Synthetic mmab genes and aav vectors to treat cobalamin b deficiency
AssigneeThe United States of America, as represented by the Secretary, Department of Health and Human Servi
InventorsCharles P. Venditti, Eun-Young Choi
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Abstract
Synthetic polynucleotides encoding human MMAB (Metabolism Of Cobalamin Associated B) enzyme (synMMAB) and exhibiting augmented expression in cell culture and/or in a mammal are described herein. Related recombinant expression vectors, host cells, populations of cells, and pharmaceutical compositions relating to the synMMAB polynucleotides are also described. Methods of treating a disease or condition mediated by MMAB enzyme are also described.
Claims26
A synthetic MMAB (Metabolism Of Cobalamin Associated B) polynucleotide (synMMAB) selected from the group consisting of:
- (a) a polynucleotide comprising the nucleic acid sequence of any one of SEQ ID NOs:
- 1-6;
- (b) a polynucleotide having a nucleic acid sequence with at least about 80% identity to the nucleic acid sequence of any one of SEQ ID NOs:
- 1-6 and encoding a polypeptide having an amino acid sequence at least about 90% identical to SEQ ID NO:
- 21, and having equivalent or increased expression in a mammalian host cell relative to expression of SEQ ID NO:
- 7 in the same host cell, wherein the polynucleotide does not have the nucleic acid sequence of SEQ ID NO:
- 7;
- and
- (c) a polynucleotide having a nucleic acid sequence with at least about 80% identity to the nucleic acid sequence of nucleotides 97-753 of any one of SEQ ID NOs:
- 1-6 and encoding a polypeptide having an amino acid sequence at least about 90% identical to SEQ ID NO:
- 23, and having equivalent or increased expression in a mammalian host cell relative to expression of nucleotides 97-753 of SEQ ID NO:
- 7 in the same host cell, wherein the polynucleotide does not have the nucleic acid sequence of nucleotides 97-753 of SEQ ID NO:
- 7.
Dependencies are inferred from the claim text.