Ларупаретиген зовапарвовек

Rec. INN (наименование, зарегистрированное ВОЗ)

USAN (наименование, принятое к употреблению в США)

2425817-08-7

recombinant, non-replicating adeno-associated virus serotype 2 (AAV2) vector, in which the capsid contains three tyrosine (Y) to phenylalanine (F) mutations (AAV2tYF), encoding codon-optimised human retinitis pigmentosa GTPase regulator (RPGR) isoform 6 (RPGR-ORF15), under control of the human G protein- coupled receptor kinase 1 promoter, preceded by a synthetic splice donor/splice acceptor, terminated with the simian virus 40 (SV40) polyadenylation (polyA) sequence, and flanked by AAV2 inverted terminal repeats (ITRs).

Поделиться этой страницей