SIN retroviral vectors expressing COL7A1 under human promoters for ex vivo gene therapy of recessive dystrophic epidermolysis bullosa.
about
Molecular therapeutics for heritable skin diseasesAdvances in understanding and treating dystrophic epidermolysis bullosaInduced pluripotent stem cells from individuals with recessive dystrophic epidermolysis bullosa.Generation of keratinocytes from normal and recessive dystrophic epidermolysis bullosa-induced pluripotent stem cells.Genomic analysis of Sleeping Beauty transposon integration in human somatic cells.A single epidermal stem cell strategy for safe ex vivo gene therapy.Evaluation of residual promoter activity in γ-retroviral self-inactivating (SIN) vectors.Meganucleases and other tools for targeted genome engineering: perspectives and challenges for gene therapy.Preclinical corrective gene transfer in xeroderma pigmentosum human skin stem cells.Pantropic retroviruses as a transduction tool for sea urchin embryos.Lentiviral Engineered Fibroblasts Expressing Codon-Optimized COL7A1 Restore Anchoring Fibrils in RDEB.From marrow to matrix: novel gene and cell therapies for epidermolysis bullosa.Allogeneic blood and bone marrow cells for the treatment of severe epidermolysis bullosa: repair of the extracellular matrix.Novel and emerging therapies in the treatment of recessive dystrophic epidermolysis bullosa.Harlequin ichthyosis: ABCA12 mutations underlie defective lipid transport, reduced protease regulation and skin-barrier dysfunction.Bone marrow transplantation in epidermolysis bullosa.The long and winding road that leads to a cure for epidermolysis bullosa.Gene therapy: pursuing restoration of dermal adhesion in recessive dystrophic epidermolysis bullosa.Therapies for inherited skin fragility disorders.COL7A1 Editing via CRISPR/Cas9 in Recessive Dystrophic Epidermolysis Bullosa.Construction and validation of an RNA trans-splicing molecule suitable to repair a large number of COL7A1 mutations.Designing Efficient Double RNA trans-Splicing Molecules for Targeted RNA Repair.Development of murine leukemia virus-based retroviral vectors with a minimum possibility of cis-activation.High levels of type VII collagen expression in recessive dystrophic epidermolysis bullosa cutaneous squamous cell carcinoma keratinocytes increases PI3K and MAPK signalling, cell migration and invasion.Keratinocyte cell lines derived from severe generalized recessive epidermolysis bullosa patients carrying a highly recurrent COL7A1 homozygous mutation: models to assess cell and gene therapies in vitro and in vivo.An RNA-targeted therapy for dystrophic epidermolysis bullosa.CRISPR/Cas9-Based Cellular Engineering for Targeted Gene Overexpression.Management of severe epidermolysis bullosa by haematopoietic transplant: principles, perspectives and pitfalls.Ex Vivo COL7A1 Correction for Recessive Dystrophic Epidermolysis Bullosa Using CRISPR/Cas9 and Homology-Directed Repair
P2860
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P2860
SIN retroviral vectors expressing COL7A1 under human promoters for ex vivo gene therapy of recessive dystrophic epidermolysis bullosa.
description
2010 nî lūn-bûn
@nan
2010年の論文
@ja
2010年論文
@yue
2010年論文
@zh-hant
2010年論文
@zh-hk
2010年論文
@zh-mo
2010年論文
@zh-tw
2010年论文
@wuu
2010年论文
@zh
2010年论文
@zh-cn
name
SIN retroviral vectors express ...... trophic epidermolysis bullosa.
@en
type
label
SIN retroviral vectors express ...... trophic epidermolysis bullosa.
@en
prefLabel
SIN retroviral vectors express ...... trophic epidermolysis bullosa.
@en
P2093
P2860
P50
P356
P1433
P1476
SIN retroviral vectors express ...... trophic epidermolysis bullosa.
@en
P2093
Agnes Brice
Audrey Décha
Laure Tonasso
Maria A Zanta-Boussif
Nathalie Pironon
Olivier Danos
Valérie Pendaries
P2860
P304
P356
10.1038/MT.2010.91
P577
2010-05-18T00:00:00Z