What is Gene Therapy?
Gene therapy is a technique used to correct defective genes genes that are responsible for disease development. Specifically, according to the American Society of Gene and Cell Therapy-
Gene therapy is defined as a set of strategies that modify the expressionof an individuals genes or that correct abnormal genes. Each strategyinvolves the administration of a specific DNA (or RNA).
Gene therapy is the manipulation of the expression of specific genes in a persons body, in hopes of treating a disease or disorder. Gene therapy is still considered experimental and only available via clinical trial. Although many successful trials have been documented (see Interesting Links below), gene therapy has a checkered history. In some gene therapy trials, there were cases of leukemia as an unintended side-effect, and even cases of death (see link on Jesse Gelsinger below).
Image courtesy of Wikimedia Commons
How Does Gene Therapy Work?
Although there are several strategies for gene therapy, the most commonly used method involves inserting a therapeutic gene into the genome to replace the abnormal or disease-causing gene. The gene that is inserted is delivered into a target cell via a vector. Usually, this vector is a virus, although non-viral vectors are in development. Viruses are a good choice for introducing genes into a cell because they typically operate by transferring their own genetic material while replicating themselves. Once target cells are infected with the viral vector, the vector releases its therapeutic gene which then incorporates into the cells DNA. The goal is that the cell will start using the new gene to make functional, healthy proteins.
There are three main strategies for using gene therapy to restore the target cells or target tissues to a normal, healthy state.
1. Insert the functional version of a gene in hopes of replacing the abnormal form. This is used to treat single-gene disorders like hemophilia A and B and cystic fibrosis.
2. Insert a gene that encodes for a therapeutic protein that treats a disease. This is used to treat acquired diseases likeinfection or ischemic heart disease.
3. Use gene transfer to down-regulate gene expression in hopes of decreasing the activity of a harmful gene.
Current Areas of Research
Although gene therapy is still experimental, many diseases have been targets for gene therapy in clinical trials. Some of these trials have produced promising results. Diseases that may be treated successfully in the future with gene therapy include (but are not limited to):* Anemias* Cardiovascular diseases* Cystic Fibrosis* Diabetes* Diseases of the bones and joints* Eye disease and Blindness* Gauschers Disease* Hemophilia* Huntingtons Disease* Lysosomal storage diseases* Muscular Dystrophy* Sickle cell disorder
The main challenges facing gene therapy are the identification of disease causing genes, the targeted delivery of the therapeutic gene specifically to the affected tissues, and the prevention of side effects (such as an immune response) in the patient.
Gene Therapy for Enhancement Purposes
If gene therapy becomes routine medical practice, then it is reasonable to believe that some will seek it out for enhancement purposes. For example, a gene therapy designed to help patients with Alzheimers disease may be appealing to a normal individual hoping to boost memory. One potential area of enhancement that has been discussed is gene doping in sports. Gene doping is defined by the World Anti-Doping Agency (WADA) as the non-therapeutic use of genes, genetic elements and/or cells that have the capacity to enhance athletic performance. The purpose of gene doping is toenhancea given gene rather thancorrecta faulty one. Potential targets of gene doping include:
* Erythropoietin (EPO) for increased production of red blood cells* Insulin-like Growth Factor-1 gene for increased muscle mass* Myostatin for increased muscle mass* Vascular Endothelial Growth Factor (VEGF) for an increase in blood flow
This form of doping would be hard to detect because the doping substances are produced directly in an individuals own cells after these genes with performance-enhancing effects have been expressed. Whether or not to use gene therapy in the future for enhancement purposes, and how to regulate it, will require a complex discussion of ethics in which there will likely be many differing opinions.
Interesting Links*The American Society of Gene and Cell Therapy* National Geographic articleon gene doping* Science Daily article onrecent gene therapy news* New York Times article on the death of Jesse Gelsinger* Scientific American article on treating blindness with gene therapy
CLICK HERE to read our case study involving ethical issues associated with gene therapy
Gene Therapy and Cell Therapy Defined. American Society of Gene and Cell Therapy, n.d. Web. 04 Nov. 2012.
