In recent years, there has been an increasing prevalence of neurodegenerative diseases worldwide. This is linked to the increasing age of the world population. Despite intensive research and clinical trials, in most cases no effective therapy has been developed to date. Typically, management of neurological disorders only alleviates the severity of symptoms without eliminating the cause of the disease. The development of new treatments for neurodegenerative diseases is hampered due to the limited permeability of the BBB; as the penetration of drugs into the central nervous system is restricted. For these reasons, it is crucial to develop therapeutic strategies that can be applied directly at the pathologically altered site. In addition, a major challenge is to develop a treatment that inhibits disease progression. Numerous studies are being conducted to find alternative treatment methods, such as the use of nanoscale carriers, mesenchymal stem cells and gene therapy. In the case of gene therapy, a number of gene-targeted drugs are under investigation, including AADC, GDNF and HTT. Innovative treatments have the potential to bring therapeutic benefits to millions of people with neurodegenerative diseases.