Optimizing lentiviral vector transduction of hematopoietic stem cells for gene therapy

Gene therapy targeting haematopoietic stem cells for inherited diseases: progress and challenges

(2019) Marina Cavazzana et al.   NATURE REVIEWS DRUG DISCOVERY

Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1

(2019) Ewelina Mamcarz et al.   NEW ENGLAND JOURNAL OF MEDICINE

High-Efficiency Lentiviral Transduction of Human CD34+ Cells in High-Density Culture with Poloxamer and Prostaglandin E2

(2019) Naoya Uchida et al.   Molecular Therapy-Methods & Clinical Development

PGE2 and Poloxamer Synperonic F108 Enhance Transduction of Human HSPCs with a β-Globin Lentiviral Vector

(2019) Katelyn E. Masiuk et al.   Molecular Therapy-Methods & Clinical Development

Resveratrol trimer enhances gene delivery to hematopoietic stem cells by reducing antiviral restriction at endosomes

(2019) Stosh Ozog et al.   BLOOD

Enhancing Lentiviral and Alpharetroviral Transduction of Human Hematopoietic Stem Cells for Clinical Application

(2019) Juliane W. Schott et al.   Molecular Therapy-Methods & Clinical Development

Production of Lentiviral Vectors Using Suspension Cells Grown in Serum-free Media

(2019) Matthew Bauler et al.   Molecular Therapy-Methods & Clinical Development

Prostaglandin E 2 Increases Lentiviral Vector Transduction Efficiency of Adult Human Hematopoietic Stem and Progenitor Cells

(2018) Garrett C. Heffner et al.   MOLECULAR THERAPY

Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia

(2018) Alexis A. Thompson et al.   NEW ENGLAND JOURNAL OF MEDICINE

Improving Lentiviral Transduction of CD34+ Hematopoietic Stem and Progenitor Cells

(2018) Ilona Hauber et al.   Human Gene Therapy Methods

Cyclosporine H Overcomes Innate Immune Restrictions to Improve Lentiviral Transduction and Gene Editing In Human Hematopoietic Stem Cells

(2018) Carolina Petrillo et al.   Cell Stem Cell

Safe and Effective Gene Therapy for Murine Wiskott-Aldrich Syndrome Using an Insulated Lentiviral Vector

(2017) Swati Singh et al.   Molecular Therapy-Methods & Clinical Development

Lentiviral vectors, two decades later

(2016) L. Naldini et al.   SCIENCE

Lentiviral hematopoietic stem cell gene therapy for X-linked severe combined immunodeficiency

(2016) Suk See De Ravin et al.   Science Translational Medicine

Improved Human Erythropoiesis and Platelet Formation in Humanized NSGW41 Mice

(2016) Susann Rahmig et al.   Stem Cell Reports

Cellular Innate Immunity and Restriction of Viral Infection: Implications for Lentiviral Gene Therapy in Human Hematopoietic Cells

(2015) Anna Kajaste-Rudnitski et al.   HUMAN GENE THERAPY

Outcomes Following Gene Therapy in Patients With Severe Wiskott-Aldrich Syndrome

(2015) Salima Hacein-Bey Abina et al.   JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

81. Development of a Second Generation Stable Lentiviral Packaging Cell Line in Support of Clinical Gene Transfer Protocols

(2015) Melissa Bonner et al.   MOLECULAR THERAPY

Cyclosporin A and Rapamycin Relieve Distinct Lentiviral Restriction Blocks in Hematopoietic Stem and Progenitor Cells

(2015) Carolina Petrillo et al.   MOLECULAR THERAPY

Quantitative Shearing Linear Amplification Polymerase Chain Reaction: An Improved Method for Quantifying Lentiviral Vector Insertion Sites in Transplanted Hematopoietic Cell Systems

(2015) Sheng Zhou et al.   Human Gene Therapy Methods

Nonirradiated NOD,B6.SCID Il2rγ−/− KitW41/W41 (NBSGW) Mice Support Multilineage Engraftment of Human Hematopoietic Cells

(2015) Brian E. McIntosh et al.   Stem Cell Reports

Rapamycin relieves lentiviral vector transduction resistance in human and mouse hematopoietic stem cells

(2014) C. X. Wang et al.   BLOOD

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

(2013) A. Aiuti et al.   SCIENCE

Poloxamer synperonic F108 improves cellular transduction with lentiviral vectors

(2012) Ines Höfig et al.   JOURNAL OF GENE MEDICINE

Preclinical Safety and Efficacy of Human CD34+ Cells Transduced With Lentiviral Vector for the Treatment of Wiskott-Aldrich Syndrome

(2012) Samantha Scaramuzza et al.   MOLECULAR THERAPY

Transduction of Human CD34+ Repopulating Cells with a Self-Inactivating Lentiviral Vector for SCID-X1 Produced at Clinical Scale by a Stable Cell Line

(2012) Michael R. Greene et al.   Human Gene Therapy Methods

Is normal hematopoiesis maintained solely by long-term multipotent stem cells?

(2011) M. Cavazzana-Calvo et al.   BLOOD

Efficient construction of producer cell lines for a SIN lentiviral vector for SCID-X1 gene therapy by concatemeric array transfection

(2009) R. E. Throm et al.   BLOOD

Towards a Clinically Relevant Lentiviral Transduction Protocol for Primary Human CD34+ Hematopoietic Stem/Progenitor Cells

(2009) Michelle Millington et al.   PLoS One

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

(2009) N. Cartier et al.   SCIENCE