Human genetics has evolved considerably since the discovery of DNA. New gene- and biotechnologies invented for the last three decades have inspired novel treatments for diseases that were once accepted as untreatable. Congenital neuromuscular disorders and neurodegenerative diseases are typical examples of these diseases. Novel emerging trials demonstrate promising results to alter the poor prognosis of these unfortunate patients.
Keywords: Genetics, neurogenetics, gene technologies, biotechnology, gene therapy