A novel small molecule approach for the treatment of propionic and methylmalonic acidemias

Propionic Acidemia (PA) and Methylmalonic Acidemia (MMA) are inborn errors of metabolism affecting the catabolism of valine, isoleucine, methionine, threonine and odd-chain fatty acids. These are multi-organ disorders caused by the enzymatic deficiency of propionyl-CoA carboxylase (PCC) or methylmal...

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Bibliographic Details
Published in:	Molecular genetics and metabolism Vol. 133; no. 1; pp. 71 - 82
Main Authors:	Armstrong, Allison J., Collado, Maria Sol, Henke, Brad R., Olson, Matthew W., Hoang, Stephen A., Hamilton, Christin A., Pourtaheri, Taylor D., Chapman, Kimberly A., Summar, Marshall M., Johns, Brian A., Wamhoff, Brian R., Reardon, John E., Figler, Robert A.
Format:	Journal Article
Language:	English
Published:	United States Elsevier Inc 01.05.2021
Subjects:	Acyl Coenzyme A - metabolism Amino Acid Metabolism, Inborn Errors - drug therapy Amino Acid Metabolism, Inborn Errors - genetics Amino Acid Metabolism, Inborn Errors - pathology Carnitine - metabolism Cell Line Citrates - metabolism Hepatocytes - drug effects Humans Methylmalonic acidemia Methylmalonyl-CoA Decarboxylase - genetics Methylmalonyl-CoA Mutase - deficiency Methylmalonyl-CoA Mutase - genetics Propionic acidemia Propionic Acidemia - drug therapy Propionic Acidemia - genetics Propionic Acidemia - pathology Small Molecule Libraries - pharmacology Methylmalonic acidemia Propionic acidemia
ISSN:	1096-7192, 1096-7206, 1096-7206
Online Access:	Get full text
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