Glioblastoma, Fanconi Anemia, HIV, Inherited immunodeficiencies, non-malignant bone marrow failure, Genetic Disorders, portable gene therapy, Gene Therapy, Health Disparities, Blood Stem Cells, Hematopoietic Stem Cells, Global Health, Gene Editing, CRISPR,
Dr. Jennifer Adair is developing gene therapies that safely fine-tune the DNA sequences of blood stem cells to treat genetic disorders, HIV and cancers. Gene therapy — the editing of our DNA to treat disease — is a clinical reality today, but only in a handful of rich countries. As the treatment process stands right now, it depends almost entirely on highly engineered viruses made in high-tech, multimillion-dollar facilities. However, Dr. Adair’s goal is to develop a safe, cost-effective, less invasive and clinically relevant process for gene editing that takes place entirely with the body of a patient – and one that can be accessible to patients worldwide. To help reach this goal, she and colleagues have developed a technology for portable gene therapy. Lab tests show the technology can produce genetically modified blood stem cells that are at least as good as those manufactured in highly specialized clean rooms, requiring less than half the staff. In 2019, she and colleagues published in Nature Materials, a proof-of-principle laboratory study showing, for the first time, that gold nanoparticles loaded with CRISPR and other gene-editing tools safely and effectively edited blood stem cells. The findings raise the possibility that gold could be a key to making gene therapy for HIV and other blood disorders more accessible – with the golden nanoparticles potentially helping to make emerging, high-tech treatments accessible and affordable worldwide. Dr. Adair is an assistant member of the Clinical Research Division at Fred Hutch. She is a research assistant professor of medical oncology at the University of Washington School of Medicine.