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POLYNUCLEOTIDES ENCODING VERY LONG-CHAIN ACYL-COA DEHYDROGENASE FOR THE TREATMENT OF VERY LONG-CHAIN ACYL-COA DEHYDROGENASE DEFICIENCY
POLYNUCLEOTIDES ENCODING VERY LONG-CHAIN ACYL-COA DEHYDROGENASE FOR THE TREATMENT OF VERY LONG-CHAIN ACYL-COA DEHYDROGENASE DEFICIENCY
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机译:编码非常长链酰基-CoA脱氢酶的多核苷酸用于处理非常长链酰基-CoA脱氢酶缺乏
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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.
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