Patient specific stem cell derived models for Alzheimer s disease and related ne...
Patient specific stem cell derived models for Alzheimer s disease and related neurodegenerative disorders
Future treatment of incurable neurodegenerative disorders, including Alzheimer’s disease (AD), frontotemporal dementia (FTD), spinocerebellar ataxia (SCA), and Huntington’s disease (HD), has to be tailored to individual patients o...
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31/12/2017
UCPH
3M€
Presupuesto del proyecto: 3M€
Líder del proyecto
KOBENHAVNS UNIVERSITET
No se ha especificado una descripción o un objeto social para esta compañía.
TRL
4-5
Fecha límite participación
Sin fecha límite de participación.
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Información proyecto STEMMAD
Líder del proyecto
KOBENHAVNS UNIVERSITET
No se ha especificado una descripción o un objeto social para esta compañía.
TRL
4-5
Presupuesto del proyecto
3M€
Fecha límite de participación
Sin fecha límite de participación.
Descripción del proyecto
Future treatment of incurable neurodegenerative disorders, including Alzheimer’s disease (AD), frontotemporal dementia (FTD), spinocerebellar ataxia (SCA), and Huntington’s disease (HD), has to be tailored to individual patients or cohorts of patients to obtain an optimal effect. The STEMMAD project is aimed to create a highly innovative international collaboration between 2 R&D intensive SME and 3 distinguished academic partners, including one ICPC Third Country partner. The program will enhance the competitiveness of the SME partners and will have a great impetus in an area of immense societal importance: Neurodegenerative diseases and their potential cure. It is our overall aim, by advanced molecular tools, to derive patient-specific in vitro neural cell models that will allow for such customized treatment. Skin biopsies and blood samples will be collected from genetically and clinically well-characterized patients. Fibroblasts and mononuclear blood cells will be isolated and reprogrammed into induced pluripotent stem cells (iPSCs), which, in turn, will be differentiated into neural progenitor cells (NPCs) and specific neurons. We expect that these neurons will express molecular characteristics of the patient’s disease phenotype and thereby be representative as patient-specific neural cell models. The models will be characterized for functional disease parameters, used for studies of molecular pathogenesis. The iPSC reprogramming and neural differentiation will be controlled by advanced molecular technologies including manipulation of microRNA (miRNA) expression as well as the use of plasmid minicircles with reprogramming factor genes, thus leaving the genome free of transgenes. To ensure the success of the project, direct transdifferentiation of fibroblasts into neurons, without an intermediate stem cell stage, will also be attempted as an alternative strategy for generating patient-specific neural cell models.