Matches in SemOpenAlex for { <https://semopenalex.org/work/W2133815318> ?p ?o ?g. }
- W2133815318 endingPage "250" @default.
- W2133815318 startingPage "245" @default.
- W2133815318 abstract "Fanconi anemia (FA) is an inherited DNA repair disorder characterized by genetic instability of cells lacking a functional FA/BRCA pathway. Previous studies have shown that in vitro stimulation of bone marrow cells (BMCs) from FA mice promotes apoptosis, reduces the reconstitution ability of the stem cells, and induces myelodysplasia and myeloid leukemia upon reinfusion of the cells. This suggests the convenience of adapting standard protocols of gene therapy to FA. Here we show that the reserve of BM progenitors in FA patients is generally below 20% of normal values. Because this reduced reserve could activate the cycling of BM progenitors, we developed a simplified protocol to transduce BMCs from FA patients with gammaretroviral vectors. We demonstrate that a short in vitro manipulation (12-24 hr) of fresh mononuclear BMCs is sufficient to transduce 42% of hematopoietic progenitors from FA-A patients, in the absence of in vitro prestimulation. When FANCA-expressing vectors were used, this simple procedure reversed the phenotype of the BM progenitors from these patients. We propose that our approach will be more efficient and safer compared with standard gene therapy protocols for FA." @default.
- W2133815318 created "2016-06-24" @default.
- W2133815318 creator A5017322730 @default.
- W2133815318 creator A5031716314 @default.
- W2133815318 creator A5042038610 @default.
- W2133815318 creator A5044688513 @default.
- W2133815318 creator A5046386647 @default.
- W2133815318 creator A5058916695 @default.
- W2133815318 creator A5059626582 @default.
- W2133815318 creator A5061715630 @default.
- W2133815318 creator A5064733020 @default.
- W2133815318 creator A5069870899 @default.
- W2133815318 creator A5077863036 @default.
- W2133815318 creator A5083196574 @default.
- W2133815318 creator A5090896983 @default.
- W2133815318 date "2006-02-01" @default.
- W2133815318 modified "2023-10-18" @default.
- W2133815318 title "A Simplified Approach to Improve the Efficiency and Safety of<i>Ex Vivo</i>Hematopoietic Gene Therapy in Fanconi Anemia Patients" @default.
- W2133815318 cites W1496836853 @default.
- W2133815318 cites W1541725266 @default.
- W2133815318 cites W1742238700 @default.
- W2133815318 cites W1991555921 @default.
- W2133815318 cites W1991778691 @default.
- W2133815318 cites W2011798292 @default.
- W2133815318 cites W2012102598 @default.
- W2133815318 cites W2015532507 @default.
- W2133815318 cites W2020538790 @default.
- W2133815318 cites W2036737199 @default.
- W2133815318 cites W2039379309 @default.
- W2133815318 cites W2082795668 @default.
- W2133815318 cites W2086682597 @default.
- W2133815318 cites W2089894429 @default.
- W2133815318 cites W2092950132 @default.
- W2133815318 cites W2093375366 @default.
- W2133815318 cites W2107278930 @default.
- W2133815318 cites W2133155245 @default.
- W2133815318 cites W2143729842 @default.
- W2133815318 cites W2156375180 @default.
- W2133815318 cites W2157153664 @default.
- W2133815318 cites W2159530833 @default.
- W2133815318 cites W2166086526 @default.
- W2133815318 cites W2170942823 @default.
- W2133815318 cites W2253940800 @default.
- W2133815318 cites W2256784153 @default.
- W2133815318 cites W2321748797 @default.
- W2133815318 doi "https://doi.org/10.1089/hum.2006.17.245" @default.
- W2133815318 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/16454658" @default.
- W2133815318 hasPublicationYear "2006" @default.
- W2133815318 type Work @default.
- W2133815318 sameAs 2133815318 @default.
- W2133815318 citedByCount "19" @default.
- W2133815318 countsByYear W21338153182012 @default.
- W2133815318 countsByYear W21338153182014 @default.
- W2133815318 countsByYear W21338153182015 @default.
- W2133815318 countsByYear W21338153182016 @default.
- W2133815318 countsByYear W21338153182017 @default.
- W2133815318 countsByYear W21338153182018 @default.
- W2133815318 countsByYear W21338153182019 @default.
- W2133815318 countsByYear W21338153182020 @default.
- W2133815318 crossrefType "journal-article" @default.
- W2133815318 hasAuthorship W2133815318A5017322730 @default.
- W2133815318 hasAuthorship W2133815318A5031716314 @default.
- W2133815318 hasAuthorship W2133815318A5042038610 @default.
- W2133815318 hasAuthorship W2133815318A5044688513 @default.
- W2133815318 hasAuthorship W2133815318A5046386647 @default.
- W2133815318 hasAuthorship W2133815318A5058916695 @default.
- W2133815318 hasAuthorship W2133815318A5059626582 @default.
- W2133815318 hasAuthorship W2133815318A5061715630 @default.
- W2133815318 hasAuthorship W2133815318A5064733020 @default.
- W2133815318 hasAuthorship W2133815318A5069870899 @default.
- W2133815318 hasAuthorship W2133815318A5077863036 @default.
- W2133815318 hasAuthorship W2133815318A5083196574 @default.
- W2133815318 hasAuthorship W2133815318A5090896983 @default.
- W2133815318 hasConcept C104317684 @default.
- W2133815318 hasConcept C109159458 @default.
- W2133815318 hasConcept C111599444 @default.
- W2133815318 hasConcept C134935766 @default.
- W2133815318 hasConcept C201750760 @default.
- W2133815318 hasConcept C202751555 @default.
- W2133815318 hasConcept C203014093 @default.
- W2133815318 hasConcept C26291073 @default.
- W2133815318 hasConcept C2778124228 @default.
- W2133815318 hasConcept C2778993383 @default.
- W2133815318 hasConcept C2780007613 @default.
- W2133815318 hasConcept C28328180 @default.
- W2133815318 hasConcept C502942594 @default.
- W2133815318 hasConcept C54355233 @default.
- W2133815318 hasConcept C86803240 @default.
- W2133815318 hasConcept C95444343 @default.
- W2133815318 hasConceptScore W2133815318C104317684 @default.
- W2133815318 hasConceptScore W2133815318C109159458 @default.
- W2133815318 hasConceptScore W2133815318C111599444 @default.
- W2133815318 hasConceptScore W2133815318C134935766 @default.
- W2133815318 hasConceptScore W2133815318C201750760 @default.
- W2133815318 hasConceptScore W2133815318C202751555 @default.
- W2133815318 hasConceptScore W2133815318C203014093 @default.
- W2133815318 hasConceptScore W2133815318C26291073 @default.
- W2133815318 hasConceptScore W2133815318C2778124228 @default.