8 results about "Piggybac transposon" patented technology

The invention relates to the technical field of gene engineering, in particular to a system and a method for gene editing by using human induced pluripotent stem cells. By means of the precise integration characteristic of the PiggyBac transposon, the target gene can be stably integrated into the genome of the iPSC cell, then the iPSC cell integrated with the target gene is subjected to directional induced differentiation, continuous passage expression can be achieved, the problem of expression attenuation of a traditional transient expression system is solved, the expression level of the target gene is improved, and the yield of target protein is increased.

The present disclosure relates to compositions and methods for treating phenylketonuria (PKU). In particular, the present disclosure relates to AAV – piggyBac transposon polynucleotide vectors and LNP compositions comprising a nucleic acid encoding a transposase, and methods of using the compositions for treating PKU.

The invention discloses a construction method and application of a sperm-specific hOdf2-GFP transgenic mouse model, and relates to the technical field of reproductive biology and transgenic animal model construction. According to the construction method disclosed by the invention, based on a piggyBac transposon expression vector system, a DNA (Deoxyribose Nucleic Acid) sequence containing a coding human source Odf2 is effectively connected with a Protamine1 promoter. The transgenic positive mouse constructed by the method can specifically express human Odf2, has a dynamic change from sperm to zygote to eight cell stages, realizes early continuous visual tracking of embryos, has genetic stability, keeps expression and phenotype consistency across generations, and has a good application prospect. The method can be applied to the fields of environmental poison evaluation, asthenospermia mechanisms, drug screening and the like.

The present disclosure relates to compositions and methods for treating phenylketonuria (PKU). In particular, the present disclosure relates to AAV-piggyBac transposon polynucleotide vectors and LNP compositions comprising a nucleic acid encoding a transposase, and methods of using the compositions for treating PKU.

The application discloses a preparation method of cat induced pluripotent stem cells, which comprises the following steps: constructing piggyBac transposon plasmids carrying cat source OCT4, SOX2, KLF4 and c-MYC four factors in series, piggyBac transposon plasmids carrying cat source or pig source NANOG and LIN28 two factors in series and piggyBac transposon plasmids carrying monkey kidney virus SV40LT plasmids, and transfecting the plasmids into cat fetal fibroblasts; picking up cloned cells with embryonic stem cell morphologies, and carrying out subculture of cell clones meeting the characteristics of embryonic stem cells through identification and screening, so that the cat induced pluripotent stem cells are obtained. The preparation method of the cat induced pluripotent stem cells is safe, efficient and has good differentiation potential, lays a scientific foundation for researching in-vitro culture conditions of feline ESCs and establishment of feline iPSCs, and lays a scientific foundation for further cultivating cloned cats and gene modified cats and researching in related disease mechanisms, screening drugs and regeneration and repair.

The invention relates to an optimized Piggybac transposon nucleic acid construct and an application of the optimized Piggybac transposon nucleic acid construct in the fields of cell therapy and biological pharmacy. According to the system, the integration efficiency is remarkably improved by shortening the sequence length of a PiggyBac transposon nucleic acid construct, using a smaller plasmid skeleton, adjusting an insulator sequence and optimizing a promoter and an enhancer. Experimental results show that the optimized vector can significantly improve the CAR positive rate, enhance the cell amplification ability and maintain the stability of CAR expression. The method has an important application prospect.

The present disclosure relates to compositions and methods for treating phenylketonuria (PKU). In particular, the present disclosure relates to AAV-piggyBac transposon polynucleotide vectors and LNP compositions comprising a nucleic acid encoding a transposase, and methods of using the compositions in the treatment of PKU.