Gene Therapy Presentation


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Areas of Clinical Laboratory Diagnostics: GENE THERAPY:

Alexandra Rasiuk Janany Sivalingam Dmitry Uchitel Areas of Clinical Laboratory Diagnostics: GENE THERAPY 1 November 24 th , 2015


Background What is it? History Uses in Medicine What is it? Experimental technique to treat or cure diseases without drugs or surgery Could involve gene mutation by: Replacing mutated, disease-causing gene with a healthy copy “Knocking out” malfunctioning mutated gene Adding a new, disease-fighting gene 2 Knocking out: inactivating


Background What is it? History Uses in Medicine History Relatively new treatment option Conceptualized in 1972 After several decades of failures, first commercial therapy, Gendicine (China) was released in 2003. 3 Gendicine : Chinese recombinant adenovirus therapy used to treat cancerous tumors with p53 mutations


Background What is it? History  Uses in Medicine Uses in Medicine Any disease with a genetic component could theoretically be helped with gene therapy Ex. inherited conditions, cancer, some viral infections, cystic fibrosis (CF, pictured above) 4 CF: Cystic Fibrosis healthy lung airway CF lung airway


Methodology  How it Works Somatic vs. Germline Vectors How it Works 5 BM: bone marrow IV : intravenous May receive chemo to clear old cells Harvest stem cells from BM or main blood line Cells are transfected with altered DNA Cells returned to patient by IV transfusion


Methodology How it works  Somatic vs. Germline Vectors Somatic vs. Germline 6 SCGT: Somatic Cell Gene Therapy GGT: Germline Gene Therapy CF: cystic fibrosis SCGT Genes are introduced into somatic cells Only affects sick individual Treats immunodeficiency, CF, haemophilia in vivo or ex vivo Most popular GGT Genes are introduced into germ cells ( i.e. sperm and egg cells) Affects future offspring in vivo or ex vivo Currently prohibited on human patients


Methodology How it works Somatic vs. Germline  Vectors 7 Vectors DNA needs a vector to be transported into the nucleus of target cell Viruses have efficient mechanisms to insert themselves into host DNA, so viral vectors are a common method Retroviruses  will insert DNA into host chromosome Adenoviruses  sends DNA to nucleus without chromosomal interaction Non-viral vectors ( e.g. plasmids or virosomes ) can also be used.


Practicality Regulations Ethical considerations In the news Regulations In Canada, no specific regulations for SCGT research/trials GGT not permitted on human patients All gene therapy research is regulated by Health Canada, and follows FDA Food and Drug Regulations If funded by NIH, must register with their office of biotechnology Trial protocol will be reviewed by NIH Recombinant DNA Advisory Committee All others must seek FDA approval 8 SCGT: Somatic Cell Gene Therapy GGT: Germline Gene Therapy FDA : Food and Drug Administration NIH : National Institutes of Health


Practicality Regulations Ethical considerations In the news Ethical Considerations Dealing with the human genome raises many ethical issues: What are “good” traits and “bad” traits? Opens possibility for discrimination Cost  are the wealthy at an advantage? In GGT, where changes affect offspring: Could fetus be affected in unpredictable (or fatal) ways? Allows option for engineering “designer” children 9 GGT: Germline Gene Therapy


Practicality Regulations Ethical considerations  In the News In the News Researchers use GT to treat patient with Choroideremia by introducing the REP1 gene to improve rod and cone function (2014). http :// / pdfs /journals/ lancet PIIS0140 -6736(13)62117-0. pdf GT conducted in HIV patients in which CD4T cells were used as vectors to render CCR5 gene inactive. Decreased blood level of HIV DNA (2014). http :// /doi/ full / 10.1056 NEJMoa1300662 GT used to treat acute lymphoblastic leukemia (ALL) in a 1 year old child (2015). http :// /science/2015 / nov / 05/ baby-girl-is -first -in-the-world-to-be -treated -with-designer-immune- cells Researchers call for international moratorium on the use of GGT in clinical trials due to lack of sufficient scientific knowledge (2015). http :// /news/don-t-edit-the-human-germ-line-1.17111 10 GT: Gene Therapy GGT: Germline Gene Therapy


Conclusions 11 Gene therapy is an exciting method for disease treatment and prevention that continues to evolve. While it promises many solutions where current treatments fail, it isn’t without its ethical roadblocks.

Regulation Documents:   National Institute of Health:   FDA Act – Health Canada:,_c._870.pdf :

Regulation Documents:   National Institute of Health: http:// /sites/default/files/NIH_Guidelines_0.pdf   FDA Act – Health Canada: http://laws- /PDF/C.R.C.,_c._870.pdf References:   Hanna, Kathi . “Germ Line Transfer”. National Human Genome Research Institute . National Institute of Health. 2006. Web. 21 November 2015.   “Biologics, Radiopharmaceuticals and Genetic Therapies”. Health Canada. Health Canada. 2012 September 28. Web. 22 November 2015.    “Gene Therapy Handbook”. Genetics Home Reference. National Institute of Health. 18 November 2015. Web. 22 November 2015. Genetic Science Learning Center (2014, June 22) Gene Delivery: Tools Of The Trade .  Learn.Genetics . Retrieved November 24, 2015, from  Rabio , I. (2003). Gene Therapy: Ethical Issues. Theoretical Medicine, 24: 31- 58 Unger, Dave. The Canadian Bioethics Companion. Chapter 5: Genetic and Reproductive Control. Web. 2011 <http:// /the- canadian -bioethics-companion/ chapter-5-genetic-and-reproductive-control/>   Wilson, JM. Gendicine : The First Commercial Gene Therapy Product; Chinese Translation of Editorial. Human Gene Therapy , 2005. 16(9): 1014-1015. 12

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