Gene Therapy

Gene therapy as a treatment for autoimmune disease of the nervous system is currently in the preclinical stages of development with multiple issues to overcome, including the choice of vector and the delivery method.38'39 As a general therapeutic approach, gene therapy offers the potential to overcome half-life issues for some protein therapies as well as overcoming the requirement for frequent administration. In addition, gene therapy can provide focal administration of a therapeutic; this is a drawback for multifocal diseases such as MS, but can, theoretically, be addressed by employing ex vivo engineered mobile cells. Since expression is to be long-term for chronic disease, the vector and transgene must be nonimmunogenic.

Gene therapy also can deliver a steady dose, as opposed to many systemic therapies that are often inefficiently administer at a high dose in order to achieve a therapeutic dose in the target tissue - the inverted V character for dose versus time typical of many systemic therapies. However, a constant dose is a drawback for acute autoimmune disease or chronic disease with acute phases; this issue can, theoretically, be overcome by having the gene expression under the control of a promoter that is responsive to the dynamics of the disease or by using a pharmacologically regulated expression system.

Direct injection is required because most viral and nonviral vectors cannot cross endothelial cell barriers. In the CNS, this is a potential problem for in vivo therapies, since the vector is likely to be transported anterogradely and retrogradely by any axon passing through the injection site - leading to broad multifocal expression.

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