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ORIGINAL ARTICLES  TRENDS IN MOLECULAR DIAGNOSIS AND THERAPY OF β-THALASSEMIA AND SICKLE CELL ANEMIA 

Minerva Biotecnologica 2003 June;15(2):129-36

Copyright © 2003 EDIZIONI MINERVA MEDICA

language: English

The use of cell culture procedures for studying fetal hemoglobin stimulating drugs

Fibach E.

Department of Hematology, Hadassah University Hospital, Jerusalem, Israel


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High ­fetal hemo­glo­bin (HbF) has ­been ­shown to ame­li­o­rate the clin­i­cal symp­toms of ­patients ­with β-hemo­glob­i­nop­a­thies, β-tha­las­se­mia (β-­thal) and sick­le ­cell ane­mia (SCA). Research is there­fore ­focus on find­ing ­drugs ­capable of reac­ti­vat­ing the γ-glo­bin ­genes and stim­u­lat­ing the pro­duc­tion of HbF. Several in ­vitro experi­men­tal mod­els ­have ­been devel­oped to ­serve ­this pur­pose. Two mod­els are the sub­ject of ­this ­review: in ­vitro estab­lished eryth­roid-­like ­cell ­lines and pri­mary cul­tures of eryth­roid ­cells ­derived ­from pro­gen­i­tors ­obtained ­from nor­mal ­donors and ­patients ­with β-­thal and SCA. These experi­men­tal mod­els are use­ful for screen­ing of com­pounds and for stud­y­ing ­their mech­a­nism of ­action at the cel­lu­lar and molec­u­lar lev­els. These stud­ies are essen­tial for find­ing, test­ing and devel­op­ing new, effec­tive and ­safe ­drugs.

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