Many diseases have a genetic cause, including more than 10,000 monogenic diseases caused by mutations in individual genes. CRISPR/Cas9 may enable us to repair the genetic defects that underlie these diseases.
CRISPR is an acronym for “clustered regularly interspaced short palindromic repeats,” which are unique DNA sequences found in some bacteria and other microorganisms. Scientists have long sought the ability to control and modify DNA—the code of life. A gene editing technology known as CRISPR-Cas9 offers the potential for substantial improvement over other gene editing technologies in that it is simple to use and inexpensive and has a relatively high degree of precision and efficiency. Many experts assert that CRISPR-Cas9 may offer the means to prevent, treat, or cure medical conditions or disease producing substantial savings in direct and indirect economic costs, in addition to reducing the toll from pain, debilitation, and death. Many in the scientific, engineering, and business communities believe that CRISPR-Cas9 may offer revolutionary advances in the investigation, prevention, and treatment of diseases; understanding of gene function; improving crop yields and developing new varieties; production of chemicals used in bio-fuels, adhesives, and fragrances; and control of invasive species. These sequences, along with the genes that are located next to them, known as CRISPR-associated or Cas genes, form an immune system that protects against viruses and other infectious DNA.
CRISPR is already widely used for scientific research, and in the not too distant future many of the plants and animals in our farms, gardens or homes may have been altered with CRISPR. In fact, some people already are eating CRISPRed food.
CRISPR technology also has the potential to transform medicine, enabling us to not only treat but also prevent many diseases. We may even decide to use it to change the genomes of our children. An attempt to do this in China has been condemned as premature and unethical, but some think it could benefit children in the future.
CRISPR is being used for all kinds of other purposes too, from fingerprinting cells and logging what happens inside them to directing evolution and creating gene drives.
idōs initiated a process to achieve the following objectives: (1) update the clinical and coordinated framework to aid in to clarify roles and responsibilities to regulate biotechnology products; (2) formulate a long-term strategy to ensure that the regulatory and ethical system can adequately assess any risks associated with future products of biotechnology while “increasing transparency and predictability and reducing unnecessary costs and burdens”; and (3) create an external, independent analysis of the future landscape of biotechnology products. idos, Inc. will aid the proper DNA sequence product to administer and proper dosing information.
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