How does gene therapy work ? |
Hey everyone this is my blog for gene therapy, any thing that I read I will try and summarize for you on this blog but I will leave links to the place I find the relevant information so you can see it for your self.
Gene therapy is an emerging technique for medical treatment that uses genes to treat genetic disorders that are inherited or acquired like cystic fibrosis and AIDS.
Gene therapy is an emerging technique for medical treatment that uses genes to treat genetic disorders that are inherited or acquired like cystic fibrosis and AIDS.
Gene therapy works by inserting a healthy gene in replacement of defective or undesirable gene, by completing a missing gene so that it expresses the desired protein or to completely "knock out" the undesired gene (U.S. National Library of Medicine, 2017). To do this the genetic material is introduced into the body where it passes through the plasma membrane and into the nucleus (U.S. National Library of Medicine, 2017). This is done by inserting the gene into a carrier called a vector which is genetically engineered (GE) to carry the gene to the nucleus.
The U.S. National Library of Medicine explain the methods of delivery in the following methods. Viruses are often used as vectors because they naturally infect cells which makes them ideal vectors (U.S. National Library of Medicine, 2017). The viruses are GE so that they do not cause disease. Using different types of viruses introduces new genes in different ways (U.S. National Library of Medicine, 2017). Retroviruses integrate theirs own genetic information into the human chromosome, adenoviruses slightly differ by introducing their DNA into the cell but not into the chromosome (U.S. National Library of Medicine, 2017). Alternatively instead of using viruses naked DNA or DNA enclosed in a chemical compound can be used for delivery (U.S. National Library of Medicine, 2017).
The vectors can be given by injection, intravenously or cell samples can be taken from patients and the to the vectors are introduced to them in a lab, after this the cells containing the vector are reintroduced into the body (U.S. National Library of Medicine, 2017).
https://ghr.nlm.nih.gov/primer/therapy/procedures
Research into gene therapy started in the 1970's and human trial happened in 1989 where 5 patients were treated for advanced melanoma (Ibraheem. D, Elaissari. A, Fessi. H, 2014). Since then over 2,000 gene therapy trials have been performed with over half of these for cancer treatment as well as many other diseases like neurological and ocular ones (Ibraheem. D, Elaissari. A, Fessi. H, 2014). The pie chart bellows shows the areas the 2000+ trials cover including healthy volunteers.
http://www.sciencedirect.com.salford.idm.oclc.org/science/article/pii/S0378517313010351?np=y&npKey=ef2b8a39bbe45a084ceed026ba25ef3693f0bc2351c2164aa61c8719db7a5625
References
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Daya, S., & Berns, K. I. (2008). Gene Therapy Using Adeno-Associated Virus Vectors. Clinical Microbiology Reviews, 21(4), 583–593. http://doi.org/10.1128/CMR.00008-08 https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2570152/
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(How does gene therapy work ? 2017) genetics Home Reference, U.S. national Library of Medicine https://ghr.nlm.nih.gov/primer/therapy/procedures
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http://www.sciencedirect.com.salford.idm.oclc.org/science/article/pii/S0378517313010351?np=y&npKey=ef2b8a39bbe45a084ceed026ba25ef3693f0bc2351c2164aa61c8719db7a5625.
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