Codon optimized cftr
AssigneeCopernicus Therapeutics, Inc.
InventorsMark J. Cooper, Linas Padegimas
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AssigneeCopernicus Therapeutics, Inc.
InventorsMark J. Cooper, Linas Padegimas
Estimating the term…
A synthetic hCFTR DNA sequence has been developed that produces remarkably high levels of hCFTR mRNA and protein in dosed murine lungs and human cells in culture compared to the natural hCFTR cDNA. This synthetic DNA addresses problems inherent in some natural cDNAs, such as premature transcriptional truncation sites introduced during cDNA synthesis. Introns are initially present in mRNA until the mRNA is processed. cDNA made from processed mRNA is devoid of introns. Thus DNA sequences (exon junctions) are present in a cDNA molecule which are not present in cells in nature. These exon junctions may affect transcription. Methods for improving expression of CFTR are based on sequence changes in cDNA molecules. The improvement methods may be applied to other cDNA molecules which are refractory to in vivo expression efforts. Compositions embodying the sequence changes increase the production of both transgenic mRNA and protein from cDNA molecules.
A composition comprising a nucleic acid molecule comprising a sequence as shown in SEQ ID NO:
A method of producing hCFTR-encoding mRNA and hCFTR protein comprising:
A method of producing hCFTR-encoding mRNA and hCFTR protein comprising:
A method to increase the expression of an mRNA or protein from a cDNA molecule, comprising:
Dependencies are inferred from the claim text.