Uploaded April 2019 | Updated September 2026, 2 weeks ago
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This week we talk about gene therapy as a possible cure for cystic fibrosis. Cystic fibrosis or CF is a common genetic disorder which currently cannot be cured. The disease is caused by mutations in CFTR, a transporter protein, which is crucial for the transport of chloride ions across cell membranes. Especially the lungs and digestive tracts are severly damaged over time in cystic fibrosis patients.
Current treatments for cystic fibrosis involve nutritional supplements and medications such as antibiotics, steroids or mucolytics. However, these treatments do not cure the disease.
In the last years, gene therapy has emerged as a very promising treatment for genetic diseases, such as cystic fibrosis. Gene therapy involves the delivery of nucleic acids, such as DNA or RNA into a patient's cell. In order to transport nucleic acids, vehicles (such as viruses or liposomes) are used. Since cystic fibrosis is caused by mutations in CFTR, we can potentially introduce intact versions of the CFTR gene into the lung tissue of CF patients. First gene therapy studies and clinical trials show beneficial effects, however, the delivery of CFTR needs to become more efficient in order to fully cure the disease. All in all, the following years will be crucial for the establishment of gene therapy as a cure for genetic diseases.
For your further research:
Alton, E. W., Armstrong, D. K., Ashby, D., Bayfield, K. J., Bilton, D., Bloomfield, E. V., ... & Carvelli, P. (2015). Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial. The Lancet Respiratory Medicine, 3(9), 684-691.
Cutting, G. R. (2015). Cystic fibrosis genetics: from molecular understanding to clinical application. Nature Reviews Genetics, 16(1), 45.
Cooney, A., McCray, P., & Sinn, P. (2018). Cystic fibrosis gene therapy: Looking back, looking forward. Genes, 9(11), 538.
Robinson, E., MacDonald, K. D., Slaughter, K., McKinney, M., Patel, S., Sun, C., & Sahay, G. (2018). Lipid nanoparticle-delivered chemically modified mRNA restores chloride secretion in cystic fibrosis. Molecular Therapy, 26(8), 2034-2046.
Music:
Intro:
Light Sting by Kevin MacLeod is licensed under a Creative Commons Attribution licence (https://creativecommons.org/licenses/...)
Source: http://incompetech.com/music/royalty-...
Artist: incompetech.com
Transitions:
Light Thought var 3 by Kevin MacLeod is licensed under a Creative Commons Attribution licence (https://creativecommons.org/licenses/...)
Artist: incompetech.com
About Clemens Steinek:
CLEMENS STEINEK is a postgraduate student/youtuber (LifeLabLearner) who is currently conducting stem cell research in Germany.
Subscribe for more awesome biomedical research: bit.ly/2SRMqhC
This week we talk about gene therapy as a possible cure for cystic fibrosis. Cystic fibrosis or CF is a common genetic disorder which currently cannot be cured. The disease is caused by mutations in CFTR, a transporter protein, which is crucial for the transport of chloride ions across cell membranes. Especially the lungs and digestive tracts are severly damaged over time in cystic fibrosis patients.
Current treatments for cystic fibrosis involve nutritional supplements and medications such as antibiotics, steroids or mucolytics. However, these treatments do not cure the disease.
In the last years, gene therapy has emerged as a very promising treatment for genetic diseases, such as cystic fibrosis. Gene therapy involves the delivery of nucleic acids, such as DNA or RNA into a patient's cell. In order to transport nucleic acids, vehicles (such as viruses or liposomes) are used. Since cystic fibrosis is caused by mutations in CFTR, we can potentially introduce intact versions of the CFTR gene into the lung tissue of CF patients. First gene therapy studies and clinical trials show beneficial effects, however, the delivery of CFTR needs to become more efficient in order to fully cure the disease. All in all, the following years will be crucial for the establishment of gene therapy as a cure for genetic diseases.
For your further research:
Alton, E. W., Armstrong, D. K., Ashby, D., Bayfield, K. J., Bilton, D., Bloomfield, E. V., ... & Carvelli, P. (2015). Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial. The Lancet Respiratory Medicine, 3(9), 684-691.
Cutting, G. R. (2015). Cystic fibrosis genetics: from molecular understanding to clinical application. Nature Reviews Genetics, 16(1), 45.
Cooney, A., McCray, P., & Sinn, P. (2018). Cystic fibrosis gene therapy: Looking back, looking forward. Genes, 9(11), 538.
Robinson, E., MacDonald, K. D., Slaughter, K., McKinney, M., Patel, S., Sun, C., & Sahay, G. (2018). Lipid nanoparticle-delivered chemically modified mRNA restores chloride secretion in cystic fibrosis. Molecular Therapy, 26(8), 2034-2046.
Music:
Intro:
Light Sting by Kevin MacLeod is licensed under a Creative Commons Attribution licence (https://creativecommons.org/licenses/...)
Source: http://incompetech.com/music/royalty-...
Artist: incompetech.com
Transitions:
Light Thought var 3 by Kevin MacLeod is licensed under a Creative Commons Attribution licence (https://creativecommons.org/licenses/...)
Artist: incompetech.com
About Clemens Steinek:
CLEMENS STEINEK is a postgraduate student/youtuber (LifeLabLearner) who is currently conducting stem cell research in Germany.
