Abstract: The present disclosure relates to AAV gene therapy vectors, AAV replicons, and pharmaceutical compositions for delivering a human CYP2U1 gene to a subject for treating hereditary spastic paraplegias, especially SPG56. In addition, methods of treatment and gene transfer are also provided as well as minimally invasive biomarkers for monitoring disease progression and other uses.

Abstract: The disclosure relates to nucleic acid expression cassettes and vectors for the treatment of neurodegenerative disorders. Methods of treating neurodegenerative disorders such as Alzheimer's disease, frontotemporal dementia, frontotemporal lobar degeneration, Pick's disease, Lewy body dementia, memory loss, cognitive impairment, and mild cognitive impairment are also provided.

Abstract: The disclosure relates to nucleic acid expression cassettes for the treatment of neurodegenerative disorders. Methods of treating neurodegenerative disorders such as Alzheimer's disease, frontotemporal dementia, frontotemporal lobar degeneration, Pick's disease, Lewy body dementia, memory loss, cognitive impairment, and mild cognitive impairment are also provided.

Abstract: The present disclosure relates to AAV gene therapy vectors, AAV replicons, and pharmaceutical compositions for delivering a human HDAC8 gene to a subject for treating Cornelia de Lange Syndrome. In addition, methods of treatment and gene transfer are provided.

Abstract: The invention provides a method of determining the likelihood that a smoker will or will not develop chronic obstructive pulmonary disease (COPD) by obtaining a sample from the smoker, analyzing the sample for the expression of a set of biomarkers associated with COPD, and comparing the expression pattern determined in the sample with a standard expression pattern to determine the likelihood that the smoker will or will not develop COPD. The invention further provides a composition, a method of treatment, and methods of determining the efficacy of treatment for COPD.

Abstract: The invention provides a gene transfer vector comprising a humanized nucleic acid sequence encoding an immunogenic portion of one or more exotoxins of Bacillus anthracis and a heterologous sorting signal. The invention also provides a method of producing an immune response against Bacillus anthracis in a host comprising administering to the host the gene transfer vector.

Abstract: The invention provides an isolated or purified nucleic acid molecule comprising SEQ ID NO:1, which contains cDNA comprising exons 1-5 of a vascular endothelial growth factor (VEGF) joined to genomic DNA comprising introns 5, 6, and 7 and exons 6, 7, and 8 of VEGF and a mutation in one or more of the splice donor, branch point, and splice acceptor regions, which promotes the production of a VEGF189 isoform. Expression constructs, compositions, and cells comprising such a nucleic acid molecule also are provided by the invention.

Abstract: The present invention provides a method of expressing an exogenous nucleic acid in a mammal. The method comprises non-systemically administering to a non-neuronal tissue of said mammal an exogenous nucleic acid operatively linked to a promoter. The exogenous nucleic acid is proximal to at least one native parvoviral inverted terminal repeat and does not require encapsidation. The expression of the exogenous nucleic acid in the tissue is not substantially diminished at 28 days after administration of the exogenous nucleic acid.