Validation of a mutated PRE sequence allowing high and sustained transgene expression while abrogating WHV-X protein synthesis: application to the gene therapy of WAS.
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Impact of age at administration, lysosomal storage, and transgene regulatory elements on AAV2/8-mediated rat liver transductionMyeloid/Microglial driven autologous hematopoietic stem cell gene therapy corrects a neuronopathic lysosomal diseaseEvidence for long-term efficacy and safety of gene therapy for Wiskott-Aldrich syndrome in preclinical models.Foamy virus vector-mediated gene correction of a mouse model of Wiskott-Aldrich syndrome.Increased functional protein expression using nucleotide sequence features enriched in highly expressed genes in zebrafish.Role of Cysteines in Stabilizing the Randomized Receptor Binding Domains within Feline Leukemia Virus Envelope ProteinsDesign of a single AAV vector for coexpression of TH and GCH1 to establish continuous DOPA synthesis in a rat model of Parkinson's diseaserAAV-compatible MiniPromoters for restricted expression in the brain and eye.Wiskott-Aldrich syndrome protein-deficient hematopoietic cells can be efficiently mobilized by granulocyte colony-stimulating factor.Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome.PAX6 MiniPromoters drive restricted expression from rAAV in the adult mouse retina.Enrichment of human hematopoietic stem/progenitor cells facilitates transduction for stem cell gene therapy.A Rev-Independent gag/pol Eliminates Detectable psi-gag Recombination in Lentiviral Vectors.Design of a novel integration-deficient lentivector technology that incorporates genetic and posttranslational elements to target human dendritic cells.Biosafety features of lentiviral vectorsAssessment of selected media supplements to improve F/HN lentiviral vector production yields.Genome editing for scalable production of alloantigen-free lentiviral vectors for in vivo gene therapy.Identification of liver-specific enhancer-promoter activity in the 3' untranslated region of the wild-type AAV2 genome.Improved microRNA suppression by WPRE-linked tough decoy microRNA sponges.Direct Intracranial Injection of AAVrh8 Encoding Monkey β-N-Acetylhexosaminidase Causes Neurotoxicity in the Primate Brain.Pre-clinical development of gene modification of haematopoietic stem cells with chimeric antigen receptors for cancer immunotherapy.Safe and Efficient Gene Therapy for Pyruvate Kinase DeficiencyHepatitis B virus nuclear export elements: RNA stem-loop α and β, key parts of the HBV post-transcriptional regulatory element.The fetal mouse is a sensitive genotoxicity model that exposes lentiviral-associated mutagenesis resulting in liver oncogenesis.Correction of the Exon 2 Duplication in DMD Myoblasts by a Single CRISPR/Cas9 System.Liver-directed lentiviral gene therapy in a dog model of hemophilia B.Design and Development of AAV-based Gene Supplementation Therapies for Achromatopsia and Retinitis Pigmentosa.
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P2860
Validation of a mutated PRE sequence allowing high and sustained transgene expression while abrogating WHV-X protein synthesis: application to the gene therapy of WAS.
description
2009 nî lūn-bûn
@nan
2009年の論文
@ja
2009年学术文章
@wuu
2009年学术文章
@zh-cn
2009年学术文章
@zh-hans
2009年学术文章
@zh-my
2009年学术文章
@zh-sg
2009年學術文章
@yue
2009年學術文章
@zh
2009年學術文章
@zh-hant
name
Validation of a mutated PRE se ...... on to the gene therapy of WAS.
@en
Validation of a mutated PRE se ...... on to the gene therapy of WAS.
@nl
type
label
Validation of a mutated PRE se ...... on to the gene therapy of WAS.
@en
Validation of a mutated PRE se ...... on to the gene therapy of WAS.
@nl
prefLabel
Validation of a mutated PRE se ...... on to the gene therapy of WAS.
@en
Validation of a mutated PRE se ...... on to the gene therapy of WAS.
@nl
P2093
P2860
P356
P1433
P1476
Validation of a mutated PRE se ...... on to the gene therapy of WAS.
@en
P2093
A Brice-Ouzet
A J Thrasher
M A Zanta-Boussif
S Charrier
P2860
P2888
P304
P356
10.1038/GT.2009.3
P577
2009-03-05T00:00:00Z
P5875
P6179
1011212900