Robert Michael Kotin,Ozan Alkan,Annaliese Jones,Douglas Anthony Kerr,Ara Karl Malakian,Matthew John Simmons,Teresa L. Wright
申请号:
US16644568
公开号:
US20200283794A1
申请日:
2018.09.07
申请国别(地区):
US
年份:
2020
代理人:
摘要:
CeDNA vectors having linear and continuous structure can be produced in high yields and used for effective transfer and expression of a transgene. ceDNA vectors comprise an expression cassette and two different ITR sequences derived from AAV genomes in a specified order. Some ceDNA vectors provided herein further comprise cis-regulatory elements and provide high gene expression efficiencies. Further provided herein are methods and cell lines for reliable and efficient production of the linear, continuous and capsid-free DNA vectors.