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meindnoch
4 months ago
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Huntington's disease treated for first time
>Remember, there's no reversal agent or half-life elimination for such genetic treatments.
Another virus that removes/breaks the inserted gene?
vibrio
4 months ago
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The bane of most gene therapy research is lack of persistence. With how serious and morbid HD is, I’d guess patients will take their chances.
pas
4 months ago
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if I understand correctly it only injects a "gene cassette" which forms an episome (a plasmid-like thing, ie. just a ring of DNA outside the nucleus, just floating around in the cell), so it's non-integrating
https://youtu.be/q8HIubDJiMI?t=41
Den_VR
4 months ago
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Mentioning that might have just been Guardrails…
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Another virus that removes/breaks the inserted gene?