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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.

Field of science

  • /medical and health sciences/medical biotechnology/cells technologies/stem cells
  • /medical and health sciences/clinical medicine/transplantation
  • /social sciences/sociology/anthropology/physical anthropology
  • /natural sciences/biological sciences/zoology/mammalogy/primatology

Call for proposal

FP7-HEALTH-2013-INNOVATION-1
See other projects for this call

Funding Scheme

CP-FP - Small or medium-scale focused research project

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)

INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE
France
EU contribution
€ 838 003
Address
Rue De Tolbiac 101
75654 Paris
Activity type
Research Organisations
COMMISSARIAT A L ENERGIE ATOMIQUE ET AUX ENERGIES ALTERNATIVES
France
EU contribution
€ 721 479
Address
Rue Leblanc 25
75015 Paris 15
Activity type
Research Organisations
CONSORZIO PER LA RICERCA SUL TRAPIANTO DI ORGANI TESSUTI CELLULE E MEDICINA RIGENERATIVA
Italy
EU contribution
€ 357 760
Address
Passagio Gaudenzio Luigi 1
35131 Padova
Activity type
Research Organisations
Administrative Contact
Giovannie Guglielmi (Mr.)
Roslin Cells Ltd.
United Kingdom
EU contribution
€ 907 408
Address
C/o Roslin Foundation, Wallace Building, Roslin Biocentre
EH25 9PP Roslin, Midlothian
Activity type
Research Organisations
Administrative Contact
Kathryn Reilly (Mrs.)
Administrative Contact
Kathryn Reilly (Mrs.)
THE UNIVERSITY OF MANCHESTER
United Kingdom
EU contribution
€ 334 176
Address
Oxford Road
M13 9PL Manchester
Activity type
Higher or Secondary Education Establishments
Westfälische Wilhelms-Universität Münster

Participation ended

Germany
Address
Schlossplatz 2
48149 Muenster
Activity type
Higher or Secondary Education Establishments
INSERM TRANSFERT SA
France
EU contribution
€ 475 056
Address
Rue Watt 7
75013 Paris
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
GEORGE-HUNTINGTON-INSTITUT GMBH
Germany
EU contribution
€ 361 873
Address
Johann Krane Weg 27
48149 Munster
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
ASSISTANCE PUBLIQUE HOPITAUX DE PARIS
France
EU contribution
€ 83 517
Address
3 Avenue Victoria
75000 Paris
Activity type
Research Organisations