PMID: 11605173Oct 18, 2001Paper

Cell culture and animal models to screen for promising fetal hemoglobin-stimulating compounds

Seminars in Hematology
Eitan Fibach

Abstract

Sickle cell anemia (SCA) and the thalassemias are globally the most common class of inherited single-gene disorders. Current treatment options are limited, especially in developing countries. More practical and cheaper therapies are urgently needed. Since high fetal hemoglobin (HbF) levels ameliorate the clinical symptoms of these diseases, one current approach is to use pharmacological agents to reactivate the gamma-globin genes and stimulate the production of HbF. Several in vitro and in vivo experimental models developed for this purpose are the subject of this review. The models include in vitro established erythroid-like cell lines and primary cultures (both in semisolid and liquid media) of erythroid progenitor cells obtained from normal donors and patients with SCA and beta-thalassemia, as well as in vivo models in genetically modified (transgenic) and unmodified animals. These experimental systems are useful for large-scale screening of compounds for HbF-stimulating potential, for determining the mechanism of action of potent compounds at the cellular and molecular levels, and for studying the pharmacology, pharmacokinetics, and toxicology of the drugs. These models are essential to find, test, and develop new drugs tha...Continue Reading

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Citations

Nov 28, 2012·Journal of Cardiovascular Translational Research·Paul ChackoSubha V Raman
Aug 7, 2009·Experimental Biology and Medicine·Tzu-Fang LouBetty S Pace
Feb 3, 2005·British Journal of Haematology·A Daisy NarayanEsmail D Zanjani
Sep 4, 2016·Journal of Translational Medicine·Lucia Carmela CosenzaNicoletta Bianchi
Nov 30, 2018·Molecular Diagnosis & Therapy·Eitan Fibach, Mutaz Dana
Sep 29, 2007·Journal of Cellular Biochemistry·Moshe J LeimbergEitan Fibach
Dec 8, 2019·Journal of Clinical Medicine·Eitan Fibach
Sep 16, 2005·The New England Journal of Medicine·Deborah Rund, Eliezer Rachmilewitz
Apr 25, 2008·Current Protocols in Immunology·E Fibach, E Prus
Oct 29, 2008·Evidence-based Complementary and Alternative Medicine : ECAM·Antara BanerjeeAnisur Rahman Khuda-Bukhsh

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