppresses Autoimmunity in a Mouse Model of IPEX Syndrome. Cell Stem Cell. 2018;: pubmed publisher
..These findings demonstrate preclinical efficacy for the treatment of IPEX patients by autologous HSC transplant and may provide valuable insights into new cell therapies for autoimmunity. ..
Cooper A, Lill G, Gschweng E, Kohn D. Rescue of splicing-mediated intron loss maximizes expression in lentiviral vectors containing the human ubiquitin C promoter. Nucleic Acids Res. 2015;43:682-90 pubmed publisher
..Reversal of the UBC promoter also prevented intron loss and restored full expression in bidirectional lentiviral vectors. ..
Kohn D, Gaspar H. How We Manage Adenosine Deaminase-Deficient Severe Combined Immune Deficiency (ADA SCID). J Clin Immunol. 2017;37:351-356 pubmed publisher
Masiuk K, Brown D, Laborada J, Hollis R, Urbinati F, Kohn D. Improving Gene Therapy Efficiency through the Enrichment of Human Hematopoietic Stem Cells. Mol Ther. 2017;25:2163-2175 pubmed publisher
..Thus, we demonstrate a translatable method to improve the clinical and commercial viability of gene therapy for genetic blood cell diseases. ..
Bradford K, Moretti F, Carbonaro Sarracino D, Gaspar H, Kohn D. Adenosine Deaminase (ADA)-Deficient Severe Combined Immune Deficiency (SCID): Molecular Pathogenesis and Clinical Manifestations. J Clin Immunol. 2017;37:626-637 pubmed publisher
..We review the pathogenic mechanisms and clinical manifestations of ADA SCID. ..
Kohn D. Historical Perspective on the Current Renaissance for Hematopoietic Stem Cell Gene Therapy. Hematol Oncol Clin North Am. 2017;31:721-735 pubmed publisher
..Gene editing using site-specific endonucleases is emerging as a promising technology for gene therapy and is moving into clinical trials. ..
Morgan R, Gray D, Lomova A, Kohn D. Hematopoietic Stem Cell Gene Therapy: Progress and Lessons Learned. Cell Stem Cell. 2017;21:574-590 pubmed publisher
..We highlight the progress, successes, and remaining challenges toward the development of HSC gene therapies and discuss lessons they provide for the development of future clinical stem cell therapies. ..
Kohn D, Kuo C. New frontiers in the therapy of primary immunodeficiency: From gene addition to gene editing. J Allergy Clin Immunol. 2017;139:726-732 pubmed publisher
..Gene therapy is providing exciting new treatment options for patients with PIDs, and advances are sure to continue. ..