Gene editing technology has rapidly evolved over the past decade. It allows the insertion, deletion or replacement or DNA in the genome of a living organism. It is performed using engineered nucleases, that are sometimes referred to as “molecular scissors.” These nucleases create double-strand breaks (DSBs) at specific locations in the genome to insert the new DNA.
Gene therapy, which involves using gene editing to treat human beings, is still in its early days. However, scientists believe they will someday be able to alter the genome of humans to remove may different genetic diseases or to treat specific illnesses. Scientists are working to determine how safe specific gene therapies are on human beings.
In a recent breakthrough, scientists have used gene editing technology to insert DNA into hematopoietic stem cells. The goal of their research was to give hematopoietic stem cells the ability to cure specific hereditary and congenital diseases.
The research, entitled “Gene Editing of Human Hematopoietic Stem and Progenitor Cells: Promise and Potential Hurdles”, was recently published in the journal Human Gene Therapy and Stem Cells & Development.
The paper discusses how previous gene addition strategies have worked and how they have informed current techniques. It then discusses state-of-the-art gene editing technology and how it may be used to repair genetic flaws in hematopoietic stem cells. They believe that using the technologies to improve hematopoietic stem cells may allow people with certain genetic illnesses to safely receive a transplant of their own cells — after they have been modified.
Dr. Terence R. Flotte, Editor-in-Chief of the journal which published the article, says of the its contents: “Gene editing is the hottest new technology in gene therapy. The use of this approach to genetically modify hematopoietic stem and progenitor cells is very promising, but requires a careful assessment, This mini-review by Dr. Dunbar’s group at NIH provides a very insightful analysis of recent advances and current limitations of this approach.”
Source: Gene editing of hematopoietic stem cells can cure many hereditary and congenital diseases
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