Gene Therapies Study

In recent years, gene therapy has emerged as an attractive tool for the treatment of Parkinson's disease (PD), providing an exciting prospect for the treatment of PD. As an innovative company in the field of neuroscience, Creative Biolabs has successfully developed advanced gene editions and delivery technologies, which provide a powerful platform for gene therapy.

Overview of Gene Therapy

Gene therapy is considered to be a rapidly evolving therapeutic approach that involves genetically modifying cells that are directly impaired in function and capable of relieving disease symptoms to cure disease. The technology is based on the use of vectors to carry DNA, RNA, or DNA/RNA editing enzymes into specific cells to regulate gene expression. These modifications can increase or decrease the expression of specific genes or genomes, and even restore the normal function of these gene products. For decades, researchers have been working to perfect gene therapies for various diseases. Fortunately, gene therapy has made great strides in certain diseases.

Fig.1 Strategies of in vivo gene therapy and ex vivo gene therapy. (Sawamoto, et al., 2018)

Tools of Gene Therapy

Currently, a variety of methods for gene delivery to target cells have been developed, including viral vectors and non-viral systems. Non-viral methods for gene transfer into the central nervous system (CNS) are rare and in vivo gene transfer relying on viral vectors is prevalent in CNS research. What's more, viral vectors, including adeno-associated virus (AAV) and lentivirus, are generally more efficient than non-viral vectors at delivering genes into cells of interest.

Gene Therapy for PD

PD is a complex neurodegenerative disease that can't adequately be treated by pharmacological approaches. In addition, PD is closely related to brain nuclear dysfunction, which can be efficiently targeted for gene transfer into brain cells by viral vector technology. For the above reasons, PD becomes an ideal target for gene therapy. Currently, there are several main strategies for gene therapy for PD: neuroprotection, restoration of dopamine synthesis, genetic neuromodulation, and resolution of disease-specific pathogenic variants. Furthermore, ongoing clinical gene therapy trials for PD mainly focus on enzymatically enhancing dopamine production and/or improving neuronal health and restoring dopaminergic signaling, resulting in lasting effects on neuronal function. There are many examples of using various gene therapy strategies to treat PD. For instance, introducing the glutamic acid decarboxylase (GAD) gene to a specific location in the brains of PD patients can increase the presence of gamma-aminobutyric acid (GABA) in brain regions that help reset abnormal circuits in the PD brain.

With our advanced technology platform and professional team, Creative Biolabs will be the best choice for your gene therapy development in terms of neurological diseases. If you have any questions about gene therapy research, please contact us in time for reasonable advice and professional help.

Reference

1. Sawamoto, K.; et al. Gene therapy for Mucopolysaccharidoses. Molecular genetics and metabolism. 2018, 123(2): 59-68.