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Therapeutic pharmaceutical compositions comprised of adeno-associated viral (AAV) vectors and recombinant adeno-associated viral (rAAV) vectors for therapeutic effect in gene therapy, namely, gene inhibition therapy and/or RNA interference (RNAi), for the treatment of genetic diseases and disorders and neurological diseases and disorders; therapeutic pharmaceutical compositions comprised of adeno-associated viral (AAV) vectors and recombinant adeno-associated viral (rAAV) vectors for therapeutic effect in gene therapy, namely, gene augmentation therapy, for the treatment of genetic diseases and disorders and neurological diseases and disorders; therapeutic pharmaceutical compositions comprised of adeno-associated viral (AAV) vectors and recombinant adeno-associated viral (rAAV) vectors for therapeutic effect in gene therapies using intrathecal administration, for the treatment of genetic diseases and disorders and neurological diseases and disorders; biologic preparations for use in the treatment of genetically based diseases and disorders; biologic preparations for medical purposes, namely, for gene therapy for treating genetic disorders; pharmaceutical preparations that modulate RNA expression for the treatment of neurological diseases and disorders, genetic diseases and disorders, and for the regulation of protein production; gene therapy products, namely, gene delivery pharmaceuticals for the treatment of genetic diseases and disorders and neurological diseases and disorders; pharmaceutical preparations for gene therapy, gene inhibition and gene augmentation, for the treatment of genetic diseases and disorders and neurological diseases and disorders; viruses, viral vectors and viral constructs, all in the nature of pharmaceutical preparations, for the treatment of neurological diseases and disorders and genetic diseases and disorders; pharmaceutical preparations for gene therapy, for the treatment of genetic diseases and disorders and neurological diseases and disorders; pharmaceutical and biopharmaceutical agents for the treatment of genetic disorders and neurological diseases and disorders; pharmaceutical preparations, namely, nucleic acid therapeutics for the treatment of genetic disorders and neurological diseases and disorders; pharmaceutical preparations, namely, DNA and/or RNA therapeutics for the treatment of genetic disorders and neurological diseases and disorders

Development of pharmaceutical preparations for use in gene therapy; genetic engineering and biotechnology research; research and development of new products in the field of gene therapeutics; scientific and medical research in the field of genetics, gene therapy and genetic engineering; laboratory research in the fields of genetics, gene therapy, drug development, biotechnology, microbiology and molecular biology; pharmaceutical drug discovery; scientific research in the field of gene therapy discovery; drug development and therapeutic drug development; genetic drug development and therapeutic drug development; biological research; biological drug development and scientific research in the field of biological development