Haplomics, Inc.;The Regents of the University of California;The United States Government as respresented by the Department of Veterans Affairs
发明人:
Vincent LATERZA,Tommy E. HOWARD
申请号：
US16396326
公开号：
US20190351073A1
申请日:
2019.04.26
申请国别(地区):
US
年份:
2019
代理人:
摘要：
Methods of treating hemophilia A in a subject with an F8 gene mutation, wherein the F8 gene is repaired and the resultant repaired gene, upon expression, confers improved coagulation functionality to the encoded FVIII protein of the subject compared to the non-repaired F8 gene. The invention also includes methods of inducing immune tolerance to a FVIII replacement product ((r)FVIII) in a subject having a FVIII deficiency, wherein the F8 gene mutation is repaired and the repaired gene, upon expression, provides for the induction of immune tolerance to an administered replacement FVIII protein product. The invention also includes isolated nucleic acids, vectors, recombinant viruses, cells, and pharmaceutical compositions to repair the F8 gene.
