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CRISPR/Cas9 technology makes possible genome editing – the precise and targeted modification of the genetic material of cells. Genome editing works by using an enzyme to make a cut at a particular sequence in the genome, followed by the deletion, repair or insertion of genetic material at the cut site, facilitated by the cell’s natural DNA repair mechanisms.

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Given its permanent effects on the target DNA, genome editing could potentially cure a disease with a single treatment course, instead of the multi-treatment or chronic dosing regimens often seen with traditional therapies, which typically have transient effects and may require life-long treatment.

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Given its permanent effects on the target DNA, genome editing could potentially cure a disease with a single treatment course, instead of the multi-treatment or chronic dosing regimens often seen with traditional therapies, which typically have transient effects and may require life-long treatment.

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CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeat.  These are DNA sequences found in the genomes of bacteria and other microorganisms.  CRISPR works through an immune mechanism by protecting the bacteria from viral invasion.  CRISPR thwarts the viral genome from replicating by destroying its genome. Cas9 stands for Capsase 9, which is an initiator of capsase enzyme in the apoptotic pathway.   Cas9 was identified in and purified from Streptococcus pyogenes.

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"CRISPR-Cas9 gives scientists the power to remove or add genetic material at will… This is a true breakthrough, the implications of which we are just beginning to imagine.” Mary-Claire King, a geneticist and the discoverer of the BRCA1 cancer gene

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Uploaded by Intellia Therapeutics on 2015-08-20.

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via Lipid Nanoparticle(LPN)

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via electroporation (an electrical charge-based technique for delivering molecules into cells.

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1) Knockout: stop genes that make harmful proteins or disease causing viruses (ex- neurodegenerative disease, cancer, and HIV)
2) Repair: edits that repair disease associated gene mutations (ex- Huntington's and muscular dystrophy)
3) Insertion: edits that correct disease associated genes (ex- sickle cell anemia, diabetes, and heart failure)

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The Centers for Disease Control and Prevention reports that genomics play a role in nine of the ten leading causes of death in the United States, most notably chronic conditions such as cancer, diabetes, and heart disease.

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Likely to create disruptive mutation and resistant alleles that did not previously exist (Reed, 2017).

Bacterial DNA could create an immune response ("CRISPR delivers proteins," 2017).

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This is an exciting technology which could end many chronic conditions.  However, safety must be ensured before making this technology widely available.  Additionally, we need to ensure cost-effectiveness so that the technology is available to all in need and not just to those who can afford treatment using genetic engineering.

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Genetic engineering requires precision by using computer-assisted gene sequencing and nanoparticle delivery systems.  Additionally, identification of specific genetic mutations is possible through the use of global genetic databases.