Gene therapy for Wiskott-Aldrich syndrome--long-term efficacy and genotoxicity.
about
A Broad Overview and Review of CRISPR-Cas Technology and Stem CellsAdvances of gene therapy for primary immunodeficienciesClinical development of gene therapy: results and lessons from recent successesIn utero stem cell transplantation and gene therapy: rationale, history, and recent advances toward clinical applicationRecent advances in treatment of severe primary immunodeficienciesProgress and prospects for engineered T cell therapiesUnraveling the repertoire in wiskott-Aldrich syndromePSMA-specific CAR-engineered T cells eradicate disseminated prostate cancer in preclinical modelsAdvantages and applications of CAR-expressing natural killer cellsRetroviral DNA IntegrationCharting a clear path: the ASGCT Standardized Pathways ConferenceGene Therapy for X-Linked Severe Combined Immunodeficiency: Where Do We Stand?Diagnosis and treatment of inherited thrombocytopenias.Assessment of Immature Platelet Fraction in the Diagnosis of Wiskott-Aldrich Syndrome.Current and emerging treatment options for Wiskott-Aldrich syndrome.Effects of eltrombopag on platelet count and platelet activation in Wiskott-Aldrich syndrome/X-linked thrombocytopeniaCytoreductive conditioning intensity predicts clonal diversity in ADA-SCID retroviral gene therapy patients.Vector integration and tumorigenesis.Single Cell-Based Vector Tracing in Patients with ADA-SCID Treated with Stem Cell Gene Therapy.Succession of transiently active tumor-initiating cell clones in human pancreatic cancer xenografts.Gene Therapy 2017: Progress and Future Directions.Platelets: still a therapeutical target for haemostatic disorders.Adoptive T-cell therapy: adverse events and safety switches.Quantitative shearing linear amplification polymerase chain reaction: an improved method for quantifying lentiviral vector insertion sites in transplanted hematopoietic cell systemsCell cycle status of CD34(+) hemopoietic stem cells determines lentiviral integration in actively transcribed and development-related genes.Clinical applications of gene therapy for primary immunodeficiencies.What has happened in the last 50 years in immunology.adLIMS: a customized open source software that allows bridging clinical and basic molecular research studiesGenetic barcode sequencing for screening altered population dynamics of hematopoietic stem cells transduced with lentivirusGeneration of a lentiviral vector producer cell clone for human Wiskott-Aldrich syndrome gene therapy.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.Gamma-Retrovirus Integration Marks Cell Type-Specific Cancer Genes: A Novel Profiling Tool in Cancer GenomicsGene activity in primary T cells infected with HIV89.6: intron retention and induction of genomic repeats.Towards a Safer, More Randomized Lentiviral Vector Integration Profile Exploring Artificial LEDGF Chimeras.INSPIIRED: A Pipeline for Quantitative Analysis of Sites of New DNA Integration in Cellular GenomesGene therapy for Wiskott-Aldrich Syndrome-Long-term reconstitution and clinical benefits, but increased risk for leukemogenesis.Multilineage polyclonal engraftment of Cal-1 gene-modified cells and in vivo selection after SHIV infection in a nonhuman primate model of AIDSGene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the β(A(T87Q))-Globin Gene.Homology-driven genome editing in hematopoietic stem and progenitor cells using ZFN mRNA and AAV6 donors.Evaluating the Safety of Retroviral Vectors Based on Insertional Oncogene Activation and Blocked Differentiation in Cultured Thymocytes.
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P2860
Gene therapy for Wiskott-Aldrich syndrome--long-term efficacy and genotoxicity.
description
2014 nî lūn-bûn
@nan
2014 թուականի Մարտին հրատարակուած գիտական յօդուած
@hyw
2014 թվականի մարտին հրատարակված գիտական հոդված
@hy
2014年の論文
@ja
2014年論文
@yue
2014年論文
@zh-hant
2014年論文
@zh-hk
2014年論文
@zh-mo
2014年論文
@zh-tw
2014年论文
@wuu
name
Gene therapy for Wiskott-Aldrich syndrome--long-term efficacy and genotoxicity.
@ast
Gene therapy for Wiskott-Aldrich syndrome--long-term efficacy and genotoxicity.
@en
type
label
Gene therapy for Wiskott-Aldrich syndrome--long-term efficacy and genotoxicity.
@ast
Gene therapy for Wiskott-Aldrich syndrome--long-term efficacy and genotoxicity.
@en
prefLabel
Gene therapy for Wiskott-Aldrich syndrome--long-term efficacy and genotoxicity.
@ast
Gene therapy for Wiskott-Aldrich syndrome--long-term efficacy and genotoxicity.
@en
P2093
P2860
P50
P1476
Gene therapy for Wiskott-Aldrich syndrome--long-term efficacy and genotoxicity.
@en
P2093
Adrian Schwarzer
Anna Paruzynski
Chris Fraser
Christoph Klein
Doris Steinemann
Gudrun Göhring
Hanno Glimm
Irina Kondratenko
Kaan Boztug
Klaus Kühlcke
P2860
P304
P356
10.1126/SCITRANSLMED.3007280
P407
P577
2014-03-01T00:00:00Z