7 results about "Inverted Terminal Repeat" patented technology

Data pipelines for string extraction, clustering, and comparison are disclosed. A method can include extracting sequence regions from each plasmid genome sequence sequenced from a genome of a plasmid based on presence of a fixed flanking sequence marker (FFSM) in the plasmid genome sequence. Each sequence region is within the FFSM and includes a candidate inverted terminal repeat (ITR) sequence. An example method further includes clustering two or more of the sequence regions based on perfect sequence identity to generate clusters; merging two or more of the clusters based on alignments between corresponding sequence regions of the two or more clusters; when a single cluster remains, identifying a genotype of a candidate ITR sequence of the single cluster based on local alignments; and using a plasmid having an ITR sequence with the genotype of the candidate ITR sequence to manufacture a plurality of AAV vectors based on the genotype of the candidate ITR sequence.

Provided is a method for producing a protein / nucleic acid complex which comprises a capsid protein derived from an adeno-associated virus and a genomic nucleic acid having at least one inverted terminal repeat sequence and has infectiousness to mammals. The genomic nucleic acid further includes a target nucleic acid that encodes a target protein. The method comprises: (a-1) a step for providing an isolated first nucleic acid that comprises a linear single-stranded nucleic acid, a linear double-stranded nucleic acid, or a circular single-stranded nucleic acid and includes at least one inverted terminal repeat sequence and a target nucleic acid that encodes a target protein; (a-2) a step for providing an isolated first nucleic acid that includes at least one inverted terminal repeat sequence and a target nucleic acid that encodes a target protein; and (b) a step for bringing a first protein into contact with the first nucleic acid in a solvent to form the protein / nucleic acid complex.

Provided are methods for treating an ocular neovascular disease in an individual by reducing Auction of central subfoveal thickness (CST) or central retinal thickness (CRT), comprising administering a unit dose of recombinant adeno-associated virus (rAAV) particles to an eye of the individual, wherein the rAAV particles comprise: a) a nucleic acid encoding a polypeptide comprising an amino acid sequence with at least about 95% identity to the amino acid sequence of SEQ ID NO: 35 and Ranked by AAV2 inverted terminal repeats (ITRs), and b) an AAV2 capsid protein comprising an amino acid sequence LGETTRP (SEQ ID NO: 14) inserted between positions 587 and 588 of the capsid protein, wherein the amino acid residue numbering corresponds to an AAV2 VP1 capsid protein.

Herein is reported a method for producing recombinant adeno-associated viral particle preparation (rAAVp) comprising the step of cultivating a mammalian cell comprising expression cassettes for a non-adeno-associated viral gene, which is interspaced between two AAV inverted terminal repeats (ITRs), an adeno-associated virus rep gene, an adeno-associated virus cap gene, an adeno-associated virus E1A gene, an adeno-associated virus E1B gene, an adeno-associated virus E2A gene, an adeno-associated virus E4orf6 and an adeno-associated virus VA RNA gene, and thereby producing the rAAVp, wherein the cultivating is at a pH value in the range of and including pH 7.4 to pH 7.6. The yield of the rAAVp produced by the cultivating at a pH value in the range of and including pH 7.4 to pH 7.6 is higher than the yield of a rAAVp produced by a cultivating at a pH value in the range of and including pH 7.0 to pH 7.2 and the rAAVp produced by the cultivating at a pH value in the range of and including pH 7.4 to pH 7.6 has a higher percentage of full particles than a rAAVp produced by a cultivating at a pH value in the range of and including pH 7.0 to pH 7.2.

Disclosed are compositions comprising a first nucleic acid sequence comprising: (a) a first inverted terminal repeat (ITR), (b) a second ITR and (c) an intra-ITR sequence, wherein the intra-ITR sequence comprises a transposon sequence, and a second nucleic acid sequence comprising an inter-ITR sequence, wherein the length of the inter-ITR sequence is between 1 and 600 nucleotides, inclusive of the endpoints. Preferably, the compositions are nanotransposons.

According to various aspects of this disclosure, the present disclosure relates to a method for treating or reducing pulmonary inflammation and / or pulmonary fibrosis in a subject in need thereof comprising intramuscularly administering to the subject a recombinant adeno-associated virus (rAAV) vector. In some aspects, the rAAV vector comprises a vector genome and an AAV capsid (e.g., an AAV1 serotype). In some aspects, the vector genome comprises AAV inverted terminal repeats (ITRs) flanking an expression construct comprising a nucleotide sequence encoding a Fibroblast growth factor 21 (FGF21) or functional fragment thereof operably linked to a ubiquitous promoter.

This disclosure generally relates to ultraminiature transposon inverse repeat (ITR) polynucleotides, compositions comprising said polynucleotides, and methods for using said polynucleotide compositions for exovivo and in vivo delivery of nucleic acids to cells, in particular for in vivo delivery of therapeutic genes for treating hereditary disorders or diseases.