Grant Award Details

A treatment for Rett syndrome using glial-restricted neural progenitor cells
Grant Number: 
DISC2-13515
Project Objective: 
  • To develop an allogeneic, PSC-derived glial-restricted progenitor cell therapy (GRNPCs) for treating Rett syndrome
Investigator: 
Disease Focus: 
Autism
Neurological Disorders
Human Stem Cell Use: 
iPS Cell
Award Value: 
$1,402,240
Status: 
Pre-Active

Grant Application Details

Application Title: 
  • A treatment for Rett syndrome using glial-restricted neural progenitor cells
Public Abstract: 

Research Objective

We developed a novel glial-restricted neural progenitor cells transplantation strategy as a treatment for Rett syndrome, reverting neuronal alterations caused by genetic mutations.

Impact

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.
Statement of Benefit to California: 

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.