POLYNUCLEOTIDES ENCODING VERY LONG-CHAIN ACYL-COA DEHYDROGENASE FOR THE TREATMENT OF VERY LONG-CHAIN ACYL-COA DEHYDROGENASE DEFICIENCY

摘要

This disclosure relates to mRNA therapy for the treatment of very long-chain specific acyl-CoA dehydrogenase deficiency (VLCADD). mRNAs for use in the invention, when administered in vivo, encode human very long-chain specific acyl-CoA dehydrogenase (VLCAD). mRNA therapies of the disclosure increase and/or restore deficient levels of VLCAD expression and/or activity in subjects. mRNA therapies of the disclosure further decrease abnormal accumulation of acylcarnitine associated with deficient VLCAD activity in subjects.