A treatment for Rett syndrome using glial-restricted neural progenitor cells

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Grant Award Details

Grant Type:
Quest – Discovery Stage Research Projects
Grant Number:
DISC2-13515
Investigator(s):
Name:
Professor Alysson R Muotri
Institution:
University of California, San Diego
Type:
PI

Disease Focus:
Autism, Neurological Disorders
Human Stem Cell Use:
iPS Cell
Award Value:
$1,402,240
Status:
Active

Progress Reports

Reporting Period:
Year 2

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.

Publications