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New approaches to gene and cell therapy for hemophilia
Author(s) -
Ohmori T.,
Mizukami H.,
Ozawa K.,
Sakata Y.,
Nishimura S.
Publication year - 2015
Publication title -
journal of thrombosis and haemostasis
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.947
H-Index - 178
eISSN - 1538-7836
pISSN - 1538-7933
DOI - 10.1111/jth.12926
Subject(s) - genetic enhancement , medicine , factor ix , coagulation , virus , antibody , vectors in gene therapy , clinical trial , adeno associated virus , vector (molecular biology) , immunology , gene , virology , recombinant dna , biology , genetics
Summary Hemophilia is considered suitable for gene therapy because it is caused by a single gene abnormality, and therapeutic coagulation factor levels may vary across a broad range. Recent success of hemophilia B gene therapy with an adeno‐associated virus ( AAV ) vector in a clinical trial showed the real prospect that, through gene therapy, a cure for hemophilia may become a reality. However, AAV ‐mediated gene therapy is not applicable to patients with hemophilia A at present, and neutralizing antibodies against AAV reduce the efficacy of AAV ‐mediated strategies. Because patients that benefit from AAV treatment (hemophilia B without neutralizing antibodies) are estimated to represent only 15% of total patients with hemophilia, the development of basic technologies for hemophilia A and those that result in higher therapeutic effects are critical. In this review, we present an outline of gene therapy methods for hemophilia, including the transition of technical developments thus far and our novel techniques.