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03/11/2017

A gene therapy protects against cognitive and memory deficits associated with aging

chillon_884

03/11/2017

The regulation of the Klotho gene in the brain protects against learning deficits and memory associated with aging through gene therapy in mice.

Researchers at the Vall d'Hebron Research Institute (VHIR) and the Institute of Neurosciences of the Autonomous University of Barcelona (INc-UAB) have shown for the first time that the regulation of the Klotho gene in the brain protects against learning deficits and memory associated with aging through gene therapy in mice.UAB researchers had previously shown that Klotho regulates processes associated with aging, increasing life expectancy when over expressed and accelerating the development of learning and memory deficits when inhibited. Now, thanks to a joint research between INc-UAB and VHIR, they have shown in vivo for the first time that a single dose of this gene injected into the central nervous system protects it from the cognitive decline associated with the aging of old animals when they have been treated when they were young.The results, which are part of the doctoral thesis of Anna Massó, first author of the article, are the result of a research led by the researchers of the INc-UAB Dr. Miguel Chillón, ICREA researcher at the Department of Biochemistry and Molecular Biology at the UAB and the VHIR, and Dr. Lydia Giménez-Llort, of the Department of Psychiatry and Legal Medicine of the University, with the collaboration of the Dr. Assumpció Bosch, also from the Department of Biochemistry and Molecular Biology."The therapy was based on the increase in the levels of this protein in the brain through an adeno-associated vector (AAV). Taking into account that the study has been carried out in animals that have aged naturally, we believe that it could have a therapeutic potential to treat dementia and neurodegenerative disorders, such as Alzheimer's or multiple sclerosis, among others," says Miguel Chillón, head of the http://www.vhir.org/portal1/grup-presentacio.asp?s=recerca&contentid=187146&idrefer=187147 Genetic Therapy of the Nervous System Research Group at the VHIR.The researchers have patented the developed therapy and have licensed it to Kogenix Therapeutics. The company, owned by the UAB and based in the United States, has been driven by Dr. Miguel Chillón and Dr. Assumpció Bosch, along with entrepreneur Menachem Abraham and Dr. Carmela Abraham, Professor of Biochemistry and Pharmacology at the School of Medicine at the Boston University, a pioneer in the study of Klotho in the central nervous system for more than a decade.The objective of Kogenix is to obtain the initial capital necessary to advance preclinical studies already initiated in animal models of Alzheimer's that allow the development of a drug for gene therapy of neurodegenerative diseases, which is sufficient in small molecules that improve the expression of the gene and/or in the use of fragments of the protein itself."In studies conducted in basic research and clinical trials, AAV has proven to be safe and effective in the implementation of gene therapy for the central nervous system. In fact, the Food and Drug Administration has already approved the first gene therapy in the United States last August and is expected to approve it soon," says Assumpció Bosch.

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