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Briefing
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- First Gene Editing Inside Body – Sangamo Therapeutics scientists developed gene therapy that transmits billions of copies of corrective gene and genetic tool that cut DNA inside body of patient with genetic disorder Hunter syndrome
- Hunter’s Syndrome – Occurs due to lack of gene that creates enzyme that breaks down certain carbohydrates, causing colds and ear infections, distorted facial features, heart, skin, eye, and bowel problems, bone and joint irregularities, as well as hearing, breathing, and mental impairments
- How It Works – Uses altered virus to send new gene and two zinc finger proteins, gene editing tool, into liver cells, where fingers cut DNA and insert new gene, allowing cells to make lacking enzyme
- Effectiveness Trials – Effectiveness of treatment will be verified in three months, from which it will be tested in up to 30 adults, with goal of eventually treating very young children
- Potential Side Effects – Include virus causing immune system attack, new gene possibly altering other genes, and virus being transmitted to heart, eggs and sperm
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