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Parkinson’s disease (PD) is a neurodegenerative disorder that gives rise to motor symptoms such as tremor, bradykinesia, rigidity, and postural instability and non-motor symptoms such as anosmia, constipation, apathia, insomnia, and depression. The disease is caused by the progressive degeneration of dopaminergic neurons in the substantia nigra pars compacta, leading to a deficiency of dopamine release in the striatum. Parkinson’s disease presents several features that make it an ideal target for gene therapy. PD is a progressive, long-lasting neurodegenerative disease that is not adequately treated with pharmacological approaches. Furthermore, the cause of the major motor symptoms is well defined and associated with dysfunctions in brain nuclei that can be effectively targeted with viral vector technologies for gene transfer to brain cells. Therefore, part of the gene therapy strategies aim at increasing the efficacy and at reducing the side effects of the current pharmacological and surgical treatments.
Vectors derived from adeno-associated viruses (AAV) are the most frequent type of vectors used in clinical trials for central nervous system (CNS) diseases. AAV vector particles can efficiently infect a broad range of cells, including postmitotic cells, and display a good neuronal tropism. They enter the host cell through receptor-mediated endocytosis, and are translocated to the nucleus where their genome is converted into double-stranded DNA. In postmitotic cells, episomal AAV genomes provide long-term (>1 yr) transgene expression. The low rate of integration of recombinant AAV vectors is considered an asset in their safety profile, as it limits the occurrence of insertional mutagenesis.
As a leader in AAV technology, Creative Biogene is proud to offer AAV viral vectors that can be used critical research tools to study the underlying mechanisms of Parkinson’s disease. We offer Parkinson’s disease tools that knockdown wild-type and mutant forms of alpha-synuclein (αSyn), as well as an A53T αSyn viral vector that overexpresses the mutation associated with familial Parkinson’s disease. They present a very good safety profile, and provide efficient transduction and durable expression of neurons. Our AAV tools for Parkinson’s disease are available immediately and delivered to you ready for in vitro or in vivo research.
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