Patent classifications
C12N15/68
Artificial nucleic acid molecules
The invention relates to an artificial nucleic acid molecule comprising at least one open reading frame and at least one 3′-untranslated region element (3′-UTR element) comprising a nucleic acid sequence which is derived from the 3′-UTR of a FIG4 gene or from a variant of the 3′-UTR of a FIG4 gene. The invention further relates to the use of such an artificial nucleic acid molecule in gene therapy and/or genetic vaccination. Furthermore, the invention relates to the use of a 3′-UTR element comprising a nucleic acid sequence which is derived from the 3′-UTR of a FIG4 gene or from a variant of the 3′-UTR of a FIG4 gene for the stabilization and/or prolongation of protein expression from a nucleic acid sequence comprising such 3′-UTR element.
Artificial nucleic acid molecules
The invention relates to an artificial nucleic acid molecule comprising at least one open reading frame and at least one 3′-untranslated region element (3′-UTR element) comprising a nucleic acid sequence which is derived from the 3′-UTR of a FIG4 gene or from a variant of the 3′-UTR of a FIG4 gene. The invention further relates to the use of such an artificial nucleic acid molecule in gene therapy and/or genetic vaccination. Furthermore, the invention relates to the use of a 3′-UTR element comprising a nucleic acid sequence which is derived from the 3′-UTR of a FIG4 gene or from a variant of the 3′-UTR of a FIG4 gene for the stabilization and/or prolongation of protein expression from a nucleic acid sequence comprising such 3′-UTR element.
STABILIZER FOR ADENO-ASSOCIATED VIRUSES AND METHOD FOR STABILIZING ADENO-ASSOCIATED VIRUSES BY USING SAME
Proposed are a stabilizer for an adeno-associated virus (AAV) and a method for stabilizing an adeno-associated virus using the same. More particularly, the present disclosure relates to a stabilizer for an adeno-associated virus including a surfactant or albumin, an adeno-associated virus liquid formulation including adeno-associated virus the stabilizer, and a production method for an adeno-associated virus with improved stability. The technology, according to the present disclosure, inhibits excessive aggregation between viruses when producing a gene delivery virus and therapeutic nanoparticles using an adeno-associated virus and forming an additional polyphenol binder for this purpose, thereby maintaining the particle size to an effective size for drug delivery and improving the stability in the liquid phase. Therefore, the technology is expected to be very useful in the field of drug delivery technology for gene therapy.
STABILIZER FOR ADENO-ASSOCIATED VIRUSES AND METHOD FOR STABILIZING ADENO-ASSOCIATED VIRUSES BY USING SAME
Proposed are a stabilizer for an adeno-associated virus (AAV) and a method for stabilizing an adeno-associated virus using the same. More particularly, the present disclosure relates to a stabilizer for an adeno-associated virus including a surfactant or albumin, an adeno-associated virus liquid formulation including adeno-associated virus the stabilizer, and a production method for an adeno-associated virus with improved stability. The technology, according to the present disclosure, inhibits excessive aggregation between viruses when producing a gene delivery virus and therapeutic nanoparticles using an adeno-associated virus and forming an additional polyphenol binder for this purpose, thereby maintaining the particle size to an effective size for drug delivery and improving the stability in the liquid phase. Therefore, the technology is expected to be very useful in the field of drug delivery technology for gene therapy.
MODIFICATION OF RNA, PRODUCING AN INCREASED TRANSCRIPT STABILITY AND TRANSLATION EFFICIENCY
It was the object of the present invention to provide RNA with increased stability and translation efficiency and means for obtaining such RNA. It should be possible to obtain increased grades of expression by using said RNA in gene therapy approaches.
MODIFICATION OF RNA, PRODUCING AN INCREASED TRANSCRIPT STABILITY AND TRANSLATION EFFICIENCY
It was the object of the present invention to provide RNA with increased stability and translation efficiency and means for obtaining such RNA. It should be possible to obtain increased grades of expression by using said RNA in gene therapy approaches.
METHYLENE BLUE STABILIZED MRNA COMPOSITIONS
Formulations of lipids and nucleic acids, including lipid nanoparticle formulations which encapsulate nucleic acids, stabilized with phenothiazinium dyes. In particular, the stabilization of LNP-mRNA formulations with methylene blue, Azure A, Azure B, Safranin O, phenosafranin or leucomethylene blue. Methods of making and of use of the formulations stabilized by chemical compounds are also provided.
METHYLENE BLUE STABILIZED MRNA COMPOSITIONS
Formulations of lipids and nucleic acids, including lipid nanoparticle formulations which encapsulate nucleic acids, stabilized with phenothiazinium dyes. In particular, the stabilization of LNP-mRNA formulations with methylene blue, Azure A, Azure B, Safranin O, phenosafranin or leucomethylene blue. Methods of making and of use of the formulations stabilized by chemical compounds are also provided.
MOLECULES AND METHODS FOR INCREASED TRANSLATION
Nucleic acid molecule comprising a coding sequence and a region of increased folding energy upstream of a stop codon are provided. Expression vectors and cells comprising the nucleic acid molecule are also provided. Methods for optimizing a coding sequence comprising increasing folding energy in a region upstream of the stop codon are also provided.
MOLECULES AND METHODS FOR INCREASED TRANSLATION
Nucleic acid molecule comprising a coding sequence and a region of increased folding energy upstream of a stop codon are provided. Expression vectors and cells comprising the nucleic acid molecule are also provided. Methods for optimizing a coding sequence comprising increasing folding energy in a region upstream of the stop codon are also provided.