Patent classifications
C12N15/69
SYNTHETIC SELF-AMPLIFYING MRNA MOLECULES WITH SECRETION ANTIGEN AND IMMUNOMODULATOR
Lipid nanoparticle (LNP) encapsulating self-amplifying mRNA, compositions, and methods of using the novel nucleic acid constructs and compositions are disclosed. LNP constructs include novel ionizable lipid. Novel sa-mRNA constructs encode a modified SARS-CoV-2 spike protein, wherein the polynucleotide has been truncated to not include nucleotides encoding a SARS-CoV-2 transmembrane domain and short cytosolic domain amino acids and immunomodulators. Sa-mRNAs are useful in for use as a therapeutic, diagnostic and/or prophylactic agent to mammalian cells or organs.
Amplifiable RNAs for Therapeutic Cell Systems
This disclosure provides, among other things, amplifiable nucleic acid constructs for expressing a gene of interest in a cell, e.g., an erythroid cell. The amplifiable nucleic acid construct may contain the gene of interest and an RNA-dependent RNA polymerase (RdRP)-responsive 5′ UTR, and may optionally further contain an RdRP-responsive 3′ UTR. RdRP may also be provided, e.g., on the same construct or a different construct.
Amplifiable RNAs for Therapeutic Cell Systems
This disclosure provides, among other things, amplifiable nucleic acid constructs for expressing a gene of interest in a cell, e.g., an erythroid cell. The amplifiable nucleic acid construct may contain the gene of interest and an RNA-dependent RNA polymerase (RdRP)-responsive 5′ UTR, and may optionally further contain an RdRP-responsive 3′ UTR. RdRP may also be provided, e.g., on the same construct or a different construct.
Gene therapy DNA vector VTvaf17, method of production; <i>Escherichia coli </i>strain SCS110-AF, method of production; <i>Escherichia coli </i>strain SCS110-AF/VTvaf17 bearing gene therapy DNA vector VTvaf17, method of production
Disclosed is a gene therapy DNA vector VTvaf17 for genetic modification of animal and human cells having SEQ ID No. 1, and methods for its synthesis, involving constructing vectors containing a promoter region of human elongation factor EF1A, a polylinker with sites for restriction endonucleases, a transcription terminator, a polyadenylation sequence of human growth factor, a regulatory element of transposon Tn10 allowing for antibiotic-free positive selection, an origin of replication, and a kanamycin resistance gene. Escherichia coli strain SCS110-AF is also provided by the present invention. The method for creating the strain involves constructing a linear DNA fragment containing regulatory element of transposon Tn10, a levansucrase gene, sacB, a chloramphenicol resistance gene, and two homologous sequences. The E. coli cells are transformed by electroporation and clones surviving chloramphenicol are chosen. The invention further discloses Escherichia coli strain SCS110-AF/VTvaf17, which carries DNA vector VTvaf17, and methods for its synthesis.
Gene therapy DNA vector VTvaf17, method of production; <i>Escherichia coli </i>strain SCS110-AF, method of production; <i>Escherichia coli </i>strain SCS110-AF/VTvaf17 bearing gene therapy DNA vector VTvaf17, method of production
Disclosed is a gene therapy DNA vector VTvaf17 for genetic modification of animal and human cells having SEQ ID No. 1, and methods for its synthesis, involving constructing vectors containing a promoter region of human elongation factor EF1A, a polylinker with sites for restriction endonucleases, a transcription terminator, a polyadenylation sequence of human growth factor, a regulatory element of transposon Tn10 allowing for antibiotic-free positive selection, an origin of replication, and a kanamycin resistance gene. Escherichia coli strain SCS110-AF is also provided by the present invention. The method for creating the strain involves constructing a linear DNA fragment containing regulatory element of transposon Tn10, a levansucrase gene, sacB, a chloramphenicol resistance gene, and two homologous sequences. The E. coli cells are transformed by electroporation and clones surviving chloramphenicol are chosen. The invention further discloses Escherichia coli strain SCS110-AF/VTvaf17, which carries DNA vector VTvaf17, and methods for its synthesis.
RECOMBINANT YEAST STRAIN HAVING STEROL PRODUCTIVITY, PREPARATION METHOD THEREFOR AND USE THEREOF
The present invention relates to a recombinant yeast strain having sterol productivity, a preparation method therefor and a use thereof, and more specifically, to a recombinant yeast strain capable of producing cholesterol and cholesterol precursors in a high yield through the deletion of ERG5 and ERG6 genes and the introduction of DHCR24 and DHCR7 genes by codon-optimizing same in multiple or with a codon context method; and a production method therefor and a use thereof. In addition, disclosed are: a method for producing a recombinant yeast strain with increased production yields of cholesterol and cholesterol precursors by the additional introduction of gene tHMG1, ERG2, ERG3, ERG27, or UPC2-1 in the prepared recombinant yeast strain; and a use thereof
RECOMBINANT YEAST STRAIN HAVING STEROL PRODUCTIVITY, PREPARATION METHOD THEREFOR AND USE THEREOF
The present invention relates to a recombinant yeast strain having sterol productivity, a preparation method therefor and a use thereof, and more specifically, to a recombinant yeast strain capable of producing cholesterol and cholesterol precursors in a high yield through the deletion of ERG5 and ERG6 genes and the introduction of DHCR24 and DHCR7 genes by codon-optimizing same in multiple or with a codon context method; and a production method therefor and a use thereof. In addition, disclosed are: a method for producing a recombinant yeast strain with increased production yields of cholesterol and cholesterol precursors by the additional introduction of gene tHMG1, ERG2, ERG3, ERG27, or UPC2-1 in the prepared recombinant yeast strain; and a use thereof
METHODS FOR REGULATING ENDOGENOUS PRODUCTION OF LACTOFERRIN AND SUB-PEPTIDES THEREOF
The present disclosure relates to the composition of one or more agents, therapies, treatments, and methods of use of the agents and/or therapies and/or treatments for upregulating production of lactoferrin or a sub-peptide of lactoferrin. Embodiments of the present disclosure can be used as a therapy or a treatment of adhesions or scarring.
METHODS FOR REGULATING ENDOGENOUS PRODUCTION OF LACTOFERRIN AND SUB-PEPTIDES THEREOF
The present disclosure relates to the composition of one or more agents, therapies, treatments, and methods of use of the agents and/or therapies and/or treatments for upregulating production of lactoferrin or a sub-peptide of lactoferrin. Embodiments of the present disclosure can be used as a therapy or a treatment of adhesions or scarring.
GENE THERAPY
Use of cyclosporin H (CsH) or a derivative thereof for increasing the efficiency of transduction of an isolated population of cells by a viral vector and/or increasing the efficiency of gene editing of an isolated population of cells when transduced by a viral vector.