Grant Award Details
- To develop an allogeneic, PSC-derived glial-restricted progenitor cell therapy (GRNPCs) for treating Rett syndrome
Grant Application Details
- A treatment for Rett syndrome using glial-restricted neural progenitor cells
We developed a novel glial-restricted neural progenitor cells transplantation strategy as a treatment for Rett syndrome, reverting neuronal alterations caused by genetic mutations.
There are no disease-modifying therapies for Rett syndrome. Our therapeutic, if successful, will be a first-in-class treatment for this devastating neurological disorder and potentially others.
Major Proposed Activities
- Characterization of the candidate glial-restricted progenitor stem cells production.
- In vitro studies of the cell transplantation in Rett syndrome brain organoids to assess the ability of the cells to revert neuronal alterations at molecular, cellular and circuit levels.
- In vivo efficacy studies upon cell transplantation in the brains of a mice model for Rett syndrome to measure the cellular, physiological, behavioral and survival impact of the treatment.
- Prepare and organize the next steps using large animals to assess immunogenicity, cytotoxicity and off-target effects before moving into clinical trials.
Brain disorders are responsible for more years lost to disability than any other medical condition. Rett syndrome is one of these conditions, affecting Californians independently of race/ethnicity and socioeconomic status. Our therapeutic strategy can be applied to several other neurological conditions, including Parkinson's and Alzheimer's Disease, but also autism spectrum disorders, affecting 1 in every 54 births worldwide, expanding the benefits of the development of this approach.