Why Cystic Fibrosis Gene Therapy is Still Years Away - Comment
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Gene therapy for cystic fibrosis carries a promise of a one off treatment that would cure patient lungs from mucus buildup. What's covered in this presentation slides: 1. Cystic Fibrosis Gene Therapy bio501.com/cystic-fibrosis-gene-therapy 2. Genetics of Cystic Fibrosis Chromosome 7 CFTR Cystic Fibrosis Transmembrane Conductance Regulator CFTR Mutation 3. Function of CFTR CFTR maintains salt-water balance in lung epithelium by pumping chloride ions across cell membranes Chloride ions attract water molecules that help clear up mucus When CFTR is absent or not functioning, there is a build up of mucus and bacterial growth in lungs 4. Inheritance of Cystic Fibrosis Carrier Carrier Child with 2 mutated copies of CFTR Not carrier CFTR mutation carrier rate is 1 in 25 among people of European descent When two carriers have children, each child has a 1 in 4 chance of getting two mutated copies of CFTR and developing cystic fibrosis 5. Goal of Gene Therapy To deliver a functional copy of CFTR gene to patient lungs by inhalation If sufficient number of lung epithelial cells start producing CFTR, mucus clearing mechanism should be restored 6. Vectors Used in Cystic Fibrosis Gene Therapy Viruses Liposomes 7. The Challenge Mucus build up prevents vector particles from reaching lung epithelial cells Since the discovery of CFTR gene there have been 27 clinical trials, but none of them produced the desired therapeutic effect 8. The Future of Gene Therapy for Cystic Fibrosis Most promising therapy, MRT5005 is in Phase 1 / 2 clinical trials If successful, it could reach the patients in 5 – 10 years The technology of gene therapy is rapidly developing New delivery vectors with improved efficiency are very likely to show up in the near future 9. To learn more visit BIO 501 website bio501.com/cystic-fibrosis-gene-therapy |
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| Posted by : peter88 | Post date : 2019-11-07 17:40 | ||
| Category : Health & Medicine | Views : 470 | ||
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