公开(公告)号：US20120309816A1

公开(公告)日：2012-12-06

申请号：US13505879

申请日：2010-11-09

申请人： Tomas Björklund ,  Anders Björklund ,  Deniz Kirik

发明人： Tomas Björklund ,  Anders Björklund ,  Deniz Kirik

IPC分类号： A61K31/7088 ,  A61P25/00 ,  A61P25/16 ,  A61P25/32 ,  A61P25/18 ,  A61P9/12 ,  A61P25/30 ,  C12N15/864 ,  A61P25/24

CPC分类号： C12N7/00 ,  A61K48/00 ,  C12N9/0071 ,  C12N9/78 ,  C12N9/88 ,  C12N15/86 ,  C12N2750/00043 ,  C12N2750/14143 ,  C12N2830/008 ,  C12N2830/34 ,  C12N2830/40 ,  C12N2830/42 ,  C12N2830/48 ,  C12N2830/50 ,  C12N2830/60 ,  C12Y114/16002 ,  C12Y305/04016

摘要： The present invention relates to a one-vector expression system comprising a sequence encoding two polypeptides, such as tyrosine hydroxylase (TH) and GTP-cyclohydrolase 1 (GCH1). The two polypeptides can be should preferentially be expressed at a ratio between 3:1 and 15:1, such as between 3:1 and 7:1. The invention is useful in the treatment of catecholamine deficient disorders, such as dopamine deficient disorders including but not limited to Parkinson's Disease. Moreover, the present invention provides a method to deliver the vector construct in order to limit the increased production of the catecholamine to the cells in need thereof.

摘要翻译： 本发明涉及包含编码两种多肽（如酪氨酸羟化酶（TH））和GTP-环化水解酶1（GCH1）的序列的单载体表达系统。 这两种多肽可优先以3:1至15:1之间的比例，例如3:1至7:1之间的比例表达。 本发明可用于治疗儿茶酚胺缺乏症，如多巴胺缺乏症，包括但不限于帕金森病。 此外，本发明提供了递送载体构建体的方法，以限制将儿茶酚胺生产增加到有需要的细胞中。

公开(公告)号：US20210107947A1

公开(公告)日：2021-04-15

申请号：US16970004

申请日：2019-02-14

申请人： Tomas BJÖRKLUND ,  Marcus DAVIDSSON

发明人： Tomas BJÖRKLUND ,  Marcus DAVIDSSON

IPC分类号： C07K14/005 ,  C12N15/10 ,  C12N15/69 ,  C12N15/86

摘要： Methods for identifying polypeptides, e.g. derived from HSV pUL22 protein, which when displayed on a capsid confer a desired property to viral particles comprising such capsids, as well as methods for designing and manufacturing viral vectors and viral particles with improved properties. The identification method is based on a capsid library in which the capsid variant does not form part of the viral genome which contains a barcode and in which the barcode associated with the capsid variant is determined by sequencing before the viral vector is used. Thus, the prevalence of the capsid variant in a biological sample may be determined by sequencing of the barcode alone.