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Human pluripotent stem cell differentiation, safety and preparation for therapeutic transplantation in Huntington’s disease

Objective

This project will tackle the huge complexity of taking stem cell therapies to clinical application for neurodegenerative disease by focusing on selective differentiation of a single neuronal phenotype (medium spiny striatal neuron: MSN) for a single well-defined disease (Huntington’s: HD). Our consortium contains expertise in all elements required to drive this technology to the point of clinical delivery, including expertise in stem cell differentiation and control of proliferation; in vitro genetic, molecular, cellular and functional characterisation; preclinical assessment in both rodents and primates models of HD; GMP knowledge, development and production; and clinical translation. Our clinical team includes world leaders in HD clinical trials, including fetal neural transplants and is well placed to design the translation process. We focus on human embryonic stem (hES) cells as our primary target for first-in-man proof-of-concept studies, as they are closest to clinical readiness. HD is the target disease as it provides both an excellent model relevant to a wide range of neurodegenerative conditions, and is a stringent test of the capacity of selectively differentiated stem cells to repair neural circuits. The starting point for the work is the existence within the consortium of three of the most advanced protocols to date for MSN differentiation, and a feature of our consortium is that the specificity of stem cell differentiation will be tested against primary fetal MSNs (current gold standard) at all stages of both in vitro and in vivo assessment. In order to maintain flexibility in an emerging ethical environment, we will develop induced pluripotent (hiPS) cells to the point of GMP validation as a second generation target to hESCs. This will build European infrastructure and capacity to deliver emergent stem cell therapies through the highest quality clinical trials into clinical practice in a broad range of human neurodegenerative diseases.
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Coordinator

CARDIFF UNIVERSITY

Address

Newport Road 30-36
Cf24 Ode Cardiff

United Kingdom

Activity type

Higher or Secondary Education Establishments

EU Contribution

€ 1 920 728

Participants (9)

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INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE

France

EU Contribution

€ 838 003

COMMISSARIAT A L ENERGIE ATOMIQUE ET AUX ENERGIES ALTERNATIVES

France

EU Contribution

€ 721 479

CONSORZIO PER LA RICERCA SUL TRAPIANTO DI ORGANI TESSUTI CELLULE E MEDICINA RIGENERATIVA

Italy

EU Contribution

€ 357 760

Roslin Cells Ltd.

United Kingdom

EU Contribution

€ 907 408

THE UNIVERSITY OF MANCHESTER

United Kingdom

EU Contribution

€ 334 176

WESTFAELISCHE WILHELMS-UNIVERSITAET MUENSTER

Germany

INSERM - TRANSFERT SA

France

EU Contribution

€ 475 056

GEORGE-HUNTINGTON-INSTITUT GMBH

Germany

EU Contribution

€ 361 873

ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS

France

EU Contribution

€ 83 517

Project information

Grant agreement ID: 602245

Status

Closed project

  • Start date

    1 October 2013

  • End date

    31 March 2018

Funded under:

FP7-HEALTH

  • Overall budget:

    € 7 820 104,75

  • EU contribution

    € 6 000 000

Coordinated by:

CARDIFF UNIVERSITY

United Kingdom

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