Myogenic vector systems
AssigneeBaylor College of Medicine
InventorsRobert J. Schwartz, Franco J. DeMayo, Bert W. O'Malley
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Abstract
The present invention includes a Myogenic vector system (MVS) for the expression of a nucleic acid sequence in myogenic tissue. The MVS contains a promoter, a cassette with the sequence of interest, a 3' untranslated region (3' UTR) and contiguous noncoding region (NCR). Further enhancements can include the addition of a leader sequence, intron sequence, initiation codon and specific This invention was partially supported by a grant from the United States government under HL-38401 awarded by the National Institute of Health. The Government has certain rights in the invention.
Claims32
A myogenic vector system (MVS) for the expression of a nucleic acid sequence in myogenic tissue, comprising a promoter;
- a cassette whose 5' end is attached to the 3' end of the promoter, said cassette containing a nucleic acid sequence which is to be expressed;
- a myogenic specific 3' untranslated region (3'UTR) and a non-coding region (NCR) contiguous to the 3' end of the 3' UTR, said NCR containing a transcriptional termination signal, wherein the 5' end of the 3' UTR is attached to the 3' end of the cassette.
A MVS for the expression of a nucleic acid sequence in myogenic tissues comprising a functional unit which expresses the nucleic acid sequence, wherein the functional unit is comprised of elements all sequentially linked together 5' to 3', said elements include a promoter;
- a 5' MRNA leader sequence;
- a first intron;
- an initiation ATG and NcoI cloning site;
- a cassette, wherein the cassette has an EcoR1 site at its 3' end and contains the nucleic acid sequence to be expressed;
- a myogenic specific 3' untranslated region (3' UTR);
- and a non-coding region (NCR) contiguous to the 3' end of the 3'UTR, said NCR containing a transcriptional termination signal.
A myogenic vector system (MVS) for the expression of a nucleic acid sequence in myogenic tissue, comprising:
- a 5' oligonucleotide, said oligonucleotide including a promoter;
- a 3' oligonucleotide containing a myogenic specific 3' untranslated region (3' UTR) and a non-coding region (NCR) contiguous to the 3' end of the 3' UTR, said NCR containing a transcriptional termination signal;
- and
- a linker, having a plurality of restriction endonuclease sites, said linker connecting the 5' oligonucleotide to the 3' oligonucleotide and said linker further providing a position for insertion of a cassette containing the nucleic acid to be expressed.
A regulatable myogenic vector system (MVS) for the expression of a specific nucleic acid sequence in myogenic tissue comprising:
- a first functional unit, elements of said first unit comprise a myogenic specific promoter;
- a nucleic acid sequence coding for a receptor;
- a myogenic specific 3' untranslated region (3' UTR) and a myogenic specific non-coding region (NCR);
- wherein the elements of the first unit are linked together sequentially starting from the 5' end with the promoter, followed by the receptor nucleic acid sequence, followed by the 3' UTR and contiguous NCR;
- a second functional unit, elements of said second unit comprise a response element corresponding to the receptor;
- a thymidine kinase promoter;
- the specific nucleic acid sequence of interest;
- a myogenic specific 3' UTR and a contiguous myogenic specific NCR;
- wherein the elements of the second unit are linked together sequentially starting from the 5' end with the response element followed by promoter followed by the specific sequence, followed by the 3' UTR and NCR;
- wherein the first unit expresses the receptor, said receptor forming an interaction between the response element, the receptor and an agent which binds to the receptor, said interaction up regulating the expression of the sequence of interest.
Dependencies are inferred from the claim text.