Natural gene therapy in monozygotic twins with Fanconi anemia
about
Molecular pathogenesis and clinical management of Fanconi anemiaAluY-mediated germline deletion, duplication and somatic stem cell reversion in UBE2T defines a new subtype of Fanconi anemia.Preclinical correction of human Fanconi anemia complementation group A bone marrow cells using a safety-modified lentiviral vector.Comparison of chromosome breakage in non-mosaic and mosaic patients with Fanconi anemia, relatives, and patients with other inherited bone marrow failure syndromes.Secondary mutations of BRCA1/2 and drug resistanceStem cell gene therapy for fanconi anemia: report from the 1st international Fanconi anemia gene therapy working group meeting.Very short telomere length by flow fluorescence in situ hybridization identifies patients with dyskeratosis congenita.Disrupted Signaling through the Fanconi Anemia Pathway Leads to Dysfunctional Hematopoietic Stem Cell Biology: Underlying Mechanisms and Potential Therapeutic Strategies.TNF-alpha induces leukemic clonal evolution ex vivo in Fanconi anemia group C murine stem cells.Revertant mosaicism for family mutations is not observed in BRCA1/2 phenocopiesInsertional mutagenesis and clonal dominance: biological and statistical considerations.Overnight transduction with foamyviral vectors restores the long-term repopulating activity of Fancc-/- stem cells.Diagnosis of Fanconi anemia in patients with bone marrow failureWhere genotype is not predictive of phenotype: towards an understanding of the molecular basis of reduced penetrance in human inherited disease.Myelodysplasia and acute leukemia as late complications of marrow failure: future prospects for leukemia prevention.Finding the needle in the hay stack: hematopoietic stem cells in Fanconi anemia.Patient-specific naturally gene-reverted induced pluripotent stem cells in recessive dystrophic epidermolysis bullosaThe Fanconi anaemia pathway orchestrates incisions at sites of crosslinked DNA.HSP90 Shapes the Consequences of Human Genetic Variation.How I manage patients with Fanconi anaemia.FANCJ/BRIP1 recruitment and regulation of FANCD2 in DNA damage responses.Validation of Fanconi anemia complementation Group A assignment using molecular analysis.Gene therapy for Fanconi anemia: one step closer to the clinic.Discovering early molecular determinants of leukemogenesis.Stem Cell Therapy for Fanconi Anemia.Somatic mosaicism of an intragenic FANCB duplication in both fibroblast and peripheral blood cells observed in a Fanconi anemia patient leads to milder phenotype.Recent advances in understanding hematopoiesis in Fanconi Anemia.Measles virus envelope pseudotyped lentiviral vectors transduce quiescent human HSCs at an efficiency without precedent
P2860
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P2860
Natural gene therapy in monozygotic twins with Fanconi anemia
description
2006 nî lūn-bûn
@nan
2006年の論文
@ja
2006年論文
@yue
2006年論文
@zh-hant
2006年論文
@zh-hk
2006年論文
@zh-mo
2006年論文
@zh-tw
2006年论文
@wuu
2006年论文
@zh
2006年论文
@zh-cn
name
Natural gene therapy in monozygotic twins with Fanconi anemia
@ast
Natural gene therapy in monozygotic twins with Fanconi anemia
@en
type
label
Natural gene therapy in monozygotic twins with Fanconi anemia
@ast
Natural gene therapy in monozygotic twins with Fanconi anemia
@en
prefLabel
Natural gene therapy in monozygotic twins with Fanconi anemia
@ast
Natural gene therapy in monozygotic twins with Fanconi anemia
@en
P2093
P2860
P1433
P1476
Natural gene therapy in monozygotic twins with Fanconi anemia
@en
P2093
Alan D'Andrea
Barbara Cox
Grover Bagby
Lora Lucas
R Keaney Rathbun
Susan Olson
Toshiyasu Taniguchi
Yassmine Akkari
P2860
P304
P356
10.1182/BLOOD-2005-07-2638
P407
P577
2006-01-05T00:00:00Z