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Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector.

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

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Gene therapy in a patient with sickle cell disease

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Stem-Cell Gene Therapy for the Wiskott–Aldrich Syndrome

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Genomic safe harbors permit high β-globin transgene expression in thalassemia induced pluripotent stem cells

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Distinct types of tumor-initiating cells form human colon cancer tumors and metastases

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Phase 2 gene therapy trial of an anti-HIV ribozyme in autologous CD34+ cells

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Quantitative evaluation of chromosomal rearrangements in gene-edited human stem cells by CAST-Seq

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TALEN-based Gene Correction for Epidermolysis Bullosa

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Evaluating the state of the science for adeno-associated virus integration: An integrated perspective

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Acute Myeloid Leukemia Case after Gene Therapy for Sickle Cell Disease

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A largely random AAV integration profile after LPLD gene therapy

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Delivery and Specificity of CRISPR/Cas9 Genome Editing Technologies for Human Gene Therapy

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

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

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Comprehensive genomic access to vector integration in clinical gene therapy

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Development of β-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease

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Successful engraftment of gene-corrected hematopoietic stem cells in non-conditioned patients with Fanconi anemia

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Genome-wide Profiling Reveals Remarkable Parallels Between Insertion Site Selection Properties of the MLV Retrovirus and the piggyBac Transposon in Primary Human CD4+ T Cells

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Liver-directed lentiviral gene therapy in a dog model of hemophilia B

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Hematologic Cancer after Gene Therapy for Cerebral Adrenoleukodystrophy

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Preclinical Evaluation of Efficacy and Safety of an Improved Lentiviral Vector for the Treatment of &amp;#946;-Thalassemia and Sickle Cell Disease

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Lentiviral Hematopoietic Cell Gene Therapy for X-Linked Adrenoleukodystrophy

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Retroviral Integrations in Gene Therapy Trials

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Tracking genetically engineered lymphocytes long-term reveals the dynamics of T cell immunological memory

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High-resolution analysis of the human T-cell receptor repertoire

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Thymus-autonomous T cell development in the absence of progenitor import

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Integration Frequency and Intermolecular Recombination of rAAV Vectors in Non-human Primate Skeletal Muscle and Liver

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Spatially clustered loci with multiple enhancers are frequent targets of HIV-1 integration

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Gene Therapy for X-Linked Severe Combined Immunodeficiency: Where Do We Stand?

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Gene therapy for severe combined immunodeficiencies and beyond

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Lentiviral vector–based insertional mutagenesis identifies genes associated with liver cancer

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Long-term correction of hemophilia B using adenoviral delivery of CRISPR/Cas9

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

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Pre-existing Functional Heterogeneity of Tumorigenic Compartment as the Origin of Chemoresistance in Pancreatic Tumors

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