High-Efficiency Transduction of Primary Human Hematopoietic Stem Cells and Erythroid Lineage-Restricted Expression by Optimized AAV6 Serotype Vectors In Vitro and in a Murine Xenograft Model In Vivo

Optimizing the transduction efficiency of capsid-modified AAV6 serotype vectors in primary human hematopoietic stem cells in vitro and in a xenograft mouse model in vivo

(2013) Liujiang Song et al.   CYTOTHERAPY

Human Galectin 3 Binding Protein Interacts with Recombinant Adeno-Associated Virus Type 6

(2012) J. Denard et al.   JOURNAL OF VIROLOGY

High-efficiency transduction of human monocyte-derived dendritic cells by capsid-modified recombinant AAV2 vectors

(2012) George V. Aslanidi et al.   VACCINE

Enhanced gene delivery to the neonatal retina through systemic administration of tyrosine-mutated AAV9

(2011) D Dalkara et al.   GENE THERAPY

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

(2011) Federico Mingozzi et al.   NATURE REVIEWS GENETICS

Engraftment of human hematopoietic stem cells is more efficient in female NOD/SCID/IL-2Rgc-null recipients

(2010) F. Notta et al.   BLOOD

Therapeutic levels of fetal hemoglobin in erythroid progeny of  -thalassemic CD34+ cells after lentiviral vector-mediated gene transfer

(2010) A. Wilber et al.   BLOOD

Comparison of human cord blood engraftment between immunocompromised mouse strains

(2010) S. P. McDermott et al.   BLOOD

Gene transfer into human cord blood−derived CD34+ cells by adeno-associated viral vectors

(2010) Natascha K. Schuhmann et al.   EXPERIMENTAL HEMATOLOGY

Optimized Transduction of Human Monocyte-Derived Dendritic Cells by Recombinant Adeno-Associated Virus Serotype 6

(2010) James E. Ussher et al.   HUMAN GENE THERAPY

Adeno-Associated Virus Serotype 6 Capsid Tyrosine-to-Phenylalanine Mutations Improve Gene Transfer to Skeletal Muscle

(2010) Chunping Qiao et al.   HUMAN GENE THERAPY

Enhanced Long-Term Transduction and Multilineage Engraftment of Human Hematopoietic Stem Cells Transduced with Tyrosine-Modified Recombinant Adeno-Associated Virus Serotype 2

(2010) M. Ariel Kauss et al.   HUMAN GENE THERAPY

Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia

(2010) Marina Cavazzana-Calvo et al.   NATURE

Epidermal growth factor receptor is a co-receptor for adeno-associated virus serotype 6

(2010) Melodie L Weller et al.   NATURE MEDICINE

Pseudotyped recombinant adeno-associated viral vectors mediate efficient gene transfer into primary human CD34+ peripheral blood progenitor cells

(2009) Marlon R. Veldwijk et al.   CYTOTHERAPY

Gaussia luciferase reporter assay for monitoring biological processes in culture and in vivo

(2009) Bakhos A Tannous   Nature Protocols

Clinical gene therapy using recombinant adeno-associated virus vectors

(2008) C Mueller et al.   GENE THERAPY

Recombinant Self-Complementary Adeno-Associated Virus Serotype Vector-Mediated Hematopoietic Stem Cell Transduction and Lineage-Restricted, Long-Term Transgene Expression in a Murine Serial Bone Marrow Transplantation Model

(2008) Njeri Maina et al.   HUMAN GENE THERAPY

Optimization of Recombinant Adeno-Associated Viral Vectors for Human β-Globin Gene Transfer and Transgene Expression

(2008) Njeri Maina et al.   HUMAN GENE THERAPY

Treatment of Leber Congenital Amaurosis Due to RPE65 Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector: Short-Term Results of a Phase I Trial

(2008) William W. Hauswirth et al.   HUMAN GENE THERAPY

A secreted luciferase for ex vivo monitoring of in vivo processes

(2008) Thomas Wurdinger et al.   NATURE METHODS

Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis

(2008) Albert M. Maguire et al.   NEW ENGLAND JOURNAL OF MEDICINE

Effect of Gene Therapy on Visual Function in Leber's Congenital Amaurosis

(2008) James W.B. Bainbridge et al.   NEW ENGLAND JOURNAL OF MEDICINE

Next generation of adeno-associated virus 2 vectors: Point mutations in tyrosines lead to high-efficiency transduction at lower doses

(2008) L. Zhong et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics

(2008) A. V. Cideciyan et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA