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Predicting genotoxicity of viral vectors for stem cell gene therapy using gene expression-based machine learning
Correction of pathology in mice displaying Gaucher disease type 1 by a clinically-applicable lentiviral vector
Successful preclinical development of gene therapy for recombinase-activating gene-1-deficient SCID
New molecular surrogate assay for genotoxicity assessment (SAGA)
Lentiviral gene therapy using cellular promoters cures type 1 Gaucher disease in mice
Successful Correction of Rag1 Severe Combined Immunedefciency at Clinically Relevant Vector Copy Numbers
Finding levels of RAG1 expression that will correct RAG1 Severe Combined Immunodeficiency
Successful RAG1-SCID gene therapy depends on the level of RAG1 expression
Self-Inactivating Lentiviral Vectors for Correction of Rag1 Severe Combined Immunodeficiency
Sleeping Beauty transposon-based system for cellular reprogramming and targeted gene insertion in induced pluripotent stem cells
Self-Inactivating Lentiviral Vectors for Correction of Rag1 Severe Combined Immunodeficiency
SELF-INACTIVATING LENTIVIRAL VECTORS FOR CORRECTION OF RAG1 SEVERE COMBINED IMMUNODEFICIENCY
Correction of murine Rag1 deficiency by self-inactivating lentiviral vector-mediated gene transfer
Correction of murine Rag1 deficiency by self-inactivating lentiviral vector-mediated gene transfer
Correction of B-cell development in Btk-deficient mice using lentiviral vectors with codon-optimized human BTK