|
medpundit |
||
 |
 |
|
Wednesday, April 30, 2003Researchers at University Hospitals and Case Western Reserve University began the clinical trial one year ago with 12 patients who received a corrected gene in a saline solution dripped into their nasal passages. Cystic fibrosis patients have a genetic defect that causes the buildup of thick mucus in the lungs and digestive system. Scientists using a gene-transfer system developed by Cleveland-based Copernicus Therapeutics Inc. found that the patients suffered no ill effects and that the therapy appeared to normalize the nasal cells. The experiment did not attempt to cure the 12 volunteers because the nasal passages were merely a test site. Doctors don't know yet whether the gene transfer will take hold in the lungs or how frequently therapy would be necessary as mature cells die off and new, defective cells take their place. But in eight of the 12 patients, the cells produced enough protein to improve the transport of salt and water, a critical balance that is upset in cystic fibrosis patients. The gene controls production of the protein responsible for maintaining the balance. The technique uses something called PLASmin™ therapeutics to roll the DNA up into small enough sizes to infiltrate the cell's nuclear envelope: The company licensed technology that compacts DNA - the structure that carries the genetic code - so the corrected gene would be small enough to enter the cell nucleus. The compacting technology is critical because viruses that are commonly used to introduce corrected genes into cells are fraught with problems for cystic fibrosis patients. Early gene therapy trials caused a devastating inflammatory response. Sounds promising. The next step is to deliver it to the lungs and see if it works there, too. posted by Sydney on 4/30/2003 08:48:00 AM 0 comments 0 Comments: |
|
