Potassium Channel Gene Regulation and Expression in the Treatment of Cystic Fibrosis
Background
- Cystic Fibrosis (CF) is the most common fatal disease in humans. At present, there are thousands of CF patients in the U.S. and despite current therapies, the median age of survival is only 26 years.
- The first manifestation is often cough, followed by progressive dyspnea, tenacious sputum formation and bacterial colonization which can be fatal.
- The protein product of the CF associated gene is called cystic fibrosis transmembrane regulator (CFTR).
- CFTR regulates the outward flow of anions from epithelial cells.
- Population studies indicate that the most common CF mutation, a deletion of the CFTR gene, is associated with approximately 70% of cases.
- This mutation results in the failure of epithelial cell chloride (Cl-) channels to respond to normal intracellular regulatory mechanisms.
- In airway cells, this leads to an imbalance in ion and fluid transport causing abnormal mucus secretion ultimately resulting in pulmonary infection and epithelial cell damage.
- Current therapeutic approaches have proven ineffective.
Description Of Current Technology
Dr. Jeffrey Whitsett of the Cincinnati Children's Research Foundation has demonstrated a relationship between the CFTR and an inwardly-rectifying potassium channel gene (Kir 4.2). Specifically, it has been discovered that the expressed peptide of the Kir 4.2 gene potentiates Cl- transport by providing K+ channels as an alternative pathway. Furthermore, these K+ channels can be activated and/or regulated by those agents typically known to regulate Cl- channel activity (ex: cAMP or IBMX). Accordingly, since Kir 4.2 activation potentiates Cl- transport, new pharmacological agents that modulates Kir 4.2 either transcriptionally or post-transcriptionally can be developed to selectively treat the described membrane transport abnormality of CF.
Objective
We are seeking a corporate partner to support further proof of concept research, preferably as part of an exclusive option agreement, and/or to license this technology for development of a novel therapeutic approach to the treatment of CF.
Contact
To review proprietary data and other information relative to this technology, please contact:
Joseph D. Fondacaro, PhD
Director, Office of Intellectual Property & Venture Development
Cincinnati Children's Research Foundation
Mail Location 7032
3333 Burnet Avenue
Cincinnati, Ohio 45229-3039Phone: 513-636-7695
Fax: 513-636-8453
Email: jdfonda@chmcc.org
| Related Study Information |
|---|
| You must have the free Adobe Acrobat" Reader software installed on your computer to read this file. You can download Adobe Acrobat" Reader at Adobe's web site by selecting the version appropriate for your type of computer. |
