Lentiviral vector ALS20 yields high hemoglobin levels with low genomic integrations for treatment of beta-globinopathies

Resolution of Sickle Cell Disease Manifestations in Patients Treated with Lentiglobin Gene Therapy: Updated Results from the Phase 1/2 Hgb-206 Group C Study

(2020) Julie Kanter et al.   BLOOD

Northstar-3: Interim Results from a Phase 3 Study Evaluating Lentiglobin Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia and Either a β0 or IVS-I-110 Mutation at Both Alleles of the HBB Gene

(2020) Ashutosh Lal et al.   BLOOD

Retroviral Insertional Mutagenesis in Humans: Evidence for Four Genetic Mechanisms Promoting Expansion of Cell Clones

(2020) Frederic D. Bushman   MOLECULAR THERAPY

Lentiviral gene therapy for X-linked chronic granulomatous disease

(2020) Donald B. Kohn et al.   NATURE MEDICINE

Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent ß-thalassemia

(2019) Sarah Marktel et al.   NATURE MEDICINE

Sunbeam: an extensible pipeline for analyzing metagenomic sequencing experiments

(2019) Erik L. Clarke et al.   Microbiome

Northstar-2: Updated Safety and Efficacy Analysis of Lentiglobin Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia and Non-β0/β0 Genotypes

(2019) Alexis A. Thompson et al.   BLOOD

Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia

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

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

Forced chromatin looping raises fetal hemoglobin in adult sickle cells to higher levels than pharmacologic inducers

(2016) L. Breda et al.   BLOOD

Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the βA(T87Q)-GlobinGene

(2016) Olivier Negre et al.   HUMAN GENE THERAPY

Cell and Gene Therapy for the Beta-Thalassemias: Advances and Prospects

(2016) Jorge Mansilla-Soto 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

The human ankyrin 1 promoter insulator sustains gene expression in a β-globin lentiviral vector in hematopoietic stem cells

(2015) Zulema Romero et al.   Molecular Therapy-Methods & Clinical Development

Estimating abundances of retroviral insertion sites from DNA fragment length data

(2012) Charles C. Berry et al.   BIOINFORMATICS

Therapeutic Hemoglobin Levels after Gene Transfer in β-Thalassemia Mice and in Hematopoietic Cells of β-Thalassemia and Sickle Cells Disease Patients

(2012) Laura Breda et al.   PLoS One

Correction of β-thalassemia major by gene transfer in haematopoietic progenitors of pediatric patients

(2010) Emanuela Anna Roselli et al.   EMBO Molecular Medicine

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

(2010) Marina Cavazzana-Calvo et al.   NATURE

In vivo selection of genetically modified erythroblastic progenitors leads to long-term correction of  -thalassemia

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

β-Globin LCR and Intron Elements Cooperate and Direct Spatial Reorganization for Gene Therapy

(2008) Alla Buzina et al.   PLoS Genetics