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Using genetic technology for rare diseases

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It's a race against a single genetic marker -- and musician turned biotech start-up CEO Casey McPherson says the stakes for him couldn't be higher.
The article from MSN discusses the application of genetic technology in addressing rare diseases, highlighting the transformative potential of gene therapies and genome editing. It explains how these technologies can offer hope to patients with conditions like cystic fibrosis, hemophilia, and sickle cell anemia by targeting the root genetic causes rather than just managing symptoms. The piece details the use of CRISPR-Cas9 for precise gene editing, which has shown promise in clinical trials for conditions like Leber congenital amaurosis, a form of inherited blindness. Additionally, it touches on the ethical considerations, regulatory challenges, and the high costs associated with these treatments. The article also emphasizes the importance of genetic screening and the potential for personalized medicine, where treatments are tailored to an individual's genetic makeup, potentially revolutionizing healthcare for rare disease patients.

Read the Full MSN Article at:
[ https://www.msn.com/en-us/science/biology/using-genetic-technology-for-rare-diseases/ar-AA1zwUiu ]