High-Efficiency Transduction of Primary Human Hematopoietic Stem/Progenitor Cells by AAV6 Vectors: Strategies for Overcoming Donor-Variation and Implications in Genome Editing

Adeno-Associated Virus: The Naturally Occurring Virus Versus the Recombinant Vector

(2016) Arun Srivastava   HUMAN GENE THERAPY

Targeted gene addition in human CD34+ hematopoietic cells for correction of X-linked chronic granulomatous disease

(2016) Suk See De Ravin et al.   NATURE BIOTECHNOLOGY

CRISPR genome engineering and viral gene delivery: A case of mutual attraction

(2015) Florian Schmidt et al.   Biotechnology Journal

Homology-driven genome editing in hematopoietic stem and progenitor cells using ZFN mRNA and AAV6 donors

(2015) Jianbin Wang et al.   NATURE BIOTECHNOLOGY

Chemically modified guide RNAs enhance CRISPR-Cas genome editing in human primary cells

(2015) Ayal Hendel et al.   NATURE BIOTECHNOLOGY

Efficient modification of CCR5 in primary human hematopoietic cells using a megaTAL nuclease and AAV donor template

(2015) Blythe D. Sather et al.   Science Translational Medicine

CRISPR genome engineering and viral gene delivery: A case of mutual attraction

(2015) Florian Schmidt et al.   Biotechnology Journal

Preclinical Evaluation of Efficacy and Safety of an Improved Lentiviral Vector for the Treatment of β-Thalassemia and Sickle Cell Disease

(2014) Olivier Negre et al.   CURRENT GENE THERAPY

High-throughput screening identifies compounds that enhance lentiviral transduction

(2014) J M Johnston et al.   GENE THERAPY

Gene Transfer Properties and Structural Modeling of Human Stem Cell-derived AAV

(2014) Laura J Smith et al.   MOLECULAR THERAPY

Gene therapy: Editorial control

(2014) Katharine Gammon   NATURE

Gene Therapy for Wiskott-Aldrich Syndrome--Long-Term Efficacy and Genotoxicity

(2014) C. J. Braun et al.   Science Translational Medicine

Efficient Production of Dual Recombinant Adeno-Associated Viral Vectors for Factor VIII Delivery

(2014) Qizhao Wang et al.   Human Gene Therapy Methods

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

Integrase-defective Lentiviral Vectors as a Delivery Platform for Targeted Modification of Adenosine Deaminase Locus

(2013) Alok V Joglekar et al.   MOLECULAR THERAPY

Genomic Editing of the HIV-1 Coreceptor CCR5 in Adult Hematopoietic Stem and Progenitor Cells Using Zinc Finger Nucleases

(2013) Lijing Li et al.   MOLECULAR THERAPY

A concept of eliminating nonhomologous recombination for scalable and safe AAV vector generation for human gene therapy

(2013) Biao Dong et al.   NUCLEIC ACIDS RESEARCH

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

(2013) Liujiang Song et al.   PLoS One

Optimization of the Capsid of Recombinant Adeno-Associated Virus 2 (AAV2) Vectors: The Final Threshold?

(2013) George V. Aslanidi et al.   PLoS One

Lentiviral Hematopoietic Stem Cell Gene Therapy in Patients with Wiskott-Aldrich Syndrome

(2013) A. Aiuti et al.   SCIENCE

Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy

(2013) A. Biffi et al.   SCIENCE

ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering

(2013) Thomas Gaj et al.   TRENDS IN BIOTECHNOLOGY

A Simple Method to Increase the Transduction Efficiency of Single-Stranded Adeno-Associated Virus Vectors In Vitro and In Vivo

(2011) Wenqin Ma 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

Large-Scale Manufacture and Characterization of a Lentiviral Vector Produced for Clinical Ex Vivo Gene Therapy Application

(2010) Otto-Wilhelm Merten et al.   HUMAN GENE THERAPY

High-efficiency Transduction and Correction of Murine Hemophilia B Using AAV2 Vectors Devoid of Multiple Surface-exposed Tyrosines

(2010) David M Markusic et al.   MOLECULAR 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

Efficacy of Gene Therapy for X-Linked Severe Combined Immunodeficiency

(2010) Salima Hacein-Bey-Abina et al.   NEW ENGLAND JOURNAL OF MEDICINE

RNA-Based Gene Therapy for HIV with Lentiviral Vector-Modified CD34+ Cells in Patients Undergoing Transplantation for AIDS-Related Lymphoma

(2010) D. L. DiGiusto et al.   Science Translational Medicine

Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy

(2009) N. Cartier et al.   SCIENCE

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