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Gene Therapies Target Defective Genes/CRISPR Advances DNA Editing, Business and Industry Trends Analysis

A major goal of DNA research and the sequencing of the genetic profiles of patients is to attack, and attempt to alter, specific defective genes within the patient.  This practice is known as gene therapy, a strategy that targets defective genes (mutations) within a patient by introducing new copies of normal genes.  These new, normal genes may be introduced through the use of viruses or proteins that carry them into the patient’s body.

Internet Research Tip: Gene Therapies
A “gene therapy” is based on first identifying the fact that a patient has a specific gene mutation related to a specific disease, followed by the introduction of healthy genes into the patient’s body with the goal of altering or replacing the defective genes.  This is the Holy Grail of the biotechnology industry, as it offers the potential to cure otherwise incurable diseases.  It has also proven to be extremely difficult to carry out without significant side effects.  The American Society of Gene & Cell Therapy, www.asgct.org, publishes extremely useful data for those researching the promise and current status of gene therapies.  Their online tools include data on clinical trials, a glossary and a list of “breakthroughs.”  The group also publishes an extremely useful newsletter.


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