81. Gene Therapy - Genetics Home Reference A service of the U.S. National Library of Medicine®. Home Conditions Genes Chromosomes Handbook Glossary Resources http://ghr.nlm.nih.gov/handbook/therapy

About Site Map Contact Us A service of the Home Conditions Genes Chromosomes ... Resources Handbook Table of Contents Cells and DNA How Genes Work Mutations and Health ... Handbook Gene Therapy Experimental techniques, safety, ethics, and availability Please choose from the following list of questions for information about gene therapy, an experimental technique that uses genetic material to treat or prevent disease. Choose a topic or view all topics on one page What is gene therapy? How does gene therapy work? Is gene therapy safe? ... Is gene therapy available to treat my disorder? Next: What is gene therapy? Published: March 28, 2008 Lister Hill National Center for Biomedical Communications U.S. National Library of Medicine ... Accessibility Indicates a page outside Genetics Home Reference. Links to web sites outside the Federal Government do not constitute an endorsement. See Selection Criteria for Web Links PDF resources may require a PDF reader

82. Cystic Fibrosis Foundation - Drug Development Pipeline Drug Development Pipeline. The Cystic Fibrosis Foundation has built a dynamic pipeline for the development of more new potential CF therapies than ever http://www.cff.org/treatments/Pipeline/

Login Find A Chapter Great Strides Volunteer ... TREATMENTS > Drug Development Pipeline Drug Development Pipeline Below is a "snapshot" of those potential CF therapies that are currently in development as of January 2008. RESEARCH PRE- PHASE PHASE PHASE TO CLINICAL PATIENTS Gene Therapy Protein Assist/Repair Restore Salt Transport Mucus Treatment ... Nutritional Supplements Search for Clinical Trials Click button to the left to search for CF clinical trials currently recruiting. Clinical Trial Descriptions GENE THERAPY Because a faulty gene causes cystic fibrosis (CF), adding normal copies of the gene to cells should correct these cells and ultimately cure the disease. Compacted DNA back to top PROTEIN ASSIST/REPAIR This therapy is designed to correct the function of the defective CFTR protein made by the CF gene to allow chloride and sodium (salt) to move properly in cells lining the lungs and other organs. PTC 124 VX-770 Curcumin (Seer Pharmaceuticals) GSNO (Nitrox, LLC) S-Nitrosoglutathione (GSNO) levels are low in the lungs of CF patients. GSNO has been shown to promote trafficking of the Delta-F508 CFTR in some, but not all, tests on cultured cells. Nitrox LLC is developing inhaled GSNO for CF and is presently carrying out required preclinical safety studies, as well as formulation studies. VX-809 back to top RESTORE SALT TRANSPORT Denufosol SPI-8811 Moli Gilead GS9411 back to top MUCUS TREATMENT The following studies are being evaluated for their effectiveness in thinning and clearing the thick mucus from the airways.

83. Mary Ann Liebert, Inc. - Cookie Absent Your browser may not have a PDF reader available. Google recommends visiting our text version of this document. http://www.liebertonline.com/doi/pdf/10.1089/blr.2005.24.257

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