![]() Scientists are not yet sure how these errors might affect patients. For example, sometimes genome editing tools cut in the wrong spot. However, treatments like the one that Layla received are still experimental because the scientific community and policymakers still have to address technical barriers and ethical concerns surrounding genome editing.Įven though CRISPR improved upon older genome editing technologies, it is not perfect. Doctors tried many treatments before this, but none of them seemed to work, so scientists received special permission to treat Layla using gene therapy. These scientists did not use CRISPR to treat Layla, and instead used another genome editing technology called TALENs. In 2015, scientists successfully used somatic gene therapy when a one-year old in the United Kingdom named Layla received a gene editing treatment to help her fight leukemia, a type of cancer. Somatic therapies, on the other hand, target non-reproductive cells, and changes made in these cells affect only the person who receives the gene therapy. Changes to the DNA of reproductive cells are passed down from generation to generation. Germline therapies change DNA in reproductive cells (like sperm and eggs). There are two different categories of gene therapies: germline therapy and somatic therapy. Genome editing tools have the potential to help treat diseases with a genomic basis, like cystic fibrosis and diabetes. ![]() ![]() Scientists are developing gene therapies - treatments involving genome editing - to prevent and treat diseases in humans. ![]()
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