Today we want to write a different tale, starting with: “Once upon a time, there was NOT … “
Until a few years ago neurodevelopmental disorders were mainly studied in animal models.
Animal models represented the only tool for studying neurological diseases and for approaching drug development. But now, a new opportunity is available: from just a few blood cells obtained from patients, it is now possible to study human neurons and brain development thanks to a process called reprogramming. This technological breakthrough is revolutionizing current knowledge and enabling what until a few years ago seemed science fiction:
NOW modeling the development of human neurons in a mini-organ (brain organoids), both healthy and diseased IS POSSIBLE.
This represents a real revolution because in the past, many therapeutics which showed successful results in preclinical animal-based studies, failed the successive clinical trials on humans due to reproducibility issues between humans and animals.
In fact, human organoid technology could better recapitulate human physiology compared to animal models.
This innovative technology is already improving research and reducing the number of laboratory animals used.
The application of brain organoids to study brain development and diseases has provided new insights into the pathophysiology of neurological disorders, including primary microcephaly, autism, Down’s syndrome Parkinson, and Alzheimer diseases.
More details about these recent studies can be found here:
https://www.nature.com/articles/nature12517
https://www.nature.com/articles/s41586-021-04358-6
https://pubmed.ncbi.nlm.nih.gov/31130512/
https://pubmed.ncbi.nlm.nih.gov/30799274/
brain organoids technology applied to research on trio
Mutations in the TRIO gene cause rare congenital forms of microcephaly/macrocephaly and intellectual disability, greatly impacting the lives of the affected children and their families.
there is still no cure for diseases linked to the TRIO gene.
In particular, to this day, the mechanisms underlying the pathoetiology of Neurodevelopmental disorders caused by TRIO gene mutation have not been fully understood, thus limiting both translational and pharmacological research.
However, we can do something to change this situation!
Carla Liaci, a Ph.D. student, and Prof. Giorgio Roberto Merlo's lab in Turin (Italy) want to take the opportunity of applying the technology of brain organoids to the TRIO research.
Researchers from the Department of Molecular Biotechnology at the University of Turin, led by Prof. Merlo, in collaboration with Prof. Giorgia Quadrato (University of Southern California, Los Angeles, USA), are working on human neurons carrying the TRIO mutation, and we would like to concretely support them.
The goal of this fundraising campaign is to finance the expenses for a specific activity within the Italian Ministerial PRIN Project "Neural Cytoskeleton and Rho GTPases in human models of Intellectual Disability and Microcephaly."
Specifically, the funds raised will be used to support the generation of these mini-brain organoids from cells with TRIO mutation, directly in the laboratory of Prof. Giorgia Quadrato at the Broad CIRM Center, Keck School of Medicine, University of Southern California (USC), USA.
Even a small donation can make a difference!
The funds raised will be managed, spent, and fully accounted for according to current laws and regulations by the Biomedical Research Foundation of the University of Turin, Turin, Italy:
http://www.forb.unito.it/
More information about the TRIO gene mutation is available at the following webpage:
Trio Gene Mutation — Team TRIO
Institutional page of Merlo’s Laboratory:
https://www.dbmss.unito.it/do/home.pl/View?doc=Gruppi_ricerca/Genetica_e_sviluppo.html
For details regarding the project "Neural Cytoskeleton and Rho GTPases in human models of Intellectual Disability and Microcephaly" you can get in touch with Prof. Merlo at the following email addresses: