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There is currently no effective treatment for Huntington’s disease, nor are there any appropriate experimental models to study possible drugs. The Cure of Huntington's Disease Initiative (CHDI) is leading an international effort to change this. This private American foundation has become interested in the stem-cell work carried out by the stem cell and regenerative medicine group, led by Dr. Josep M. Canals, and the pathological processes cell biology group at the Institute for Biomedical Investigation August Pi i Sunyer (IDIBAPS) and the Department of Cell Biology, Immunology and Neurosciences of the University of Barcelona (UB) Faculty of Medicine.

These researchers, which drive the UB Cell Therapy Program (TCUB), have discovered new differentiation factors that may contribute to turning stem cells into neurons. Now, thanks to funding from the CHDI, these discoveries will be applied to the production of neurological tissue to test new drugs for this disease in the laboratory. The project will be carried out jointly by the laboratories of Dr. Elena Cattaneo of the University of Milan and those of Dr. Nick Allen and Dr. Paul Kempt of the University of Cardiff.

New IDIBAPS facilities

This funding has allowed the institute to hire two postdoctoral researchers, one technician and one student, who have already started working. After summer, some of these experiments are expected to be moved to the UB Faculty of Medicine’s new clinical grade cell production facility. These new facilities are being built thanks to a donation from the Cellex Foundation.

"Thanks to the new personnel, new facilities and new services hired by the CHDI, we will work on producing striatum neurons from stem cells,” explained Dr. Josep M. Canals, who participated today in the workshop organized by Biocat and the TCUB entitled Challenges and Risks of Research in Advanced Therapies at the CEK in Barcelona.

The process of differentiation into neurons lasts approximately three months, and establishing a production protocol is one of the main objectives of this project. Initially, the team will work to obtain healthy neurons. Later it will be possible to produce neurons affected by Huntington’s disease, allowing for experiments to be carried out to discover new differentiation factors involved in the process.

These cells will be used to replace the current animal models used in the study of drugs, thus obtaining an experimental model much closer to human Huntington’s disease. These cells may come from both pre-implantation diagnosis embryos discarded during in vitro fertilization, or from induced pluripotent fibroblasts similar to embryonic cells.

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