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Di laman Onasemnogen abeparvovek:
"SMA ialah gangguan neuron-otot yang disebabkan oleh mutasi dalam gen SMN1, yang membawa kepada kekurangan protein SMN, protein yang diperlukan demi kelangsungan neuron motor. Onasemnogen abeparvovek ialah ubat biologi yang terdiri daripada kapsid virus AAV9 yang mengandungi transgen SMN1 bersama dengan promoter sintetik. Selepas pentadbiran, vektor virus AAV9 menyampaikan transgen SMN1 kepada neuron motor yang terjejas, di mana ia membawa kepada peningkatan protein SMN.[perlu rujukan]
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