ЛАБОРАТОРЬОС ДЕЛЬ ДР. ЭСТЕВЕ; С.А. (ES);УНИВЕРСИДАД АУТОНОМА ДЕ БАРСЕЛОНА (ES);ЛАБОРАТОРЬОС ДЕЛЬ ДР. ЭСТЕВЕ, С.А. (ES)
发明人:
БОШ ТУБЕРТ Фатима (ES),АЮСО ЛОПЕС Эдуард (ES),РУСО МАТИАС Альберт (ES)
申请号：
RU2013100176/10
公开号：
RU2013100176A
申请日:
2011.06.10
申请国别(地区):
RU
年份:
2014
代理人:
摘要：
1. The selected nucleotide sequence having at least 85% sequence identity with SEQ ID NO: 1, which encodes a protein of SEQ ID NO: 2.2. The isolated nucleotide sequence of claim 1, wherein said sequence is SEQ ID NO: 1.3. A genetic construct comprising a nucleotide sequence according to any one of claims 1 or 2.4. The genetic construct of claim 3, wherein said genetic construct is a vector. The genetic construct of claim 4, wherein said vector is an expression vector. The expression vector according to claim 5, wherein said vector is an adeno-associated vector. The expression vector according to claim 6, where the serotype is represented by serotype 1, 2, 5, 7, 8 or 9.8. The expression vector according to claim 7, where the serotype is represented by serotype 9.9. The expression vector of claim 6, comprising a CAG promoter operably linked to SEQ ID NO: 1.10. The expression vector AAV9-CAG-co-hu-SFMD according to claim 9, where the adeno-associated vector refers to serotype 9.11. The plasmid vector pAAV-CAG-co-hu-SFMD of claim 10 with a DSM registration number of 24817.12. The expression vector of claim 5, comprising the hAAT promoter operably linked to SEQ ID NO: 1.13. The plasmid vector pAAV-hAAT-co-hu-SFMD according to claim 12, wherein the adeno-associated vector is of serotype 8 or 9.14. The plasmid vector pAAV9-hAAT-co-hu-SFMD according to claim 13, wherein the adeno-associated vector is of serotype 9.15. A pharmaceutical composition comprising: a nucleotide sequence according to any one of claims 1 or 2； a genetic construct according to any one of claims 3 to 5； or an expression vector according to any one of claims 6-14.16. The pharmaceutical composition of claim 15 for parenteral administration, preferably for intravenous or intracranial1. Выделенная нуклеотидная последовательность, имеющая по меньшей мере 85% идентичность последовательности с SEQ ID NO:1, которая кодирует белок SEQ ID NO:2.2. Выделенная нуклеотидная последовательность по п.1, где указанная
