The present invention provides polynucleotide vectors for high expression of heterologous genes. Some vectors further comprise novel transposons and transposases that further improve expression. Further disclosed are vectors that can be used in a gene transfer system for stably introducing nucleic acids into the DNA of a cell. The gene transfer systems can be used in methods, for example, gene expression, bioprocessing, gene therapy, insertional mutagenesis, or gene discovery.

The present invention provides polynucleotide vectors for high expression of heterologous genes. Some vectors further comprise novel transposons and transposases that further improve expression. Further disclosed are vectors that can be used in a gene transfer system for stably introducing nucleic acids into the DNA of a cell. The gene transfer systems can be used in methods, for example, gene expression, bioprocessing, gene therapy, insertional mutagenesis, or gene discovery.

Provided are a drug and method for treating temporal lobe epilepsy (TLE) in a subject, the method comprising: administering a nucleic acid encoding a NeuroD1 polypeptide to glial cells in hippocampus region of the subject, so that the glial cells express NeuroD1 polypeptides and are transformed into GABAergic neurons. Also provided are a drug and method for forming GABAergic neurons in hippocampus region of a living mammalian brain, the method comprising: administering a nucleic acid encoding NeuroD1 polypeptides to glial cells located in hippocampus region, so that the glial cells express NeuroD1 polypeptides and are transformed into GABAergic neurons.

Aspects of the disclosure relate to compositions (e.g., nucleic acids, rAAV vectors, rAAVs, etc.) and methods for treating neurological diseases including Canavan disease. The disclosure is based, in part, on nucleic acids encoding an aspartoacylase (ASPA) operably linked to a mouse ASPA (mAspa) promoter. Aspects of the disclosure also provide methods of treating neurological diseases including Canavan disease by administering the nucleic acids to a subject.

Aspects of the disclosure relate to compositions and methods for expressing one or more Ganglioside GM3 synthase (GM3S) isoforms in a cell or subject. In some aspects, the disclosure relates to methods for treating GM3 synthase deficiency in a subject in need thereof.

The present invention provides polynucleotide vectors for high expression of heterologous genes. Some vectors further comprise novel transposons and transposases that further improve expression. Further disclosed are vectors that can be used in a gene transfer system for stably introducing nucleic acids into the DNA of a cell. The gene transfer systems can be used in methods, for example, gene expression, bioprocessing, gene therapy, insertional mutagenesis, or gene discovery.