Gene Therapy..Human Genome Project Information, n.d. We. 04 Nov. 2012.
Pawliuk R et al. Correction of sickle cell disease in transgenic mouse models by gene therapy. Science. 2001; 294:2368-2371.
Unal M, Unal DO. Gene doping in sports. Sports Medicine. 2004; 34:357-362.
Wells DJ. Gene doping: the hype and the reality. British Journal of Pharmacology. 2008 January; 154: 623-631.
Read the original post:
- Gene therapy - Wikipedia - January 3rd, 2019
- Gene Therapy Retrovirus Vectors Explained - January 3rd, 2019
- Gene Therapy Net - News, Conferences, Vectors, Literature ... - January 3rd, 2019
- Stem cell - January 1st, 2019
- Stem Cell Therapy - Get Back To Your Life | Stem Cell Centers - January 1st, 2019
- Stem Cell Treatment | Arizona | Stem Cell Rejuvenation Center - January 1st, 2019
- Stem cell - Wikipedia - January 1st, 2019
- Stem Cell Therapy for Anti-Aging and Sexual Performance ... - January 1st, 2019
- NSI Stem Cell | What Is Stem Cell Therapy? - January 1st, 2019
- Stem Cell Therapy for Osteoarthritis - StemGenex - January 1st, 2019
- Stem Cell Research & Therapy | Home page - January 1st, 2019
- Stem Cells in Milwaukee, WI | Wisconsin Stem Cell Therapy - January 1st, 2019
- Stem Cell Therapy | Advanced Regenerative Orthopedics - January 1st, 2019
- Stem Cell Therapy for Arthritis - January 1st, 2019
- National Stem Cell Centers | Stem Cell Therapy in New York ... - January 1st, 2019
- Is Stem Cell Therapy Covered by Medicare? - January 1st, 2019
- Home - Stem Cell Therapy in Orlando, Florida - January 1st, 2019
- Stem Cell Therapy & Treatment Center | TruStem Cell Therapy - January 1st, 2019
- Regenexx Stem Cell Procedures | 100% Non-Surgical Pain Treatment - January 1st, 2019
- Stem Cell Therapy for Knee Injuries and Arthritis - January 1st, 2019
- Stem Cell Therapy in Richmond Start Healing Chronic Pain ... - January 1st, 2019
- Stem Cell Basics I. | stemcells.nih.gov - January 1st, 2019
- 15 Stem Cell Therapy For Pain Questions, Answered - January 1st, 2019
- Startup Claims Its Underwear Stay Odor-Free Through Weeks of Wear - December 29th, 2018
- Microorganisms That Eat Seaweed Can Create Biodegradable Plastic - December 29th, 2018
- Apollo Astronaut: It Would Be “Stupid” to Send People to Mars - December 29th, 2018
- Elon Musk Tweets Image of SpaceX’s Stainless Steel Starship - December 29th, 2018
- New Multi-Sensory Mask Lets You Smell and Feel the Virtual World - December 29th, 2018
- New Fiber Could Be the Foundation for Futuristic Smart Garments - December 29th, 2018
- Cacti-Inspired Tech Could Keep You Hydrated After the Apocalypse - December 29th, 2018
- Your Christmas Tree Could Be Recycled Into Paint or Sweeteners - December 29th, 2018
- Space Travel Doesn’t Seem to Shorten Astronauts’ Lives, Says Study - December 29th, 2018
- Elon Musk Pledges Tesla Superchargers For All of Europe Next Year - December 29th, 2018
- China Is Building Its First Huge Battery Storage Facility - December 29th, 2018
- Australian Autonomous Train Is The “World’s Largest Robot” - December 29th, 2018
- Chinese Scientists Reportedly Lost Track of Gene-Edited Patients - December 29th, 2018
- Netflix’s Bandersnatch Teases the Future of Entertainment - December 29th, 2018
