Gene therapy for diseases affecting the CNS (GT4CNS)

• Gene Therapy for rare diseases in the central nervous system; specific expression of the transgene; tropism analysis for gene therapy vectors; analysis of levels and time of expression of therapeutic genes. 

• Study of the molecular mechanisms involved in cognitive deficits in aging and Alzheimer's. 

• Gene transfer vectors; adenovirus; adeno-associated viruses; development of viral vectors; Generation and production of viral vectors for gene therapy.

1. Natural and chimeric chronokines for age-associated disorders 
2. Design, generation and production of AAV and adenovirus vectors 
3. AAV gene therapy strategies for rare diseases affecting the CNS:  

• Spastic Paraplegia type 52 (SPG52) 
• GNB1 Encephalopathy 
• ADSL Deficiency 

Our research group GT4CNS is focused on the study of the molecular mechanisms involved in genetic diseases that cause nervous system dysfunction, with the final goal of developing effective and safe gene therapy strategies for those diseases. WIth this main goal in mind, we apply state-of-the-art techniques such as 2D and 3D cultures of iPSC-derived neurons from patients, in vivo gene therapy strategies in animal models (including transgenic models), -omic analysis and novel viral vectors.