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RFI for Hungtington's Disease


The mission of NIH is to support biomedical discoveries that will improve health and save lives. To further the development of therapeutic target validation for Huntington's disease, the NINDS would like input on the following questions:


Best Practice Guidelines

Animal Models

If so, please describe the model including genetic background, length of time to onset of disease, and the behavior and disease pathology relevant to human Huntington's disease.
(For example, reporting of commercial source or reference to original paper describing model, catalog number, strain background, average life expectancy, range of CAG repeat length across generations, differences in disease phenotype between males and females)
(For example, use of enriched cage environment, maintenance of HD mouse models through breeding with specific background strains). If breeding recommendations exist for specific Huntington's disease models, please provide details.

Study Design

(Blinding to genotype is feasible at the beginning of a study prior to symptom onset).
(For example, determination of plasma and target tissue exposure, determination of maximum tolerated dose (MTD) for each genotyped studied, verifying compound activity at the target (e.g. pharmacodynamic data), etc)

Outcome measures

(For example, MTD, target tissue exposure, route of administration, formulation details for the compound, pharmacodynamic data (on target activity),timing of intervention, etc)

Standardized protocols

If so please provide protocol(s).
If so, please provide protocol(s), and any known variation in background strain response.
(applicable to Huntington's disease models demonstrating neuronal loss)? Is so please provide protocol.
If so please provide protocol.
If so please provide the protocol.
If so, please provide protocol.

General Information



Responses must be submitted electronically using this web form, and will be accepted through January 29, 2010. Replies to individual questions are optional, and the site will permit anonymous responses. The information provided will be analyzed and may appear in various reports.

Specific questions about this RFI should be directed to the contact listed below:

Margaret Sutherland, PhD
Program Director NINDS
Neurodegeneration Cluster
NSC Rm 2203
6001 Executive Blv.
Rockville, MD 20852
Phone (301) 496-5680
Fax (301) 480-1080
E-mail: sutherlandm@ninds.nih.gov

Last updated May 2, 2011