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Cystic Fibrosis and Stem Cells

What is Cystic Fibrosis?

Although technically a rare disease, cystic fibrosis is ranked as one of the most widespread life-shortening genetic diseases. Cystic Fibrosis (CF) is the most common life threatening, recessive genetic condition affecting the Western World and is most prevalent in Mediterranean countries and least prevalent in Asian countries.

Cystic Fibrosis is a genetic disease that affects a number of organs in the body (especially the lungs and pancreas) by clogging them with thick, sticky mucus. Other Symptoms can include poor weight gain, troublesome coughs, repeated chest infections, salty sweat and abnormal stools.

Repeated infections and blockages can cause irreversible lung damage and death. Mucus can also cause problems in the pancreas preventing the release of enzymes needed for the digestion of food. This means that people with CF can have problems with nutrition.

CF is an inherited condition. For a child to be born with CF both parents must be genetic carriers for CF. They do not have CF themselves.Many male CF sufferers are infertile due to the male reproductive organs failing to develop properly prior to birth.
Thanks to Cystic Fibrosis in Australia and Wikipedia for the above information

Articles

Stem cells and cystic fibrosis

M. Conese, J. Rejman Journal of Cystic Fibrosis, Volume 5, Issue 3, Pages 141-143

...This review presents an overview of recent attempts to identify lung- or bone marrow-derived populations of stem cells or progenitor cells and to apply such cells, heterologous or gene-corrected autologous, to colonize the airways while differentiating into functional respiratory columnar epithelial cells. The most successful approaches thus far appear to be obtained with bone marrow-derived cells such as mesenchymal stem cells, although the transdifferentiation rate thus far has been limited to below the 1% level. As an alternative the proven multipotent nature of bronchioalveolar stem cells isolated from lung tissue may provide another promising approach for successful stem cell therapy.

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The Potential for Stem Cell Therapy in Cystic FibrosisDownload PDF here

Helen Spencer ChB MRCP Adam Jaffe MRCP FRCPCH
JOURNAL O F THE ROYAL SOCIETY OF MEDICINE Volume 97 Supplement N o. 442004

... Numerous studies have now demonstrated the ability of hematopoietic stem cells and mesenchymal stromal cells to home to the lung and differentiate into epithelial cells of both the conducting airways and the alveolar region. However, engraftment of bone marrow-derived stem cells into the airways is a very inefficient process. Detailed knowledge of the cellular and molecular determinants governing homing to the lung and transformation of marrow cells into lung epithelial cells would benefit this process. Despite a very low level of engraftment of donor cells into the nose and gut, significant CFTR mRNA expression and a measurable level of correction of the electrophysiological defect were observed after transplantation of wild-type marrow cells into CF mice. It is uncertain whether this effect is due to the presence of CFTR-expressing epithelial cells derived from donor cells or to the immunomodulatory role of transplanted cells. Finally, initial studies on the usefulness of umbilical cord blood and embryonic stem cells in the generation of airway epithelial cells will be discussed in this review.

Stem Cell Therapy for Cystic Fibrosis: Current Status and Future Prospects

Donatella Piro; Joanna Rejman; Massimo Conese Published: 07/24/2008 www.medscape.com

Limited Restoration of Cystic Fibrosis Lung Epithelium In Vivo with Adult Bone Marrow–derived Cells

Roberto Loi, Travis Beckett, Kaarin K. Goncz, Benjamin T. Suratt, and Daniel J. Weiss Pulmonary and Critical Care, University of Vermont College of Medicine, Burlington, Vermont

Rationale: Recent literature suggests that adult bone marrow– derived cells can localise to lung and acquire immunophenotypic characteristics of lung epithelial cells. We speculated this might be a potential therapeutic approach for correcting defective lung epithelium in cystic fibrosis.

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