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- W2783297616 abstract "The β-hemoglobinopathies sickle cell anemia and β-thalassemia are the focus of many gene-therapy studies. A key disease parameter is the abundance of globin chains because it indicates the level of anemia, likely toxicity of excess or aberrant globins, and therapeutic potential of induced or exogenous β-like globins. Reversed-phase high-performance liquid chromatography (HPLC) allows versatile and inexpensive globin quantification, but commonly applied protocols suffer from long run times, high sample requirements, or inability to separate murine from human β-globin chains. The latter point is problematic for in vivo studies with gene-addition vectors in murine disease models and mouse/human chimeras. This study demonstrates HPLC-based measurements of globin expression (1) after differentiation of the commonly applied human umbilical cord blood–derived erythroid progenitor-2 cell line, (2) in erythroid progeny of CD34+ cells for the analysis of clustered regularly interspaced short palindromic repeats/Cas9-mediated disruption of the globin regulator BCL11A, and (3) of transgenic mice holding the human β-globin locus. At run times of 8 min for separation of murine and human β-globin chains as well as of human γ-globin chains, and with routine measurement of globin-chain ratios for 12 nL of blood (tested for down to 0.75 nL) or of 300,000 in vitro differentiated cells, the methods presented here and any variant-specific adaptations thereof will greatly facilitate evaluation of novel therapy applications for β-hemoglobinopathies." @default.
- W2783297616 created "2018-01-26" @default.
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- W2783297616 date "2018-02-01" @default.
- W2783297616 modified "2023-10-10" @default.
- W2783297616 title "Rapid and Sensitive Assessment of Globin Chains for Gene and Cell Therapy of Hemoglobinopathies" @default.
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- W2783297616 cites W1599625483 @default.
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- W2783297616 cites W1926422702 @default.
- W2783297616 cites W1964155649 @default.
- W2783297616 cites W1965124400 @default.
- W2783297616 cites W1968354447 @default.
- W2783297616 cites W1974742081 @default.
- W2783297616 cites W1980913087 @default.
- W2783297616 cites W1999613272 @default.
- W2783297616 cites W2006519535 @default.
- W2783297616 cites W2007062339 @default.
- W2783297616 cites W2009503795 @default.
- W2783297616 cites W2015378087 @default.
- W2783297616 cites W2015704152 @default.
- W2783297616 cites W2019433045 @default.
- W2783297616 cites W2020066606 @default.
- W2783297616 cites W2020306029 @default.
- W2783297616 cites W2022747526 @default.
- W2783297616 cites W2025091036 @default.
- W2783297616 cites W2028135932 @default.
- W2783297616 cites W2031211461 @default.
- W2783297616 cites W2033737798 @default.
- W2783297616 cites W2037439664 @default.
- W2783297616 cites W2045435533 @default.
- W2783297616 cites W2053495832 @default.
- W2783297616 cites W2058571172 @default.
- W2783297616 cites W2062254735 @default.
- W2783297616 cites W2065590076 @default.
- W2783297616 cites W2066625873 @default.
- W2783297616 cites W2086146777 @default.
- W2783297616 cites W2093229747 @default.
- W2783297616 cites W2099538751 @default.
- W2783297616 cites W2100908666 @default.
- W2783297616 cites W2101111741 @default.
- W2783297616 cites W2101742876 @default.
- W2783297616 cites W2106070783 @default.
- W2783297616 cites W2112513056 @default.
- W2783297616 cites W2115505342 @default.
- W2783297616 cites W2116767791 @default.
- W2783297616 cites W2118254429 @default.
- W2783297616 cites W2136274459 @default.
- W2783297616 cites W2138231299 @default.
- W2783297616 cites W2138733871 @default.
- W2783297616 cites W2139139020 @default.
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- W2783297616 cites W2153899268 @default.
- W2783297616 cites W2160888392 @default.
- W2783297616 cites W2167575052 @default.
- W2783297616 cites W2266790363 @default.
- W2783297616 cites W2293398762 @default.
- W2783297616 cites W2315423695 @default.
- W2783297616 cites W2339807029 @default.
- W2783297616 cites W2465447473 @default.
- W2783297616 cites W2511090954 @default.
- W2783297616 cites W2513071654 @default.
- W2783297616 cites W2513179752 @default.
- W2783297616 cites W2555597093 @default.
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- W2783297616 cites W269747772 @default.
- W2783297616 cites W2758269208 @default.
- W2783297616 cites W36670911 @default.
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- W2783297616 doi "https://doi.org/10.1089/hgtb.2017.190" @default.
- W2783297616 hasPubMedCentralId "https://www.ncbi.nlm.nih.gov/pmc/articles/5806072" @default.
- W2783297616 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/29325430" @default.
- W2783297616 hasPublicationYear "2018" @default.
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