Lentiviral gene therapy for X-linked chronic granulomatous disease

Allogeneic hematopoietic cell transplantation in patients with GATA2 deficiency—a case report and comprehensive review of the literature

(2018) Alexander Simonis et al.   ANNALS OF HEMATOLOGY

X-linked carriers of chronic granulomatous disease: Illness, lyonization, and stability

(2018) Beatriz E. Marciano et al.   JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY

Neurological complications of hematopoietic cell transplantation in children and adults

(2018) AdrianaOctaviana Dulamea et al.   Neural Regeneration Research

Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia

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

Non-Clinical Efficacy and Safety Studies on G1XCGD, a Lentiviral Vector for Ex Vivo Gene Therapy of X-Linked Chronic Granulomatous Disease

(2018) Christian Brendel et al.   Human Gene Therapy Clinical Development

Dynamics of genetically engineered hematopoietic stem and progenitor cells after autologous transplantation in humans

(2018) Serena Scala et al.   NATURE MEDICINE

Allogeneic Reduced-Intensity Hematopoietic Stem Cell Transplantation for Chronic Granulomatous Disease: a Single-Center Prospective Trial

(2017) Mark Parta et al.   JOURNAL OF CLINICAL IMMUNOLOGY

Gene Therapy in a Patient with Sickle Cell Disease

(2017) Jean-Antoine Ribeil et al.   NEW ENGLAND JOURNAL OF MEDICINE

INSPIIRED: A Pipeline for Quantitative Analysis of Sites of New DNA Integration in Cellular Genomes

(2017) Eric Sherman et al.   Molecular Therapy-Methods & Clinical Development

INSPIIRED: Quantification and Visualization Tools for Analyzing Integration Site Distributions

(2017) Charles C. Berry et al.   Molecular Therapy-Methods & Clinical Development

In Vivo Tracking of Human Hematopoiesis Reveals Patterns of Clonal Dynamics during Early and Steady-State Reconstitution Phases

(2016) Luca Biasco et al.   Cell Stem Cell

Hyperinflammation in patients with chronic granulomatous disease leads to impairment of hematopoietic stem cell functions

(2016) Maren Weisser et al.   JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY

In vivo tracking of T cells in humans unveils decade-long survival and activity of genetically modified T memory stem cells

(2015) Luca Biasco et al.   Science Translational Medicine

Reduced-intensity conditioning and HLA-matched haemopoietic stem-cell transplantation in patients with chronic granulomatous disease: a prospective multicentre study

(2013) Tayfun Güngör et al.   LANCET

Estimating abundances of retroviral insertion sites from DNA fragment length data

(2012) Charles C. Berry et al.   BIOINFORMATICS

Chronic Granulomatous Disease

(2012) Steven M. Holland   HEMATOLOGY-ONCOLOGY CLINICS OF NORTH AMERICA

Bis-SNP: Combined DNA methylation and SNP calling for Bisulfite-seq data

(2012) Yaping Liu et al.   GENOME BIOLOGY

Bismark: a flexible aligner and methylation caller for Bisulfite-Seq applications

(2011) Felix Krueger et al.   BIOINFORMATICS

A framework for variation discovery and genotyping using next-generation DNA sequencing data

(2011) Mark A DePristo et al.   NATURE GENETICS

The Genome Analysis Toolkit: A MapReduce framework for analyzing next-generation DNA sequencing data

(2010) A. McKenna et al.   GENOME RESEARCH

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

Gene Therapy of Chronic Granulomatous Disease: The Engraftment Dilemma

(2010) Manuel Grez et al.   MOLECULAR THERAPY

Biochemical Correction of X-CGD by a Novel Chimeric Promoter Regulating High Levels of Transgene Expression in Myeloid Cells

(2010) Giorgia Santilli et al.   MOLECULAR THERAPY

Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease

(2010) Stefan Stein et al.   NATURE MEDICINE

Residual NADPH Oxidase and Survival in Chronic Granulomatous Disease

(2010) Douglas B. Kuhns et al.   NEW ENGLAND JOURNAL OF MEDICINE

Retrovirus gene therapy for X-linked chronic granulomatous disease can achieve stable long-term correction of oxidase activity in peripheral blood neutrophils

(2009) E. M. Kang et al.   BLOOD

Validation of a mutated PRE sequence allowing high and sustained transgene expression while abrogating WHV-X protein synthesis: application to the gene therapy of WAS

(2009) M A Zanta-Boussif et al.   GENE THERAPY

Ultrafast and memory-efficient alignment of short DNA sequences to the human genome

(2009) Ben Langmead et al.   GENOME BIOLOGY