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Gene therapy for diseases involving the central nervous system- TherCNS

Organisation profile

Organisation profile

The research group is focused on defining and applying gene therapy strategies for the treatment of diseases that affect the Nervous System such as neurodegenerative and neurometabolic diseases, as well as in the study of the molecular mechanisms involved in the development and progression of pathological processes. This main objective is defined in 3 main lines: 1.- Development of gene therapy strategies for rare diseases affecting the central nervous system. 2.- Study of the molecular mechanisms involved in cognitive deficits in aging and Alzheimer's. 3.- Development, generation, and production of gene therapy vectors.

Directed by:

Assumpció Bosch

Research lines

  • Research lines:
  • Gene therapy for rare diseases affecting the central nervous system (spastic paraplegia type 52 -SPG52-, GNB1 encephalopathy, megalencephalic leukoencephalopathy with subcortical cysts -MLC-, Wolfram syndrome, amyotrophic lateral sclerosis -ALS-, and Rett's syndrome) using AAV vectors.
  • Study of molecular mechanisms involved in cognitive deficits associated with aging and Alzheimer's disease.
  • Generation and production of viral vectors for gene therapy; Development of viral vectors.

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Collaborations and top research areas from the last five years

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