Parkinson’s disease (PD) is a chronic progressive neurological disorder characterized by bradykinesia, tremor, muscular rigidity, and postural instability. The world-wide prevalence is expected to rise further with increasing mean age. Theoretically, if all desired elements required for dopamine synthesis are functionally available, need for exogenous L-DOPA administration can be eliminated or markedly reduced. However, lack of effective long-term treatment has led to extensive gene therapy research focusing both on disease modifying as well as non-disease modifying aspects. Since genetic lesions are found in both familial as well as sporadic PD cases, the principle of introducing a normal gene to cure a disease can also be applied in PD. Success in effective gene delivery to the target brain regions and its tolerability owing to negligible immune response against the vector has further encouraged the work. The likelihood of gene therapy becoming future and true cure for PD is very high. This commentary describes status of non-disease modifying gene therapy in PD.
Shaikh, Muhammad Shariq; Ali, Syed Ahsan; and Kanwar, Dureshahwar
"Current Status of Non-Disease Modifying Gene Therapy in Parkinson’s Disease,"
Pakistan Journal of Neurological Sciences (PJNS): Vol. 16:
3, Article 7.
Available at: https://ecommons.aku.edu/pjns/vol16/iss3/7