10 results about "Minicircle" patented technology

Aspects of the disclosure relate to methods for producing recombinant adeno-associated virus (rAAV) particles. The disclosure is based, in part, on minicircle DNA dual-transfection, which in some embodiments, significantly reduces plasmid consumption, vector impurity, and empty capsid formation, while at the same time generates a comparable or higher rAAV titer.

Current minicircle production methods are slow, expensive, and difficult to perform under GMP conditions because, in most cases, the product is derived from bacteria. In contrast, HTLA-and CHTLA-based synthetic circular supercoiled DNA production can be done completely in a test tube, using chemically or enzymatically synthesized oligonucleotides, long single stranded DNA, and / or double stranded DNA.

The present invention relates to extracellular vesicles (EVs) derived from human dendritic cells (hDCs) comprising a RVG-hLAMP peptide construct, having sequence SEQ ID NO. 1, expressed on the surface thereof, wherein the extracellular vesicles are loaded with anti-α-synuclein shRNA minicircles (ShRNA-MCs). Likewise, the invention relates to the clinical use of said vesicles in the treatment of synucleinopathies. Lastly, the present invention relates to a method for producing the extracellular vesicles from human dendritic cells (hDCs).

Present methods for the production of microrings are slow, expensive and difficult to carry out under GMP conditions, because in most cases, the product is derived from bacteria. In contrast, synthetic circular superhelix DNA production based on HTLA and CHTLA can be completed completely in a test tube, using chemically or enzymatically synthesized oligonucleotides, long single-stranded DNA, and / or double-stranded DNA.

Methods and materials for making and using extracellular vesicles (EVs) engineered to include BMP2 or BMP2 in combination with BMP7, and cells that can be used to generate such EVs, are provided herein. For example, methods and materials for generating EVs that include BMP2 or BMP2 and BMP7, and methods and materials for using such EVs to induce bone formation, are provided. Methods and materials for making and using minicircle DNAs (mcDNAs) to overexpress BMP2 or BMP2 and BMP7 also are provided herein. For example, methods and materials for generating mcDNAs that encode BMP2 or BMP2 and BMP7, and methods and materials for using such mcDNAs to induce bone formation, are provided.

The present disclosure relates to plasmids that may be used to promote prolonged expression of recombinant proteins in vivo, such as in animal models, and their methods of construction and use, for instance in animals. In some embodiments, the plasmids, following injection into an animal, are converted to minicircles by recombination, such as in the liver of the animal.

A conjugated composition and methods of using the same for treatment of a living cell, are provided. The conjugated composition includes a nucleic acid sequence directly or indirectly conjugated to at least one N-Acetylgalactosamine (GalNAc) molecule, or to a derivative of a GalNAc molecule. The conjugation is provided by a linker. The conjugated composition is structured to enable the at least one GalNAc molecule or derivative to deliver the nucleic acid sequence into a cell, for example, into a liver cell. The nucleic acid sequence can be a therapeutic supercoiled DNA minicircle that has a therapeutic gene for delivery to the cell.

The present disclosure relates to methods for producing minicircle DNA (mcDNA) and methods of using the mcDNA as a scaffold to produce R-loops. Further disclosed are methods of screening for an agent that binds an R-loop.

Current minicircle production methods are slow, expensive, and difficult to perform under GMP conditions because, in most cases, the product is derived from bacteria. In contrast, HTLA- and CHTLA-based synthetic circular supercoiled DNA production can be done completely in a single container, for example, using precursor DNA fragments generated using rolling circle amplification.

The present invention relates to extracellular vesicles (EVs) derived from human dendritic cells (hDCs) comprising a RVG-hLAMP peptide construct, having sequence SEQ ID NO. 1, expressed on the surface thereof, wherein the extracellular vesicles are loaded with anti-α-synuclein shRNA minicircles (ShRNA-MCs). Likewise, the invention relates to the clinical use of said vesicles in the treatment of synucleinopathies. Lastly, the present invention relates to a method for producing the extracellular vesicles from human dendritic cells (hDCs).