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  • Special - Rare Disease Day Info Episode Special - Rare Disease Day Info Episode
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zakuromochi

Science fact: Many current (as of 2020) effective gene therapies use special (inactive) viruses containing a correct form of a gene that will replace a bad or missing gene in a patient (Example: Zolgensma for spinal muscular atrophy - https://www.curesma.org/zolgensma/).

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