The present application discloses methods for synthetic production and cell-free synthesis of DNA vectors, particularly closed-ended linear DNA vectors having one or more gaps (e.g., nicked ceDNA vectors, “neDNA”) and adenoassociated-virus (AAV) vector which is single strand DNA having linear and continuous structure, for delivery and expression of a transgene in the host cell. The present invention also relates to an in vitro process for production of closed-ended DNA vectors, corresponding DNA vector products produced by the methods and uses thereof, and oligonucleotides and kits useful in the process of the present invention.

Disclosed herein are contiguous DNA sequences encoding highly compact multi-input genetic logic gates for precise in vivo cell targeting, and methods of treating disease using a combination of in vivo delivery and such contiguous DNA sequences.

The disclosure relates to optimized polynucleotide sequences for LAMP-2B, expression cassettes, vectors, and methods of use thereof in treating disease, e.g. Danon disease.

The present invention relates to viral particles which exhibit self-regulatory or regulatable features.

Provided herein are compositions of recombinant adeno-associated virus (rAAV) particles capable of dampening the humoral immune response against rAAV in the host into which the rAAV particles are introduced. The modified genomes of these rAAV particles comprise heterologous insert nucleic acid and/or inverted terminal repeat (ITR) sequences containing one or more sequences associated with the human leukocyte antigen gene complex DR (HLA-DR) promoter. Also provided herein are methods for transducing host cells with modified rAAV particles to induce a dampened humoral immune responses in order to improve transduction efficiencies. Also provided herein are complexes comprising a modified rAAV particle and a Regulatory Factor X (RFX) transcription factor.

A new promoter comprising: (i) an hCMV enhancer sequence; (ii) an hCMV promoter sequence; (iii) a splice donor region; (iv) a cell-derived enhancer sequence; and (v) a splice acceptor region.

The present inventions generally relate to site-specific delivery of nucleic acid modifiers and includes novel DNA-binding proteins and effectors that can be rapidly programmed to make site-specific DNA modifications. The present inventions also provide synthetic all-in-one genome editor (SAGE) systems comprising designer DNA sequence readers and a set of small molecules that induce double-strand breaks, enhance cellular permeability, inhibit NHEJ and activate HDR, as well as methods of using and delivering such systems.

Provided are methods for reducing the risk of occurrence and/or the severity of pathogenic diseases (e.g., diseases associate with varicella zoster virus reactivation, influenza infection, and/or S. pneumoniae infection) in a subject that is receiving a gene therapy and an accompanying immunosuppressant regimen.

This application describes transcriptional units, synthetic expression systems, and host cells comprising transcriptional units and synthetic expression systems, wherein the synthetic expression system is capable of expressing a gene of interest. Also described are methods for the production of bioproducts (including, but not limited to, proteins or RNA expressed from the gene of interest). In some embodiments, bioproducts are produced from host cells under culture conditions without addition of methanol.

Provided is a polynucleotide, including a cis-regulatory element and a nucleotide sequence encoding a vestigial like 4 protein, wherein the cis-regulatory element includes an uncoupling protein 1 enhancer and an uncoupling protein 1 promoter. Also provided is a viral vector including said polynucleotide. Also provided is a method of transfecting a cell or a subject with said polynucleotide or said viral vector.