AHLERS, Jeffrey,BARTLETT, Jeffrey,LEE, Chi-Lin,RINGPIS, Gene-Errol Eugenio,SYMONDS, Geoffrey Phillip,YAN, Ming
申请号：
AU2018302098
公开号：
AU2018302098A1
申请日:
2018.07.17
申请国别(地区):
AU
年份:
2020
代理人:
摘要：
The present disclosure provides expression vectors comprising at least two nucleic acid sequences, namely a nucleic acid sequence encoding an anti-HPRT RNAi, and a nucleic acid sequence encoding a gamma globin gene. In some embodiments, the viral vector is a self-inactivating lentiviral vector. In some embodiments, the gamma-globin gene is used to genetically correct sickle cell disease or β-thalassemia or to reduce symptoms thereof.
