Predicting genotoxicity of viral vectors for stem cell gene therapy using gene expression-based machine learning

Successful Preclinical Development of Gene Therapy for Recombinase-Activating Gene-1-Deficient SCID

(2020) Laura Garcia-Perez et al.   Molecular Therapy-Methods & Clinical Development

The cis-Regulatory Atlas of the Mouse Immune System

(2019) Hideyuki Yoshida et al.   CELL

Lentiviral Vector Promoter is Decisive for Aberrant Transcript Formation

(2017) Simone J. Scholz et al.   HUMAN GENE THERAPY

Lentivirus Mediated Correction of Artemis-Deficient Severe Combined Immunodeficiency

(2017) Divya Punwani et al.   HUMAN GENE THERAPY

Why Batch Effects Matter in Omics Data, and How to Avoid Them

(2017) Wilson Wen Bin Goh et al.   TRENDS IN BIOTECHNOLOGY

Engineering Next-Generation BET-Independent MLV Vectors for Safer Gene Therapy

(2017) Sara El Ashkar et al.   Molecular Therapy-Nucleic Acids

Lentivector Iterations and Pre-Clinical Scale-Up/Toxicity Testing: Targeting Mobilized CD34 + Cells for Correction of Fabry Disease

(2017) Ju Huang et al.   Molecular Therapy-Methods & Clinical Development

Update on the safety and efficacy of retroviral gene therapy for immunodeficiency due to adenosine deaminase deficiency

(2016) Maria Pia Cicalese et al.   BLOOD

Combining location-and-scale batch effect adjustment with data cleaning by latent factor adjustment

(2016) Roman Hornung et al.   BMC BIOINFORMATICS

MLL-AF9 Expression in Hematopoietic Stem Cells Drives a Highly Invasive AML Expressing EMT-Related Genes Linked to Poor Outcome

(2016) Vaia Stavropoulou et al.   CANCER CELL

Evaluating the Safety of Retroviral Vectors Based on Insertional Oncogene Activation and Blocked Differentiation in Cultured Thymocytes

(2016) Sheng Zhou et al.   MOLECULAR THERAPY

A 17-gene stemness score for rapid determination of risk in acute leukaemia

(2016) Stanley W. K. Ng et al.   NATURE

Safety of Gene Therapy: New Insights to a Puzzling Case

(2014) Michael Rothe et al.   CURRENT GENE THERAPY

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

Uncovering and Dissecting the Genotoxicity of Self-inactivating Lentiviral Vectors In Vivo

(2014) Daniela Cesana et al.   MOLECULAR THERAPY

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

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

BET-independent MLV-based Vectors Target Away From Promoters and Regulatory Elements

(2014) Sara El Ashkar et al.   Molecular Therapy-Nucleic Acids

Preclinical safety and efficacy of an anti–HIV-1 lentiviral vector containing a short hairpin RNA to CCR5 and the C46 fusion inhibitor

(2014) Orit Wolstein et al.   Molecular Therapy-Methods & Clinical Development

Biosafety Features of Lentiviral Vectors

(2013) Axel Schambach et al.   HUMAN GENE THERAPY

Activation of Evi1 inhibits cell cycle progression and differentiation of hematopoietic progenitor cells

(2012) O S Kustikova et al.   LEUKEMIA

An empirical assessment of validation practices for molecular classifiers

(2011) P. J. Castaldi et al.   BRIEFINGS IN BIOINFORMATICS

Hallmarks of Cancer: The Next Generation

(2011) Douglas Hanahan et al.   CELL

Stem cell gene expression programs influence clinical outcome in human leukemia

(2011) Kolja Eppert et al.   NATURE MEDICINE

Effect of the Internal Promoter on Insertional Gene Activation by Lentiviral Vectors with an Intact HIV Long Terminal Repeat

(2010) S. Knight et al.   JOURNAL OF VIROLOGY

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

Stem-Cell Gene Therapy for the Wiskott–Aldrich Syndrome

(2010) Kaan Boztug et al.   NEW ENGLAND JOURNAL OF MEDICINE

Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients

(2008) Steven J. Howe et al.   JOURNAL OF CLINICAL INVESTIGATION