#rare-genetic-diseases

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#gene-editing #base-editing #prime-editing #personalized-medicine #crispr #sickle-cell-disease
Medicine
fromThe Washington Post
1 week ago

David Liu unlocks the power of gene editing to treat rare genetic diseases

Base and prime gene-editing technologies can precisely correct genetic mutations, enabling personalized therapies that can cure life-threatening inherited diseases.
fromThe Atlantic
2 months ago

The Rarest of All Diseases Are Becoming Treatable

For a decade after its discovery, CRISPR gene editing was stuck on the cusp of transforming medicine. Then, in 2023, scientists started using it on sickle-cell disease, and Victoria Gray, a patient who lived with constant pain-like lightning inside her body, she has said -got the first-ever FDA-approved CRISPR gene-editing treatment. Her symptoms vanished; so did virtually everyone else's in the clinical trial she was a part of.
Science
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