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Accelerating Therapeutic Development for Huntington's Disease

Research tools & reagents

Research Tools & Reagents

Easy access to validated biological reagents removes resource barriers for researchers currently working on Huntington’s disease (HD) and for others new to the field. CHDI recognizes that HD therapeutic development is dependent on the research scientists’ unobstructed access to quality-controlled research tools, and we are continually developing methods, tools, and other resources for all researchers working on HD. Our goal is to broaden the participation of the HD research community by providing ever sharper tools for understanding the disease and for developing effective therapies. Easier access should also help accelerate research and increase the participation of the HD research community.

HD Community BioRepository

Working together with the Coriell Institute for Medical Research, CHDI has established a secure, centralized biorepository called the HD Community BioRepository (HDCB) to store and distribute quality-controlled and reliable research reagents.

The BioRepository currently contains:

  • Huntingtin cDNAs: These include exon 1 and full-length constructs with various CAG repeat lengths in different vectors, that have been fully sequenced and verified.
  • Antibodies: These include CHDI-initiated productions directed at HTT or other therapeutic targets for HD and their corresponding antigenic peptides.
  • Cell lines: These include CHDI-initiated lines as well as lines from other HD researchers which have been expanded and checked for sterility (including mycoplasma).

Our goal is to make all biomaterials with detailed supporting information (including vector maps, sequences, and culture protocols) available to the HD research community. We continuously seek out and commission other biological reagents that may be useful for HD research in order to make them more widely available. We are grateful to all those in the HD research community who have helped build the HDCB and encourage anyone interested in making their HD research biological reagents available through this biorepository to contact us at

Frequently Asked Questions

How can I obtain reagents from the HDCB?

Visit the Coriell website at HDCB collection.  Researchers currently not supported by CHDI may request these resources from the HDCB by creating an online account. Reagents will be sent after completing a simple online MTA (see the terms of the MTA here). Recipients are asked to cover the nominal cost of the bioreagent as well as shipping and handling.

To place an order create an account and follow the instructions.

Please note that CHDI-supported collaborators are asked to contact the CHDI science director/program manager associated with the collaboration, to ascertain whether the requested reagent is already included within their agreement’s Foundation Provided Materials.

I have a reagent that I’d like to make available to the HD research community, how do I do that?

Please email and we will advise how to proceed.

I have a suggestion for a reagent that would be important to the HD research community, who do I contact to discuss?

We continuously seek out and commission biological reagents that may be useful to HD researchers. Please forward your suggestions to

HD Stem Cell Initiative

HD therapeutic development requires cellular models that can accurately recapitulate the cell types most affected in the disease. Embryonic stem (ES) cells are pluripotent and can be induced to differentiate into any cell type. CHDI is pursuing several approaches for the generation and characterization of ES cell lines that contain mutant huntingtin alleles.

The HD Stem Cell Initiative is an international collaboration of HD investigators, stem-cell researchers, and CROs that aims to generate mouse and human HD ES cells from various sources, including:

  • Available HD mouse models
  • Genetic manipulation of existing human and murine ES lines

ES cells will be characterized, checked for sterility, and banked. They will also undergo existing and novel neuronal differentiation protocols.

Commercial Reagents

Working with CHDI, a catalog of high quality HTT proteins and antibodies for HD research are available through Curia Global. Please contact Val Graziano or James Pace for additional information.

The following links list suppliers of additional key research reagents:

Huntingtin Protein Quantification Assays

The HD research community has developed several immuno-based assays to quantify huntingtin (HTT) protein in a variety of samples, including tissues and biofluids from preclinical and clinical studies.

A platform of assays is now available to detect and measure expanded mutant HTT, HTT in a polyQ-independent manner, soluble, aggregated HTT states, as well as rodent and nonhuman primate HTT proteins. Many of these assays are now available on a fee-for-service basis at various contract research organizations (CROs; see below).

Examples of immuno-based assay technologies used for HTT quantitation:

  • Time-resolved fluorescence resonance energy transfer (TR-FRET) is an ultra-high throughput, homogenous assay technology with picomolar sensitivity.
  • Meso Scale Discovery (MSD) is a high-throughput, electro-chemiluminescence assay detection technology with picomolar sensitivity.
  • Single Molecule Counting (SMC) is an ultra-sensitive detection technology employing magnetic particle assisted confocal laser detection system with femtomolar sensitivity suitable for HD CSF samples.

 

Frequently Asked Questions

What huntingtin protein quantitation assays are available and where?

Charles River

Evotec, SE

IRBM, S.p.A.

How do I access these assays?

HD researchers currently not collaborating with CHDI can request these resources from the following CROs:

Charles River

Evotec

IRBM

* Note that all CHDI-supported collaborators should contact the science director/program manager associated with their project for further information about these assays.

Research Tool Registration and Submission

To obtain a Foundation ID (CHDI#), collaborators must submit a CHDI Foundation Materials Registration/Submission Request and complete the questionnaire. Please be advised that the submission of a completed questionnaire is obligatory, and it will take a minimum of 10 business days to process. If you have any questions and/or concerns, please contact the Foundation Science Director associated with your collaboration.