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Straight, No Chaser: In the News: Gene Therapy

By Jeffrey Sterling, MD January 27, 2020


This is happening. If you can get past the politics and ethics of the debates on stem cell and gene therapy, you likely will be fascinated. I talk a lot about healthy eating, exercise and calorie control, because it represents a “back to basics” approach to health. These considerations are the ultimate “back to basics” approach to disease.
In the last several months, there have been news reports on isolated cases of reversal of blindness and improvement of muscular dystrophy and Huntington’s chorea as a result of experimental gene therapy. This is very exciting and worthy of a review of where the science is on this potential medical breakthrough. (My apologies in advance to my colleagues for an overly simplistic presentation.)
The unfortunate reality is that although scientists and clinicians have long believed that gene therapy would be the solution to many existing medical problems (simply because it makes sense theoretically), the ability to determine the application of gene therapy has continued to evade us. Even these success stories have not yet been followed by successful large clinical trials confirming the exact path to success. But, oh the possibilities!


Folks, we’re talking DNA, the building blocks of humanity, the genes that contain the code for our development. Gene therapy is a medical process that allows manipulation of one’s condition by inserting healthy genes to replace diseased genes. Alternatively, gene therapy involves targeting inactivate genes that are mutated or otherwise malfunctioning, then simply adding genes that target an existing disease. Various means to this end have been tried with varying degrees of success.
The possibilities of successful gene therapy in combating diseases involving damaged DNA (e.g., HIV, cancers, Alzheimer’s, etc.) are mind-blowing. Imagine the ability to avoid chemotherapy as a cure for cancer or surgery to remove a damaged organ. The tantalizing nature of this cat and mouse chaser also represents a virtual race against the clock as more and more people suffer daily.
Part of the recent excitement involves the achievement of “proof of concept,” which speaks to scientists accomplishing a study’s assertions of theoretical benefits through medical intervention or treatment. In this case, an entire genetic disease was eradicated in an adult animal. This is a critical step along the route of human testing and implementation of a treatment strategy.


Although gene therapy is a promising yet still theoretical treatment option for a large number of diseases, that’s not yet enough. Our societies still must render judgment on ethical questions and medical risks. Perhaps it’s good that these conversations aren’t drowning out the relative successes thus far.
Stay tuned to stay current on important medical research developments. You may be asked to support it. I welcome any questions or comments you may have.
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