The present invention relates to a polynucleotide comprising at least one promoter and an S/MAR element, wherein said S/MAR element is located downstream of said promoter in the 3 UTR of the transcription unit and wherein the said S/MAR element is flanked by a 5 splice donor site and a 3 splice acceptor site; the present invention further relates to a composition comprising said polynucleotide, and to the polynucleotide for use in medicine and for use in treating genetic disease.

The present invention relates to the field of self-replicating non-integrative episomal vertebrate expression vectors useful for in gene therapy, ex vivo cell therapy, stem cell therapy, and more particularly, for improving the expression of vector encoded antigens or therapeutic genes. Such recombinant DNA molecules are useful in biotechnology, transgenic organisms, gene therapy, stem cell therapy, therapeutic vaccination, agriculture and DNA vaccines. More specifically, relates to a polynucleotide comprising at least one promoter and an S/MAR element, wherein said S/MAR element is located downstream of said promoter and wherein the nucleic acid sequence of said S/MAR element (S/MAR sequence) comprises at least 3 sequence motifs ATTA (SEQ ID NO:1) per 100 nucleotides over a stretch of at most 200 nucleotides; the present invention further relates to a composition and to a host cell comprising said polynucleotide, and to the polynucleotide for use in medicine and for use in treating genetic disease. The present invention also relates to a kit and to a device comprising said polynucleotide, and to methods and uses related to the polynucleotide.

Disclosed are systems and methods for whole-genome replacement through a massively multiplexed homologous template repair process. Disclosed aspects include a method of substantially changing a DNA sequence of an organism, the method including one or more of the following steps: determining a desired DNA sequence, the desired DNA sequence being a DNA sequence to which it is desired that the DNA sequence of the organism be substantially changed; preparing a treatment configured to cause the organism DNA sequence to be substantially changed to the desired DNA sequence; applying the treatment to the organism; wherein the treatment is configured to cause the organism DNA sequence to be substantially changed to the desired DNA sequence by causing, at each of multiple sites in the organism DNA sequence, genetic code at the site to be substantially changed to genetic code at a responding site in the desired DNA sequence; each of the multiple sites in the organism DNA sequence is a respective sub-sequence of the organism DNA sequence; applying the treatment includes delivering to the organism at least one dose; and each dose includes respective change agent material that causes the changing of the genetic code at a respective plurality of the multiple sites. In certain embodiments, the DNA sequence of the organism is a whole-genome DNA sequence of the organism, and the desired DNA sequence is substantially the organism's germline whole-genome DNA sequence, an intentionally modified version of the organism's germline whole-genome DNA sequence, or a whole-genome DNA sequence of another organism.

Disclosed herein include methods, compositions, culture media, and devices for in vitro culture of synthetic embryos from mammalian pluripotent stem cells. The in vitro embryo model is generated with embryonic and extraembryonic lineages derived from embryonic stem cells through transcription-factor-mediated reprogramming and can undergo advanced development to late headfold stages.

The present invention provides an isolated nucleic acid molecule comprising, or consisting of, the nucleic acid sequence of SEQ ID NO:1 or a nucleic acid sequence of at least 400 bp having at least 80% identity to said sequence of SEQ ID NO:1, wherein said isolated nucleic acid molecule specifically leads to the expression in direction selective retinal ganglion cells of a gene when operatively linked to a nucleic acid sequence coding for said gene.

The present disclosure relates to compositions and methods for tissue regeneration.

Improved DNA vaccine plasmids are disclosed that contain novel immunostimulatory RNA compositions. The improved plasmids eliminate all extraneous sequences, incorporate a novel antibiotic free short RNA based selectable marker, increase eukaryotic expression using a novel chimeric promoter, improve yield and stability during bacterial production, and improve immunostimulation. These vectors are utilized in immunization to elicit improved immune responses or therapy to induce type 1 interferon production.

The present invention relates to a polynucleotide comprising at least one promoter and an S/MAR element, wherein the S/MAR element is located downstream of the promoter in the 3 UTR of the transcription unit and wherein the S/MAR element is flanked by a 5 splice donor site and a 3 splice acceptor site; the present invention further relates to a composition comprising the polynucleotide, and to the polynucleotide for use in medicine and for use in treating genetic disease.

Provided herein are methods of co-culturing a myogenic cell with a support cell to increase myotube formation from the myogenic cells. For example, provided herein is a method comprising co-culturing a myogenic cell with a support cell wherein the support cell comprises a polynucleotide comprising a coding sequence of a gene of interest; and culturing the myogenic cell and the support cell in a cultivation infrastructure under conditions sufficient to induce proliferation of the myoblast cell.