Research for Rare - Research for rare diseases

CARPuD 2 - Network for Rare Pulmonary Diseases

Only limited or no therapeutic options are available for the genetic diseases α1-antitrypsin deficiency, cystic fibrosis, and surfactant deficiencies. These usually lethal pulmonary diseases severely impair the human respiratory tract. So far, lung transplantation is the only therapeutic option when terminal organ failure occurs. The generation of induced pluripotent stem cells (iPSCs) opens new avenues for the exploration of novel cellular therapies based on genetically corrected patient-specific stem cells.

iPS cells are re-programmed somatic cells, which, in contrast to adult or embryonic stem cells, can easily be grown in large quantities. Additionally, they can be differentiated into any cell type of the human body. Thus, iPSCs are a valuable tool for the therapy of rare diseases.

Aim of the CARPuD 2 consortium is to genetically correct the genetic defects, to differentiate the cells into the desired pulmonary cell derivative, and to finally re-transplant these cells, thereby replacing the affected cell type.

Focus of the project is the experimental development, optimisation, and evaluation of the therapeutic concepts. Additionally, a patients’ fibroblast bank will be established to enable the generation of a variety of patient specific stem cells. Mostly, these projects are basic research. In the near future, though, together with clinical partners, also pre-clinical and clinical phase-1 studies will be performed to allow for wide applications of iPSC-derived cells to treat rare pulmonary diseases also with the perspective to be transferred to general clinical practice.

Prof. Dr. rer. nat. Ulrich Martin
Leibniz Forschungslaboratorien für Biotechnologie und künstliche Organe (LEBAO)
Klinik für Herz-, Thorax-, Transplantations- und Gefäßchirurgie (HTTG)
Hannover Medical School (MHH)
30625 Hannover, Germany
Phone: +49 (511) 532-8820 (-8821)

PD Dr. med. Tobias Cantz
REBIRTH – Center for Regenerative Medicine
Hannover Medical School (MHH)
Carl-Neuberg-Straße 1
30625 Hannover, Germany
Phone: +49 (0)511 532-5251


Weiterführende Informationen

Artikel des Verbundes CARPuD in: Verbundforschung für seltene Erkrankungen. Gemeinsam zu Diagnose und Therapie, Februar 2014

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