Platelet gene therapy corrects the hemophilic phenotype in immunocompromised hemophilia A mice transplanted with genetically manipulated human cord blood stem cells

Full reconstitution of human platelets in humanized mice after macrophage depletion

(2012) Z. Hu et al.   BLOOD

Recent Progress in Gene Therapy for Hemophilia

(2012) Marinee K. Chuah et al.   HUMAN GENE THERAPY

Lentivirus-mediated platelet gene therapy of murine hemophilia A with pre-existing anti-factor VIII immunity

(2012) E. L. KUETHER et al.   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

NOD/scid IL-2Rgnull mice: a preclinical model system to evaluate human dendritic cell-based vaccine strategies in vivo

(2012) Stefani Spranger et al.   Journal of Translational Medicine

An Improved Protocol for Efficient Engraftment in NOD/LTSZ-SCIDIL-2RγNULL Mice Allows HIV Replication and Development of Anti-HIV Immune Responses

(2012) Maneesh Singh et al.   PLoS One

Gene therapy for haemophilia: prospects and challenges to prevent or reverse inhibitor formation

(2011) David W. Scott et al.   BRITISH JOURNAL OF HAEMATOLOGY

Parallel use of by-passing agents in haemophilia with inhibitors: a critical review

(2011) Jørgen Ingerslev et al.   BRITISH JOURNAL OF HAEMATOLOGY

Current Options and New Developments in the Treatment of Haemophilia

(2011) Trisha Wong et al.   DRUGS

Gene therapy for haemophilia: a long and winding road

(2011) K. A. HIGH   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Factor IX ectopically expressed in platelets can be stored in  -granules and corrects the phenotype of hemophilia B mice

(2010) G. Zhang et al.   BLOOD

Targeting FVIII expression to endothelial cells regenerates a releasable pool of FVIII and restores hemostasis in a mouse model of hemophilia A

(2010) Q. Shi et al.   BLOOD

In vivo efficacy of platelet-delivered, high specific activity factor VIII variants

(2010) T. K. Greene et al.   BLOOD

Comparison of human cord blood engraftment between immunocompromised mouse strains

(2010) S. P. McDermott et al.   BLOOD

Human B Cell Development and Antibody Production in Humanized NOD/SCID/IL-2Rγnull (NSG) Mice Conditioned by Busulfan

(2010) Bongkum Choi et al.   JOURNAL OF CLINICAL IMMUNOLOGY

Genome-wide high-throughput integrome analyses by nrLAM-PCR and next-generation sequencing

(2010) Anna Paruzynski et al.   Nature Protocols

Human platelets produced in nonobese diabetic/severe combined immunodeficient (NOD/SCID) mice upon transplantation of human cord blood CD34+ cells are functionally active in an ex vivo flow model of thrombosis

(2009) I. I. Salles et al.   BLOOD

Syngeneic transplantation of hematopoietic stem cells that are genetically modified to express factor VIII in platelets restores hemostasis to hemophilia A mice with preexisting FVIII immunity

(2008) Q. Shi et al.   BLOOD

Busulfan Produces Efficient Human Cell Engraftment in NOD/LtSz-Scid IL2RγNullMice

(2008) Jun Hayakawa et al.   STEM CELLS

Optimized Lentiviral Transduction of Mouse Bone Marrow-Derived Mesenchymal Stem Cells

(2008) David M. Ricks et al.   STEM CELLS AND DEVELOPMENT