Hyperfunctional coagulation factor IX improves the efficacy of gene therapy in hemophilic mice

Hepatocyte-targeted expression by integrase-defective lentiviral vectors induces antigen-specific tolerance in mice with low genotoxic risk

(2011) Janka Mátrai et al.   HEPATOLOGY

A MicroRNA-regulated and GP64-pseudotyped Lentiviral Vector Mediates Stable Expression of FVIII in a Murine Model of Hemophilia A

(2011) Hideto Matsui et al.   MOLECULAR THERAPY

Understanding the contribution of synonymous mutations to human disease

(2011) Zuben E. Sauna et al.   NATURE REVIEWS GENETICS

Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges

(2011) Federico Mingozzi et al.   NATURE REVIEWS GENETICS

Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B

(2011) Amit C. Nathwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

Recent Advances in Lentiviral Vector Development and Applications

(2010) Janka Mátrai et al.   MOLECULAR THERAPY

In vivo delivery of a microRNA-regulated transgene induces antigen-specific regulatory T cells and promotes immunologic tolerance

(2009) A. Annoni et al.   BLOOD

X-Linked Thrombophilia with a Mutant Factor IX (Factor IX Padua)

(2009) Paolo Simioni et al.   NEW ENGLAND JOURNAL OF MEDICINE

D-dimer antigen: current concepts and future prospects

(2008) S. S. Adam et al.   BLOOD

Long-term expression of murine activated factor VII is safe, but elevated levels cause premature mortality

(2008) Majed N. Aljamali et al.   JOURNAL OF CLINICAL INVESTIGATION