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Clinical Translational Core Currently in Pilot Phase

Core Goals

• Provide direction and coordination for translational projects utlizing CHDD Cores, ITHS programs, and/or related UW resources;
• Establish and maintain an IDD Research Navigator Service for CHDD Research Affiliates to obtain facilitated access to key services and resources that will advance their translational research goals;
• Provide consultation on several key aspects of IDD-specific translational research including:
  • moving from gene identification to functional assays amenable to a compound screening approach
  • CNS specific approaches to understanding pharmacokinetics and toxicology
  • developing new animal models
  • identification of special issues regarding clinical research in IDD populations including access and ethics;
• Increase knowledge and understanding of the process and practice of translational research among CHDD Research Affiliates through educational seminars and research symposia;
• Promote the development of research collaborations among basic scientists, clinical researchers, and practicing clinicians working on common problems; and
• Educate the CHDD Research Affiliate community on how to organize their research programs towards advancing diagnosis, prevention, treatment, or amelioration of Intellectual and Developmental Disability (IDD).

Specific Aims

• Establish and maintain an IDD Research Navigator Service for CHDD Research Affiliates to obtain facilitated access to key services and resources that will advance their translational research goals.
• Provide CHDD Research Affiliate investigators with access to experienced consultants in specific areas where resource gaps have been identified.
• Educate the CHDD Research Affiliate community on how to organize their research programs towards advancing diagnosis, prevention, treatment, or amelioration of IDD syndromes.

Services Provided

IDD Research Navigator Service

Beth Hacker, Ph.D., bhacker@uw.edu

This service coordinates and integrates UW and regional resources to enhance translational research in IDD for CHDD Research Affiliates. The effective utilization of all of these resources requires more knowledge and experience than can be acquired by any individual investigator.

Consulting Service

Translational research projects typically require investigators to perform research using disciplines or technical approaches with which they do not have extensive experience. We provide four areas, listed below, that receive special attention through our Consulting Service to supplement other CTC services.

Functional Assays and Compound Screening Consultation

Edwin Rubel Ph.D., rubel@uw.edu

This service provides Research Affiliates with information on how to access CTC resources for the performance of tissue expression analysis of genes identified as related to IDD. Affiliates also receive consultation on the potential utility of cell or model organisms. Services previously provided by the Cellular Morphology Core will now be provided as part of a cost center operated by the Clinical Translational Core.

Pharmacology Consultation

 Nephi Stella, Ph.D., nstella@uw.edu

This service first consults on drug pre-formulation and helps identify potential vehicle compounds that will maximize both the desired pharmacokinetics and CNS penetration. This will be followed with information on how to best interact with the Pharmacokinetics Laboratory in the Department of Medicinal Chemistry to obtain information about how to assess tissue specific compound concentration following drug delivery.

Animal Model Development Consultation

Kathleen Millen, Ph.D.,kathleen.millen@seattlechildrens.org

This service provides information and consultation on vector design for gene targeting and transgenic approaches. Consultation will also provide information on how to effectively utilize internal and external resources to generate and characterize mouse models. Information on resources for models using alternate organisms (e.g., rats, zebrafish, or invertabrates), non-genetic approaches, and existing models at other institutions will also be provided.

Clinical Research Consultation

Forrest C. Bennett, M.D, fbennett@uw.edu

This service assists investigators with obtaining access by providing information for researchers to organized clinical populations, registries, and UCEDD EMR. Dr. Bennett also provides critical consults on special considerations for patient recruitment and community outreach in IDD populations, assistance with the design of consent documents for participation in research, and consultation on special ethical consideration for intervention trials on subjects with IDD.

Educational Program

The Educational Program consists of several types of activities: (1) symposia on translational research progress, (2) educational lectures on core services, (3) lectures on special topics in IDD clinical research and, (4) Collaborative Research Area (CRA) focused symposia. The most effective type of education is often the demonstration of research progress which is manifested in an annual research symposium.

To Use CTC Services

All components of the CTC are overseen by Dr. Garden. More information on utilizing components can be acquired by contacting the researchers listed above, or contacting Dr.Garden at gagarden@uw.edu, 206-616-9402.

University of Washington • Center on Human Development and Disability Box 357920 • Seattle WA 98195-7920 USA • 206-543-7701 •chdd@uw.edu

Copyright © 1996—2014 Center on Human Development and Disability. Updated: May 8, 2014