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Interagency Autism Coordinating Committee (IACC)
Autism Research Database
Office of Autism Research Coordination (OARC)
 
Project Element Element Description

Project Title

Project Title

Studying Rett and Fragile X syndrome in human ES cells using TALEN technology

Principal Investigator

Principal Investigator

Li, Yun

Description

Description

Using the newly-developed TALEN technology, Dr. Yi has generated novel stem cell models of Rett and Fragile X syndromes (FXS) in human cells. Neural and glial cell types will be subjected to extensive phenotypic characterization with respect to structure, electrophysiology, transcription, translation and intracellular signaling. Findings may provide critical insights into the pathogenic mechanism of Rett and FXS in human cells, and could serve as a platform for future therapeutic identification efforts that could lead to treatments.

Funder

Funder

Brain & Behavior Research Foundation

Fiscal Year Funding

Fiscal Year Funding

30000

Current Award Period

Current Award Period

2013-2015

Strategic Plan Question

Strategic Plan Question

Question 2: How Can I Understand What Is Happening?

Strategic Plan Objective

Strategic Plan Objective

Green dot: Objective has greater than or equal to the recommended funding. 2SD. Launch three studies that target improved understanding of the underlying biological pathways of genetic conditions related to autism (e.g. Fragile X, Rett syndrome, tuberous sclerosis complex) and how these conditions inform risk assessment and individualized intervention by 2012. IACC Recommended Budget: $9,000,000 over 5 years.

Project Link

Project Link

No link available.

Institution

Institution

Whitehead Institute for Biomedical Research

State/Country

State/Country

Massachusetts

Project Number

Project Number

Federal or Private?

Federal or Private?

Private

Received ARRA Funding?

Received ARRA Funding?

No

History/Related Projects

History/Related Projects

Studying Rett and Fragile X syndrome in human ES cells using TALEN technology | 0 | 2012 | N/A
Studying Rett and Fragile X syndrome in human ES cells using TALEN technology | 30000 | 2014 |

 
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