Пасемноген глепарвовек

Prop. INN (наименование, предложенное ВОЗ)

2913126-89-1

recombinant non-replicating adeno-associated virus serotype 9 (AAV9) vector, encoding a codon-optimised human survival of motor neuron 1 (SMN1) gene, under control of the cytomegalovirus (CMV) enhancer/promoter and terminated by a human growth hormone polyadenylation signal, flanked by AAV2 inverted terminal repeats. A human beta-globin intron has been inserted between the promoter and the SMN1 coding region. The virus vector also contains mutations in the viral capsid protein VP1 (F422W and Y446F)

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