The present disclosure provides nucleic acid molecules comprising a first inverted terminal repeat (ITR), a second ITR, and a genetic cassette encoding a target sequence. In some embodiments, the first ITR and/or the second ITR is an ITR of human bocavirus. Also disclosed are methods of using the nucleic acid molecules in gene therapy applications.

Compositions and methods for treating disorders affecting motor function, such as motor function affected by disease or injury to the brain and/or spinal cord, are disclosed.

The invention provides methods for the production of recombinant adeno-associated virus vectors (rAAV), comprising contacting a host cell with a solution comprising at least one compound of formula (I), (I-A), (I-B), (II), (III), or (IV), or a salt thereof, or a vitamin B, or any combination(s) thereof. Also provided are methods for increasing the production of rAAV by a host cell, comprising contacting a host cell with a solution comprising at least one compound of formula (I), (I-A), (I-B), (II), (III), or (IV), or a salt thereof, or a vitamin B, or any combination(s) thereof.

Provided herein are double strand DNA molecules comprising inverted repeats, expression cassette and one or more restriction sites for nicking endonucleases, the methods of use thereof, and the methods of making therefor.

Described herein are constructs used for liver-specific expression of a transgene.

The present disclosure provides novel vectors and methods useful in treating genetic diseases, brain disorders, and neurological diseases and disorders, including gene therapy vectors and methods of administering such to a subject in need thereof.

The present invention provides for a process for producing a plurality of SUMOylation target-site modified AAV vectors, SUMOylation target-site modified AAV vectors, and application of the SUMOylation target-site modified AAV vectors in gene therapy. The present invention provides for manipulation of SUMOylation specific amino acids on AAV2 capsid protein, thereby regulating role of the same. Furthermore, the development of SUMOylation target modified AAV2 vectors presents an exciting opportunity for hepatic or ocular gene transfer with the safest AAV vector in human gene therapy applications. The plurality of SUMOylation target-site modified AAV vectors are not immunogenic in comparison to wildtype AAV2 vectors and possess significantly higher gene expression, with respect to wild type, thereby improving efficiency of hepatic and ocular gene transfer.

The present invention concerns a new gene therapy approach to increase light-sensitivity in degenerating cones in advanced stages of rod-cone dystrophy (RCD) mediated by G-protein-gated-K+ channel (GIRK), in particular GIRK2, activated by G proteins recruited by cone opsin expressed in degenerating cones.

Described herein are muscle-specific targeting moieties and compositions including the muscle specific targeting motifs. Also described herein are uses of the muscle-specific targeting motifs and compositions including the muscle specific targeting moieties. In some embodiments, the muscle-specific targeting moieties and compositions including the muscle specific targeting moieties can be used to direct delivery of a cargo to a muscle cell.

The present disclosure relates to methods of producing monoclonal antibodies in animals. In particular, the disclosure provides a method of producing, in vivo, antibodies against viral capsids (VCs) derived from a non-enveloped virus (NEV). The method includes administering to a subject, by hydrodynamic-based transfection, a first set of genetic material encoding NEV structural proteins to induce the subject's intracellular translation and assembly of the proteins into viral capsids. The method also includes administering a second set of genetic material encoding NEV non-structural proteins to facilitate the intracellular assembly of the NEV structural proteins. Thus, this method may be used to produce subject-generated antibody-producing cells that secrete anti-VC antibodies that may be harvested and screened for monoclonal anti-VC antibodies.