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Induced Pluripotent Stem Cells for the Development of Novel Drug Therapies for Inborn Errors of Metabolism (iPSC-IEM)

2019-09-25 06:13:27 | BioPortfolio

Summary

Human induced pluripotent stem cells (iPSCs), are reprogrammed from somatic cells that can self-renew indefinitely and produce different types of cells. They provide human model cell lines for orphan drug development. It is the goal of this study to define new cellular disease models for Inborn Erors of Metabolism, as enabling tools for both drug discovery and development.

Study Design

Conditions

Inborn Errors of Metabolism

Intervention

Skin biopsy, Withdraw of blood

Location

Centogene AG
Rostock
Germany
18055

Status

Recruiting

Source

Centogene AG Rostock

Results (where available)

View Results

Links

Published on BioPortfolio: 2019-09-25T06:13:27-0400

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Medical and Biotech [MESH] Definitions

Errors in the metabolism of LIPIDS resulting from inborn genetic MUTATIONS that are heritable.

Errors in metabolic processes resulting from inborn genetic mutations that are inherited or acquired in utero.

Errors in metabolic processing of STEROIDS resulting from inborn genetic mutations that are inherited or acquired in utero.

Dysfunctions in the metabolism of metals resulting from inborn genetic mutations that are inherited or acquired in utero.

Dysfunctions in the metabolism of PURINES or PYRIMIDINES resulting from inborn genetic mutations that are inherited or acquired in utero.

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