发明名称 |
VARIANT AAV AND COMPOSITIONS, METHODS AND USES FOR GENE TRANSFER TO CELLS, ORGANS AND TISSUES |
摘要 |
The invention relates to adeno-associated virus (AAV) serotype AAV-Rh74 and related AAV vectors, and AAV-Rh74 and related AAV vector mediated gene transfer methods and uses. In particular, AAV-Rh74 and related AAV vectors target polynucleotides to cells, tissues or organs for expression (transcription) of genes encoding therapeutic proteins and peptides, and polynucleotides that function as or are transcribed into inhibitory nucleic acid sequences. |
申请公布号 |
SG11201600518W(A) |
申请公布日期 |
2016.02.26 |
申请号 |
SG11201600518W |
申请日期 |
2014.07.22 |
申请人 |
THE CHILDREN'S HOSPITAL OF PHILADELPHIA |
发明人 |
HIGH, KATHERINE A.;YAZICIOGLU, MUSTAFA N.;ANGUELA, XAVIER |
分类号 |
A61K48/00;C07H21/04;C07K14/015;C12N7/01;C12N15/864 |
主分类号 |
A61K48/00 |
代理机构 |
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代理人 |
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主权项 |
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地址 |
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