Pharmaceutical preparations for the treatment of neuromuscular diseases and disorders, muscular dystrophies, central nervous system diseases and disorders, genetic diseases and disorders; pharmaceutical preparations based on morpholino oligomer technology platform for the treatment of neuromuscular diseases and disorders, muscular dystrophies, central nervous system diseases and disorders, genetic diseases and disorders, and for the regulation of protein production; pharmaceutical preparations that modulate RNA expression for the treatment of neuromuscular diseases and disorders, muscular dystrophies, central nervous system diseases and disorders, genetic diseases and disorders, and for the regulation of protein production; gene therapy products, namely, gene delivery pharmaceuticals; pharmaceutical preparations for gene therapy, gene editing, and genome editing; gene therapy products for the treatment of neuromuscular diseases and disorders, muscular dystrophies, central nervous system diseases and disorders, genetic diseases and disorders; biological preparations for the treatment of neuromuscular diseases and disorders, muscular dystrophies, central nervous system diseases and disorders, genetic diseases and disorders; viruses, viral vectors and viral constructs for the treatment of neuromuscular diseases and disorders, muscular dystrophies, central nervous system diseases and disorders, genetic diseases and disorders; pharmaceutical preparations, biological preparations and gene therapy products in the nature of pharmaceutical and biological preparations for the treatment of genetic disorders and diseases.