Reverse mosaicism in Fanconi anemia: natural gene therapy via molecular self-correction.
about
Molecular pathogenesis and clinical management of Fanconi anemiaDiagnosis of Fanconi Anemia: Chromosomal Breakage AnalysisAluY-mediated germline deletion, duplication and somatic stem cell reversion in UBE2T defines a new subtype of Fanconi anemia.Disease-corrected haematopoietic progenitors from Fanconi anaemia induced pluripotent stem cells.Inherited bone marrow failure syndromes in adolescents and young adults.Neonatal manifestations of inherited bone marrow failure syndromes.Molecular analysis of Fanconi anemia: the experience of the Bone Marrow Failure Study Group of the Italian Association of Pediatric Onco-Hematology.CRISPR/Cas9-Mediated Correction of the FANCD1 Gene in Primary Patient CellsPreclinical correction of human Fanconi anemia complementation group A bone marrow cells using a safety-modified lentiviral vector.Spontaneous abrogation of the G₂DNA damage checkpoint has clinical benefits but promotes leukemogenesis in Fanconi anemia patients.Comparison of chromosome breakage in non-mosaic and mosaic patients with Fanconi anemia, relatives, and patients with other inherited bone marrow failure syndromes.Stem cell gene therapy for fanconi anemia: report from the 1st international Fanconi anemia gene therapy working group meeting.In vivo reversion to normal of inherited mutations in humans.Unrelated donor bone marrow transplantation for the treatment of Fanconi anemia.Natural gene therapy in monozygotic twins with Fanconi anemiaDisrupted Signaling through the Fanconi Anemia Pathway Leads to Dysfunctional Hematopoietic Stem Cell Biology: Underlying Mechanisms and Potential Therapeutic Strategies.Revertant mosaicism for family mutations is not observed in BRCA1/2 phenocopiesIdentification of point mutations and large intragenic deletions in Fanconi anemia using next-generation sequencing technologyOvernight transduction with foamyviral vectors restores the long-term repopulating activity of Fancc-/- stem cells.A comprehensive strategy for the subtyping of patients with Fanconi anaemia: conclusions from the Spanish Fanconi Anemia Research Network.Diagnosis of Fanconi anemia in patients with bone marrow failureFinding 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 bullosaCurrent clinical management of Fanconi anemia.HSP90 Shapes the Consequences of Human Genetic Variation.Germline Genetic Predisposition to Hematologic Malignancy.How I manage patients with Fanconi anaemia.Pediatric laryngeal carcinoma in a heterozygous carrier of Fanconi anemia.Targeted gene therapy and cell reprogramming in Fanconi anemia.Diagnostic Overlap between Fanconi Anemia and the Cohesinopathies: Roberts Syndrome and Warsaw Breakage SyndromeDiagnosis of Fanconi's Anemia by Diepoxybutane Analysis in Children from Serbia.Vaccine-associated varicella and rubella infections in severe combined immunodeficiency with isolated CD4 lymphocytopenia and mutations in IL7R detected by tandem whole exome sequencing and chromosomal microarray.Induced pluripotent stem cells as a tool for gaining new insights into Fanconi anemia.Gene therapy for Fanconi anemia: one step closer to the clinic.Myeloid lineage-selective growth of revertant cells in Fanconi anaemia.Current and emerging therapeutic strategies for Fanconi anemia.Therapeutic gene editing in CD34+ hematopoietic progenitors from Fanconi anemia patients.Clonal hematopoiesis in patients with dyskeratosis congenita.Somatic mosaicism of an intragenic FANCB duplication in both fibroblast and peripheral blood cells observed in a Fanconi anemia patient leads to milder phenotype.A "late-but-fitter revertant cell" explains the high frequency of revertant mosaicism in epidermolysis bullosa.
P2860
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P2860
Reverse mosaicism in Fanconi anemia: natural gene therapy via molecular self-correction.
description
2002 nî lūn-bûn
@nan
2002年の論文
@ja
2002年論文
@yue
2002年論文
@zh-hant
2002年論文
@zh-hk
2002年論文
@zh-mo
2002年論文
@zh-tw
2002年论文
@wuu
2002年论文
@zh
2002年论文
@zh-cn
name
Reverse mosaicism in Fanconi anemia: natural gene therapy via molecular self-correction.
@en
type
label
Reverse mosaicism in Fanconi anemia: natural gene therapy via molecular self-correction.
@en
prefLabel
Reverse mosaicism in Fanconi anemia: natural gene therapy via molecular self-correction.
@en
P2093
P356
P1476
Reverse mosaicism in Fanconi anemia: natural gene therapy via molecular self-correction.
@en
P2093
Dietrich R
Hanenberg H
Herterich S
Schindler D
P304
P356
10.1159/000069805
P577
2002-01-01T00:00:00Z