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並前往https://www1.racgp.org.au/ajgp/2022/january-february/spinal-muscular-atrophy-therapeutic-landscape

Spinal muscular atrophy therapeutic landscape - RACGP

SMN1 gene replacement therapy, using an adeno-associated virus capsid to supply a functional SMN1 gene to motor neuron cells. Single dose of virus vector given ...

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