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One or more keywords matched the following items that are connected to Schambach, Axel
Item TypeName
Academic Article Gene therapy: X-SCID transgene leukaemogenicity.
Academic Article Self-inactivating gammaretroviral vectors for gene therapy of X-linked severe combined immunodeficiency.
Academic Article Correction of murine SCID-X1 by lentiviral gene therapy using a codon-optimized IL2RG gene and minimal pretransplant conditioning.
Academic Article Development of novel efficient SIN vectors with improved safety features for Wiskott-Aldrich syndrome stem cell based gene therapy.
Academic Article Rescue of ATP7B function in hepatocyte-like cells from Wilson's disease induced pluripotent stem cells using gene therapy or the chaperone drug curcumin.
Academic Article Promoter competition for gene therapy of SCID-X1.
Academic Article Alpharetroviral vector-mediated gene therapy for X-CGD: functional correction and lack of aberrant splicing.
Academic Article Tumor cells escape suicide gene therapy by genetic and epigenetic instability.
Academic Article Cell and virus genetics at the roots of gene therapy, retrovirology, and hematopoietic stem cell biology: Wolfram Ostertag (1937-2010).
Academic Article From bench to bedside: preclinical evaluation of a self-inactivating gammaretroviral vector for the gene therapy of X-linked chronic granulomatous disease.
Academic Article Sustained knockdown of a disease-causing gene in patient-specific induced pluripotent stem cells using lentiviral vector-based gene therapy.
Academic Article Gene therapy on the move.
Academic Article Promoter and lineage independent anti-silencing activity of the A2 ubiquitous chromatin opening element for optimized human pluripotent stem cell-based gene therapy.
Academic Article Biosafety challenges for use of lentiviral vectors in gene therapy.
Academic Article Gene therapy for Wiskott-Aldrich syndrome--long-term efficacy and genotoxicity.
Academic Article Alpharetroviral vectors: from a cancer-causing agent to a useful tool for human gene therapy.
Academic Article Safety of gene therapy: new insights to a puzzling case.
Academic Article Modified lentiviral LTRs allow Flp recombinase-mediated cassette exchange and in vivo tracing of "factor-free" induced pluripotent stem cells.
Academic Article Gene therapy cures the anemia and lethal bone marrow failure in a mouse model of RPS19-deficient Diamond-Blackfan anemia.
Academic Article Successful RAG1-SCID gene therapy depends on the level of RAG1 expression.
Academic Article Lentiviral gene therapy using cellular promoters cures type 1 Gaucher disease in mice.
Academic Article Gene Insertion Into Genomic Safe Harbors for Human Gene Therapy.
Academic Article Safe and Efficient Gene Therapy for Pyruvate Kinase Deficiency.
Academic Article Retroviral Vectors for Cancer Gene Therapy.
Academic Article Eliminating HIV-1 Packaging Sequences from Lentiviral Vector Proviruses Enhances Safety and Expedites Gene Transfer for Gene Therapy.
Academic Article Integrating Vectors for Gene Therapy and Clonal Tracking of Engineered Hematopoiesis.
Academic Article Analyzing the Genotoxicity of Retroviral Vectors in Hematopoietic Cell Gene Therapy.
Academic Article Hematopoietic stem cell gene therapy for IFN?R1 deficiency protects mice from mycobacterial infections.
Academic Article T-cell gene therapy for perforin deficiency corrects cytotoxicity defects and prevents hemophagocytic lymphohistiocytosis manifestations.
Academic Article Non-Clinical Efficacy and Safety Studies on G1XCGD, a Lentiviral Vector for Ex Vivo Gene Therapy of X-Linked Chronic Granulomatous Disease.
Academic Article Preclinical Development of a Lentiviral Vector for Gene Therapy of X-Linked Severe Combined Immunodeficiency.
Academic Article Pre-clinical Development of a Lentiviral Vector Expressing the Anti-sickling ßAS3 Globin for Gene Therapy for Sickle Cell Disease.
Academic Article Hematopoietic Stem Cell-Targeted Neonatal Gene Therapy with a Clinically Applicable Lentiviral Vector Corrects Osteopetrosis in oc/oc Mice.
Academic Article Effective hematopoietic stem cell-based gene therapy in a murine model of hereditary pulmonary alveolar proteinosis.
Academic Article RETRACTED: Novel and safer self-inactivating vectors for gene therapy of Wiskott-Aldrich Syndrome
Academic Article Biosafety Studies of a Clinically Applicable Lentiviral Vector for the Gene Therapy of Artemis-SCID.
Academic Article Lentiviral gene therapy and vitamin B3 treatment enable granulocytic differentiation of G6PC3-deficient induced pluripotent stem cells.
Academic Article Preclinical Evaluation of a Novel Lentiviral Vector Driving Lineage-Specific BCL11A Knockdown for Sickle Cell Gene Therapy.
Academic Article Successful Preclinical Development of Gene Therapy for Recombinase-Activating Gene-1-Deficient SCID.
Academic Article Successful gene therapy of Diamond-Blackfan anemia in a mouse model and human CD34+ cord blood hematopoietic stem cells using a clinically applicable lentiviral vector.
Academic Article Gene therapy for infantile malignant osteopetrosis: review of pre-clinical research and proof-of-concept for phenotypic reversal.
Academic Article Preclinical Optimization and Safety Studies of a New Lentiviral Gene Therapy for p47phox-Deficient Chronic Granulomatous Disease.
Academic Article Retroviral gene therapy in Germany with a view on previous experience and future perspectives.
Academic Article Predicting genotoxicity of viral vectors for stem cell gene therapy using gene expression-based machine learning.
Academic Article Gene Therapy "Made in Germany": A Historical Perspective, Analysis of the Status Quo, and Recommendations for Action by the German Society for Gene Therapy.
Search Criteria
  • Gene Therapy
Funded by the NIH National Center for Advancing Translational Sciences through its Clinical and Translational Science Awards Program, grant number UL1TR002541.