C12N2840/105

RNA CLEAVAGE-INDUCED TRANSCRIPT STABILIZER AND USES THEREOF

Provided herein are genetic circuits and encoded RNA transcripts that produce an output molecule in response to an RNA cleavage event that removes a degradation signal. In some embodiments, the genetic circuits described herein may be used for detecting RNA cleaver activities (e.g., in a cell). Methods of using the genetic circuits described herein in diagnostic or therapeutic applications are also provided.

Hepatocyte based insulin gene therapy for diabetes

A method and vectors for controlling blood glucose levels in a mammal are disclosed. In one embodiment, the method comprises the steps of: treating the hepatocyte cells of a patient with a first, second or third vector, wherein the first vector comprises a promoter enhancer, glucose inducible regulatory elements, a liver-specific promoter, a gene encoding human insulin with modified peptidase and an albumin 3UTR and lacks an HGH intron, wherein the second vector comprises an HGH intron, glucose inducible regulatory elements, a liver-specific promoter, a gene encoding human insulin with modified peptidase site and an albumin 3UTR and lacks a promoter enhancer, wherein the third vector comprises an HGH intron, glucose inducible regulatory elements, a liver-specific promoter, a gene encoding human insulin with modified peptidase site, an albumin 3UTR and a promoter enhancer and observing the patient's insulin levels, wherein the patient's insulin levels are controlled.

MATERIALS AND METHODS FOR MAKING A RECESSIVE GENE DOMINANT
20190300901 · 2019-10-03 · ·

The subject invention provides materials and method for making a recessive gene dominant. This is accomplished by interfering with the natural mechanisms that inhibit expression of the recessive gene and/or by interfering with the expression of the naturally dominant gene. In a preferred embodiment, the method of the subject invention comprises both reducing inhibition of expression of the recessive gene and increasing inhibition of the dominant gene.

Minimal UTR sequences

Described are DNA molecules which can be transcribed into an mRNA harbouring novel UTR sequences combining the advantages of being extremely short and at the same time allowing for high translation efficiencies of RNA molecules containing them. Further, described are vectors comprising such a DNA molecule and to host cells comprising such a vector. Moreover, described are corresponding RNA molecules containing such UTRs. Further, described is a pharmaceutical composition comprising the described RNA molecule and optionally a pharmaceutically acceptable carrier as well as to the use of the described UTRs for translating a coding region of an RNA molecule into a polypeptide or a protein encoded by said coding region.

SPATIOTEMPORAL REGULATORS
20190233844 · 2019-08-01 · ·

Provided herein, in some embodiments, are methods, compositions, systems and kits that enable spatiotemporal regulation of nucleic acid expression in engineered cells.

GENETIC ERASERS
20190169634 · 2019-06-06 ·

Provided herein, in some embodiments, are methods, compositions, systems and kits that enable removal of heterologous nucleic acid from engineered cells.

COMPOSITIONS AND METHODS FOR ENHANCING GENE EXPRESSION

The present disclosure generally relates to nucleic acid molecules for use in regulating gene expression. Disclosed herein include nucleic acid molecules containing one or more structural elements of the viral capsid enhancer operably linked to a coding sequence of a gene of interest. In some embodiments, the viral capsid enhancer comprises a Downstream Loop (DLP) from a viral capsid protein, or a variant of the DLP.

Method of detecting Polycomb Repressive Complex activity

A method of detecting Polycomb Repressive Complex (PRC) activity in a cell providing a cell with a DNA having a protein binding site and at least one reporter gene expression site is operatively connected to the protein binding site, and with a DNA containing a recombinant gene of a binding protein, the binding protein being capable of binding to the protein binding site, wherein the binding protein is fused to a member of the PRC, the method including the step of expressing the recombinant gene, letting the fused binding protein bind to the protein binding site and detecting at least one reporter gene expression.

NOVEL MINIMAL UTR SEQUENCES

Described are DNA molecules which can be transcribed into an mRNA harbouring novel UTR sequences combining the advantages of being extremely short and at the same time allowing for high translation efficiencies of RNA molecules containing them. Further, described are vectors comprising such a DNA molecule and to host cells comprising such a vector. Moreover, described are corresponding RNA molecules containing such UTRs. Further, described in a pharmaceutical composition comprising the described RNA molecule are optionally a pharmaceutically acceptable carrier as well as to the use of the described UTRs for translating a coding region of an RNA molecule into a polypeptide or a protein encoded by said coding region.

RECOMBINANT ZIKA VACCINES

The present invention relates to the provision of immunogenic or vaccine compositions comprising at least one recombinant Zika virus antigen, wherein the at least one recombinant Zika virus antigen is encoded by at least one nucleic acid sequence encoding at least one E-protein of a Zika virus or a functional fragment thereof. Further provided are nucleic acid molecules and a recombinant chimeric virus encoding and/or comprising selected antigens from a Zika virus, which are suitable as vaccine compositions. Preferably, the sequences encoding at least one Zika virus antigens suitable for eliciting an immune response are operably linked to a non-flavivirus derived vector backbone. Further provided are methods for purifying the recombinant chimeric virus particles or the immunogenic composition. Finally, there is provided an immunogenic/vaccine composition for use in a method of preventing or treating a Zika virus disease.