Ontology highlight
ABSTRACT:
SUBMITTER: Park CY
PROVIDER: S-EPMC4999461 | biostudies-literature | 2016 Sep
REPOSITORIES: biostudies-literature
Park Chul-Yong CY Lee Dongjin R DR Sung Jin Jea JJ Kim Dong-Wook DW
Human genetics 20160629 9
Hemophilia is caused by various mutations in blood coagulation factor genes, including factor VIII (FVIII) and factor IX (FIX), that encode key proteins in the blood clotting pathway. Although the addition of therapeutic genes or infusion of clotting factors may be used to remedy hemophilia's symptoms, no permanent cure for the disease exists. Moreover, patients often develop neutralizing antibodies or experience adverse effects that limit the therapy's benefits. However, targeted gene therapy i ...[more]