Caption: This image represents an infection-fighting cell called a neutrophil. In this artist’s rendering, the cell’s DNA is being “edited” to help restore its ability to fight bacterial invaders.
Credit: NIAID, NIH
For gene therapy research, the perennial challenge has been devising a reliable way to insert safely a working copy of a gene into relevant cells that can take over for a faulty one. But with the recent discovery of powerful gene editing tools, the landscape of opportunity is starting to change. Instead of threading the needle through the cell membrane with a bulky gene, researchers are starting to design ways to apply these tools in the nucleus—to edit out the disease-causing error in a gene and allow it to work correctly.
While the research is just getting under way, progress is already being made for a rare inherited immunodeficiency called chronic granulomatous disease (CGD). As published recently in Science Translational Medicine, a team of NIH researchers has shown with the help of the latest CRISPR/Cas9 gene-editing tools, they can correct a mutation in human blood-forming adult stem cells that triggers a common form of CGD. What’s more, they can do it without introducing any new and potentially disease-causing errors to the surrounding DNA sequence .
When those edited human cells were transplanted into mice, the cells correctly took up residence in the bone marrow and began producing fully functional white blood cells. The corrected cells persisted in the animal’s bone marrow and bloodstream for up to five months, providing proof of principle that this lifelong genetic condition and others like it could one day be cured without the risks and limitations of our current treatments.
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adult stem cells, bacteria, CGD, chronic granulomatous disease, clinical trials, CRISPR, CRISPR-Cas, CRISPR/Cas9, DNA editing, fungi, gene therapy, genetics, hematopoietic stem cells, immunodeficiency, immunology, infectious disease, inherited immuodeficiency, neutrophil, rare disease, translational medicine, X chromosome, X-linked chronic granulomatous disease
When people think about the human microbiome—the scientific term for all of the microbes that live in and on our bodies—the focus is often on bacteria. But Keisha Findley, the young researcher featured in today’s LabTV video, is fascinated by a different part of the microbiome: fungi.
While earning her Ph.D. at Duke University, Durham, N.C., Findley zeroed in on Cryptococcus neoformans, a common, single-celled fungus that can lead to life-threatening infections, especially in people with weakened immune systems. Now, as a postdoctoral fellow at NIH’s National Human Genome Research Institute, Bethesda, MD, she is part of an effort to survey all of the fungi, as well as bacteria, that live on healthy human skin. The goal is to get a baseline understanding of these microbial communities and then examine how they differ between healthy people and those with skin conditions such as acne, athlete’s foot, skin ulcers, psoriasis, or eczema.
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acne, athlete's foot, bacteria, Cryptococcus neoformans, eczema, fungi, fungus, microbes, microbiome, psoriasis, skin, skin diseases, skin ulcers