CRISPR Applications

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CRISPR combined with the Cas9 enzyme is the most efficient method for genome engineering and research. Applications of the CRISPR technology include gene therapy, drug development, agriculture, animal modeling, biofuel and more.

Animal Modeling

The production of physiologically relevant animal models is crucial to the process of drug discovery; with increased correlation to the clinical setting candidate drug attrition is reduced, accelerating the process of drug discovery, cutting costs and limiting the number of animal models used throughout the process. CRISPR is a versatile tool for engineering a wide variety of precise genetic alterations including:

– Gene knockouts
– Deletions
– Point mutations
– Short insertions (e.g. loxP sites, epitope tags)

High specificity and low cell toxicity enable the production of sophisticated animal models that better mimic human disease as proteins can be studied at native levels rather than the overexpression we have relied on in the past.

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Gene Therapy

The buzz of CRISPR has centered on potential uses for gene therapy; the method is faster, cheaper and most importantly, more accurate than any DNA editing technique used previously. Perhaps the most important and exciting advances for CRISPR therapeutics are aimed at human genetic disorders; a field which has been notoriously difficult to tackle and one which could deliver prolonged, and better quality, human life. CRISPR has so far made advances in:

– Sickle cell disease
– Duchenne muscular dystrophy
– Cystic fibrosis

2016 also saw the first approvals for CRISPR to be tested in humans for the first time ever; the trial will focus on use of an immune therapy to improve the body’s ability to fend off cancers such as melanoma, myeloma and sarcoma.

Agriculture

With an ever-increasing population and shrinking natural resources, CRISPR offers a precise way to modify crops in hopes of making them yield more food, and resist drought and disease more effectively. Since its 2013 demonstration as a genome editing tool in Arabidopsis and tobacco, both widely used laboratory plants, CRISPR has been used in various crops including:

– Grains – wheat and maize
– Fruits – oranges and tomatoes
– Vegetables – potatoes and cabbage

The first CRISPR/Cas9 modified organism got a green light from the US government in 2016; mushrooms that are resistant to browning. This may seem simple, but keeping food fresher for longer could be the first step in reducing the $162 billion that North America spends on wasted food each year.

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Biofuel

CRISPR methods for producing more robust agriculture yields can also be applied to creating sustainable sources for biofuel; a process that will allow us to develop new precursors for specialty polymers, adhesives and fragrances. Current CRISPR-mediated advances in this field include:

– Modification of oil-producing yeast to increase conversion of sugars to lipids and hydrocarbons. Previously these molecules were produced from non-renewable petroleum-based sources; this method improves production rates without sacrificing the environment
– Modification of acetogenic bacteria to develop a model for commercial production of ethanol from synthesis gas. This method enables for ethanol production in any geographic region without competing for food or land resources
– Systematic knockout of multiple genes in cyanobacteria in order to increase biofuel and chemical productivities

Drug Development

By providing a more effective method to probe the function of specific genes, CRISPR-mediated genome editing provides a novel and highly efficient way to accelerate the drug discovery and validation process, whilst simultaneously reducing cost. Its impact on drug discovery is vast, including:

– Enabling gene and cell replacement therapies
– Identifying novel drug targets through functional genomic screens
– Facilitating understanding drug mechanisms of action
– Simplifying the production of disease models

CRISPR technology is now also paving the way to patient stratification; grouping patients based on the genetic make-up of their disease or medical condition has the potential to revolutionize therapeutics and improve survival rates in hard-to-treat populations.

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Materials

CRISPR is relatively new in materials science, but it’s clear that genome engineering can be applied to naturally occurring materials such as:

– Silk and spider-webs
– Shells and membranes of mollusks
– Sucker ring teeth of squid

Superior versions have been produced at huge expense in past, but utilizing such a cheap and effective method will allow for accelerated advances. Previous products have focused on the domesticated silkworm; researchers have created glow in the dark silk by adding fluorescent tags to the gene. They’ve even proven that the need to dye fabrics may soon be obsolete by generating silkworms with in-built silk pigment. Now that CRISPR/Cas9 has been successfully used to mediate efficient genome engineering in the domesticated silkworm, the possibility of increase silk production with reduced costs looks set to change the world of textiles.

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