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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

TrkB agonist therapy for sensorimotor dysfunction in Rett syndrome

Principal Investigator

Principal Investigator

Katz, David

Description

Description

The aim of this study is to evaluate a novel therapeutic approach for the treatment of brain dysfunction in a mouse model of Rett syndrome (RTT), a severe autism spectrum disorder (ASD). In addition, it will define how a common variation in one gene, Brain-Derived Neurotrophic Factor (BDNF), may influence brain function in RTT, as well as the response to therapy. The basis of this approach is the use of newly developed compounds that mimic the activity of BDNF, a critical brain molecule which is in deficit in RTT. These compounds bind to and activate the BDNF receptor, TrkB. Recent studies by the research team demonstrated that RTT mice exhibit abnormalities in a measure of cognitive function that is also disrupted in other ASDs, and that normal function can be restored with one of these TrkB compounds. These studies aim both to shed further light on the role of BDNF availability in cognitive dysfunction in RTT and to determine how variant BDNF alleles may influence the efficacy of TrkB agonist treatment. Together, the research addresses the Autism Speaks priority areas: 1) identify novel treatments for ASDs; and 2) understand the molecular pathophysiology of ASDs to inform translational research. Given the shared features of RTT and other ASDs, it is hoped that these studies will yield insight into the potential role of TrkB agonists in the treatment not only of RTT patients but of those afflicted with other ASDs as well.

Funder

Funder

Autism Speaks

Fiscal Year Funding

Fiscal Year Funding

0

Current Award Period

Current Award Period

2011-2014

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

TrkB agonist therapy for sensorimotor dysfunction in Rett syndrome (External web link)

Institution

Institution

Case Western Reserve University

State/Country

State/Country

Ohio

Project Number

Project Number

7667

Federal or Private?

Federal or Private?

Private

Received ARRA Funding?

Received ARRA Funding?

No

History/Related Projects

History/Related Projects

TrkB agonist therapy for sensorimotor dysfunction in Rett syndrome | 147806 | 2012 | 7667
TrkB agonist therapy for sensorimotor dysfunction in Rett syndrome | 147806 | 2014 | 7667
TrkB agonist therapy for sensorimotor dysfunction in Rett syndrome | 5867 | 2015 | 7667
TrkB agonist therapy for sensorimotor dysfunction in Rett syndrome | 141976 | 2013 | 7667

 
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