This section introduces the background of gene therapy in Parkinson’s Disease, especially on brain cells targeting therapy. It provides the rationale for further proposal of brain cell targeted PD therapy based on Ultrasound-meditated microbubble delivered AAV vector or RNAi.
1.Parkinson’s Disease, a common CNS disorder with no adequate treatment in pharmacological approaches Parkinson's disease (PD) is a progressive, central nervous system (CNS) disorder, belonging to a group of conditions called motor system disorders. It is primarily characterized by the depletion of dopamine-producing brain cells and degeneration of dopamine, with an abnormal accumulation and aggregation of alpha-synuclein protein (Galpern 2006). PD belongs to the Parkinsonism, …show more content…
Parkinson's disease is the most common form of Parkinsonism. Most of the cases are idiopathic, which means that there is no specific known cause. A partial of cases can be attributed to known genetic factors (!!! INVALID CITATION !!! !!! INVALID CITATION !!!). Though the cause of PD is still unclear, limited lesion area and relatively low variety of Involved neurons make concisely targeting possible. Therefore, PD is considered as one of the most promising target for gene …show more content…
This kind of vectors can infect a wide range of cell type with high efficiency, and shows a good neuronal tropism in the mouse model (Paterna 2004). AAV has a great family of serotypes discovered from different animal species, and these serotypes are classified mainly by the capsid properties. Non-viral vectors are less appropriate for Parkinson’s Disease treatment Though the design of non-viral vector is more straight-forward, limitations of non-viral vectors are obvious: The duration of gene expression is short, while a long-term effect is expected in the chronic neurodegenerative disorder. Multiple uptake of vectors will be needed, such as repeated intracerebral injections, which will cause both a high risk of complication and a resistance of further vector interaction [Neuroprotection in a 6-hydroxydopamine-lesioned Parkinson model using lactoferrin-modified nanoparticles]. Besides, non-viral vectors needs modifications to pass the blood brain barrier(BBB), though the transduction efficiency is still relatively
1.Parkinson’s Disease, a common CNS disorder with no adequate treatment in pharmacological approaches Parkinson's disease (PD) is a progressive, central nervous system (CNS) disorder, belonging to a group of conditions called motor system disorders. It is primarily characterized by the depletion of dopamine-producing brain cells and degeneration of dopamine, with an abnormal accumulation and aggregation of alpha-synuclein protein (Galpern 2006). PD belongs to the Parkinsonism, …show more content…
Parkinson's disease is the most common form of Parkinsonism. Most of the cases are idiopathic, which means that there is no specific known cause. A partial of cases can be attributed to known genetic factors (!!! INVALID CITATION !!! !!! INVALID CITATION !!!). Though the cause of PD is still unclear, limited lesion area and relatively low variety of Involved neurons make concisely targeting possible. Therefore, PD is considered as one of the most promising target for gene …show more content…
This kind of vectors can infect a wide range of cell type with high efficiency, and shows a good neuronal tropism in the mouse model (Paterna 2004). AAV has a great family of serotypes discovered from different animal species, and these serotypes are classified mainly by the capsid properties. Non-viral vectors are less appropriate for Parkinson’s Disease treatment Though the design of non-viral vector is more straight-forward, limitations of non-viral vectors are obvious: The duration of gene expression is short, while a long-term effect is expected in the chronic neurodegenerative disorder. Multiple uptake of vectors will be needed, such as repeated intracerebral injections, which will cause both a high risk of complication and a resistance of further vector interaction [Neuroprotection in a 6-hydroxydopamine-lesioned Parkinson model using lactoferrin-modified nanoparticles]. Besides, non-viral vectors needs modifications to pass the blood brain barrier(BBB), though the transduction efficiency is still relatively