- Musk: Tesla’s Fully Autonomous Capabilities “About to Accelerate” - December 29th, 2018
- An App That Does Your Homework for You Is Now Worth $3 Billion - December 29th, 2018
- Virtual Reality Tumors Could Help Lead to New Cancer Treatments - December 29th, 2018
- Stem Cell Therapy for Arthritis and Injuries - Regenexx - December 27th, 2018
- Is Stem Cell Therapy for Arthritis Safe and Effective? - December 27th, 2018
- The Forever Fix: Gene Therapy and the Boy Who Saved It ... - December 23rd, 2018
- Gene therapy - Mayo Clinic - December 22nd, 2018
- Gene therapy | medicine | Britannica.com - December 22nd, 2018
- Cryptocurrency News: Looking Past the Bithumb Crypto Hack - December 16th, 2018
- Cryptocurrency News: This Week on Bitfinex, Tether, Coinbase, & More - December 16th, 2018
- Ripple Price Forecast: XRP vs SWIFT, SEC Updates, and More - December 16th, 2018
- Cryptocurrency Price Forecast: Trust Is Growing, But Prices Are Falling - December 16th, 2018
- Cryptocurrency News: Vitalik Buterin Doesn’t Care About Bitcoin ETFs - December 16th, 2018
- Cryptocurrency News: New Exchanges Could Boost Crypto Liquidity - December 16th, 2018
- Cryptocurrency News: Bitcoin ETF Rejection, AMD Microchip Sales, and Hedge Funds - December 16th, 2018
- Cryptocurrency News: What You Need to Know This Week - December 16th, 2018
- Cryptocurrency News: XRP Validators, Malta, and Practical Tokens - December 16th, 2018
- Cryptocurrency News: Bitcoin ETFs, Andreessen Horowitz, and Contradictions in Crypto - December 16th, 2018
- Gene therapy | medicine | Britannica.com - December 14th, 2018
- Dublin Aerospace - December 1st, 2018
- Home - Aerospace Industries Association - December 1st, 2018
- North American Aerospace Defense Command (NORAD) - December 1st, 2018
- AsMA | Aerospace Medical Association - December 1st, 2018
- Free gattaca Essays and Papers - 123helpme.com - November 27th, 2018
- Eugenics in the United States - Wikipedia - November 27th, 2018
- Eugenics - Wikipedia - November 27th, 2018
- Gene therapy - Mayo Clinic - November 25th, 2018
- Gene Therapy - Sickle Cell Anemia News - November 15th, 2018
- Gene therapy | medicine | Britannica.com - November 15th, 2018
- Gene therapy - Mayo Clinic - November 15th, 2018
- Gene therapy - Wikipedia - November 7th, 2018
- What is Gene Therapy? - Learn.Genetics - November 7th, 2018
- Gene therapy - Mayo Clinic - November 7th, 2018
- Gene Therapy Retrovirus Vectors Explained - November 6th, 2018
- Gene Therapy Net - News, Conferences, Vectors, Literature ... - November 6th, 2018
- A Stem Cell Transplant Let a Wheelchair-Bound Man Dance Again - October 29th, 2018
- AI Dreamed Up These Nightmare Fuel Halloween Masks - October 29th, 2018
- Robot Security Guards Will Constantly Nag Spectators at the Tokyo Olympics - October 29th, 2018
- People Would Rather a Self-Driving Car Kill a Criminal Than a Dog - October 29th, 2018
- Scientists Say New Material Could Hold up an Actual Space Elevator - October 29th, 2018
- This AI Lie Detector Flags Falsified Police Reports - October 29th, 2018
- These Bacteria Digest Food Waste Into Biodegradable Plastic - October 29th, 2018
- You Can Now Preorder a $150,000 Hoverbike - October 29th, 2018