Research Highlights

The research in the Kohn lab focuses on the development of new methods to treated genetic diseases of blood cells by gene modification of hematopoietic stem cells. Dr. Kohn’s group was the first (and still the only one) to perform a clinical trial with gene transfer to umbilical cord blood CD34+ cells for a genetic disorder (ADA-SCID in 1993) and the first in the U.S. to initiate clinical trials of gene therapy for pediatric HIV/AIDS using bone marrow stem cells. Current projects include:

  1. Performing clinical trials for ADA-deficient SCID, XSCID, Sickle Cell Disease and X-linked Chronic Granulomatous Disease
  2. Developing new lentiviral vectors for regulated gene expression,
  3. Advancing techniques for site-specific gene editing for sickle cell disease and primary immune deficiency diseases
  4. Iimproving clinical hematopoietic stem cell processing. Our research group spans the bench-to-bedside spectrum and provides an excellent training environment for physician/scientists.

Featured Publications

Kuo CY, Long JD, Campo-Fernandez B, … Hollis RP, Kohn DB. Site-Specific Gene Editing of Human Hematopoietic Stem Cells for X-Linked Hyper-IgM Syndrome. Cell Rep 23:2606-2616, 2018.

Lomova A, Clark DN, Miyahira EY, … and Kohn DB. Improving Gene Editing Outcomes in Human Hematopoietic Stem and Progenitor Cells by Temporal Control of DNA Repair. Stem Cells, In Press. PMID: 30372555

Masiuk KE, Laborada J, Roncarolo MG, Hollis RP and Kohn DB. Lentiviral Gene Therapy in HSCs Restores Lineage-Specific Foxp3 Expression and Suppresses Autoimmunity in a Mouse Model of IPEX Syndrome. Cell Stem Cells, In Press.

Featured News

March 25, 2019

Celebrating the launch of The Centennial Campaign for UCLA, this video offers a glimpse into the miraculous work of Broad...... Read More