发明申请
WO2022061000A1 CLOSED-ENDED DNA VECTORS AND USES THEREOF FOR EXPRESSING PHENYLALANINE HYDROXYLASE (PAH)
审中-公开
基本信息:
- 专利标题： CLOSED-ENDED DNA VECTORS AND USES THEREOF FOR EXPRESSING PHENYLALANINE HYDROXYLASE (PAH)
- 申请号：PCT/US2021/050695 申请日：2021-09-16
- 公开(公告)号：WO2022061000A1 公开(公告)日：2022-03-24
- 发明人： LIU, Leah, Yu , MONDS, Russell , CHIOCCO, Matthew, James , SILVER, Nathaniel , SAMAYOA, Phillip , KERR, Douglas, Anthony , KEENAN, Jessica, Lynn
- 申请人： GENERATION BIO CO.
- 申请人地址： 301 Binney Street, 4th Floor
- 专利权人： GENERATION BIO CO.
- 当前专利权人： GENERATION BIO CO.
- 当前专利权人地址： 301 Binney Street, 4th Floor
- 代理机构： CLARKE, Marcie, B. et al.
- 优先权： US63/078,954 2020-09-16
- 主分类号： C12N9/02
- IPC分类号： C12N9/02 ; C12N15/85 ; A61K48/00
摘要：
Described herein are ceDNA vectors having linear and continuous structure for delivery and expression of a transgene. ceDNA vectors comprise an expression cassette flanked by two ITR sequences, where the expression cassette comprises a codon optimized nucleic acid sequence encoding a PAH protein, in combination with particular promoter sequences and cis-regulatory elements. Further provided herein are methods and cell lines for reliable gene expression of PAH protein in vitro, ex vivo and in vivo using the ceDNA vectors. Also provided herein are methods and compositions comprising ceDNA vectors useful for the expression of PAH protein in a cell, tissue or subject, and methods of treatment of diseases with said ceDNA vectors expressing PAH protein. Such PAH protein can be expressed for treating disease, e.g., Phenylketonuria (PKU).