Effect of 5-aminolevulinic acid on erythropoiesis: a preclinical in vitro characterization for the treatment of congenital sideroblastic anemia

Congenital sideroblastic anemia (CSA) is a hereditary disorder characterized by microcytic anemia and bone marrow sideroblasts. The most common form of CSA is attributed to mutations in the X-linked gene 5-aminolevulinic acid synthase 2 (ALAS2). ALAS2 is a mitochondrial enzyme, which utilizes glycin...

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Veröffentlicht in:	Biochemical and biophysical research communications 2014-11, Vol.454 (1), p.102-108
Hauptverfasser:	Fujiwara, Tohru, Okamoto, Koji, Niikuni, Ryoyu, Takahashi, Kiwamu, Okitsu, Yoko, Fukuhara, Noriko, Onishi, Yasushi, Ishizawa, Kenichi, Ichinohasama, Ryo, Nakamura, Yukio, Nakajima, Motowo, Tanaka, Tohru, Harigae, Hideo
Format:	Artikel
Sprache:	eng
Schlagworte:	5-Aminolevulinate Synthetase - antagonists & inhibitors 5-Aminolevulinate Synthetase - genetics 5-Aminolevulinate Synthetase - metabolism 60 APPLIED LIFE SCIENCES Amino Acid Transport Systems - genetics Amino Acid Transport Systems - metabolism AMINOBUTYRIC ACID AMINOLEVULINIC ACID Aminolevulinic Acid - pharmacology Anemia, Sideroblastic - drug therapy Anemia, Sideroblastic - genetics Anemia, Sideroblastic - metabolism ANEMIAS Animals BENZIDINE BONE MARROW BONE MARROW CELLS CONGENITAL DISEASES Drug Evaluation, Preclinical ENZYMES Erythroblasts - cytology Erythroblasts - drug effects Erythroblasts - metabolism ERYTHROPOIESIS Erythropoiesis - drug effects Erythropoiesis - genetics Erythropoiesis - physiology gamma-Aminobutyric Acid - pharmacology Gene Knockdown Techniques GENES Genetic Diseases, X-Linked - drug therapy Genetic Diseases, X-Linked - genetics Genetic Diseases, X-Linked - metabolism GLYCINE HEME Heme - biosynthesis Heme Oxygenase-1 - genetics Heme Oxygenase-1 - metabolism HEMOGLOBIN Hemoglobin A - genetics Hemoglobin A - metabolism Hemoglobins, Abnormal - genetics Hemoglobins, Abnormal - metabolism HUMAN POPULATIONS Humans Induced Pluripotent Stem Cells - cytology Induced Pluripotent Stem Cells - drug effects Induced Pluripotent Stem Cells - metabolism K562 Cells Mice MITOCHONDRIA POLYMERASE CHAIN REACTION RNA STEM CELLS Symporters - genetics Symporters - metabolism Up-Regulation - drug effects
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