Mount Sinai Hospital;The Sydney Children's Hospitals Network (Randwick and Westmead)(Incorporating the Royal Alexandra;Children's Medical Research Institute
发明人:
Ian Alexander,Sharon Cunningham,Andras Nagy
申请号：
US15127743
公开号：
US20170216456A1
申请日:
2015.03.23
申请国别(地区):
US
年份:
2017
代理人:
摘要：
Provided herein are methods for facilitating or inducing stable transgene integration and expression in a proliferating cell, comprising administering to the cell (i) a recombinant AAV (rAAV) vector comprising the transgene flanked by transposon-derived inverted terminal repeat sequences, which sequences are in turn flanked by AAV-derived inverted terminal repeat regions, and (ii) a source of a transposase that recognises said transposon-derived inverted terminal repeat sequences and directs the genomic integration of the transgene into the genome of the proliferating cell. Also provided are methods and transgene delivery systems for the treatment or prevention of diseases affecting, associated with or characterised by proliferating cells, including paediatric liver diseases, bone marrow diseases and cancer.
