Rewriting the Blueprint
Gene editing for improved agriculture, healthcare, and beyond.
Healthcare
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"At CRISPR Therapeutics, we are focused on developing transformative gene-based medicines for serious human diseases" -CRISPR Therapeutics
CRISPR Therapeutics has been working on a pipeline full of potential solutions for cancer, hemoglobinopathies (blood disorders), diabetes, and potentially dozens of other previously debilitating diseases. CRISPR Therapeutics’ headquarters are located in Zug, Switzerland, with additional operations in Cambridge, Massachusetts, and San Francisco, California.
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CRISPR Therapeutics is beginning clinical trials for β-thalassemia and sickle cell disease--both of which are genetic mutations that cause an abnormal form of hemoglobin to be produced. Hemoglobin is a protein found in the blood that acts to transport oxygen throughout the body, and of course, with insufficient oxygen, a patient has very low chances of living a full, healthy life. These conditions are life-threatening for patients as lifelong monitorization and transfusions are required to sustain life. As a solution, CRISPR Therapeutics has designed the CTX001 program, which aims to isolate blood stem cells in patients for modification to produce HbF (fetal hemoglobin) which can reduce symptoms--fetal versions of this protein are much more viable than their adult counterparts and can multiply quickly to replace the problematic hemoglobin.
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One disease still in the research process intends to treat cystic fibrosis by correcting the mutated cystic fibrosis transmembrane conductance regulator (CFTR) gene via in-vivo methods. Other CRISPR disease treatments still being researched include Duchenne muscular dystrophy and glycogen storage disease (type la)
Editas Medicine, based out of Cambridge, Massachusetts, is another therapeutic company utilizing CRISPR for treatments--specifically for ocular and blood diseases, as well as cancer. They work closely with two CRISPR nucleases: Cas9 and Cas12a.
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Edit-301 is their program similar to CRISPR Therapeutics’ CTX001 that aims to treat sickle cell disease and β-thalassemia by manipulating genes to produce more fetal hemoglobin.
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They are currently in early-stage clinical trials for Edit-101, their treatment program for Leber congenital amaurosis 10 (LCA10) which is a disease that causes impaired vision by the degeneration of photoreceptors in the eyes. This CRISPR program holds promise for correcting the CEP290 mutation to normalize the photoreceptor protein formation and function. Additionally, the fact that eyes are immunoprivileged works to researchers’ advantage in that the chances of immune rejection of CRISPR are reduced.
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Other ocular diseases they are trying to cure include Usher Syndrome--which causes progressive vision and hearing loss--and autosomal dominant retinitis pigmentosa 4--a disease that causes loss of night and peripheral vision in children.
"Our mission is to translate the power and potential of genome editing into a broad class of CRISPR-based medicines that transform [the] lives of people living with serious diseases." - Editas Medicine
Economic Impact
Due to the novelty and complexity of these treatments, gene therapies come at en extremely hefty cost to patients. Over the course of these treatments, patients can expect to pay anywhere from around $375,000-$875,000, which is significantly higher than traditional treatments. Extensive regulatory procedures also contribute to these sky-high medical bills since, in order for a therapy to be offered, it must undergo approval by the FDA, the Office of Biotechnology Activities and the Recombinant DNA Advisory Committee--coming in at around $5 million for each therapy. Additionally, since gene therapies are extremely specific to the individual, clinical trials are costly on manufacturers: participants in clinical trials are owed approximately $1 million each.
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“Price is the Achilles' heel of precision medicine," Professor Arthur Caplan, head of the medical ethics division at NYU School of Medicine in New York City, tells Vantage. “Price is just as important as the science if you’re going to see the genomic revolution fulfill its promise.”
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Healthcare for the modern world.
Animals
The University of Guelph in Ontario, Canada developed a genetically modified pig known as the Enviropig™. They intended for the GM piglets to be an environmental solution for large-scale, factory farms to lessen the amount of harmful phosphorus run-off. Phosphorus is produced as a by-product of pig manure, and while it is utilized by plants, excess amounts of this molecule can trickle and run off into and toxify nearby water supplies. Scientists engineered this pig by introducing the phytase enzyme (created from E. coli phytase genes and mice DNA promoters) into their system to be produced in salivary glands. Phytase works to more efficiently digest phytate, which is found in animal feed. While the project was originally funded by Ontario Pork, the company has since withdrawn its funds in fear that consumers would not respond well to genetically altered pork. The piglets are no longer available for purchase.
AquaBounty has been breeding its AquAdvantage salmon using genome editing tools. Scientists have carefully bred these fatty fish to grow at quicker rates than ordinary Atlantic salmon by inserting Chinook salmon growth hormone genes and ocean pout promoter regions into fertilized Atlantic salmon eggs. While the growth hormone genes of Atlantic and Chinook salmon are about 95% similar, the ocean pout promoter sequence was used since it controls genes that are always being expressed--salmon promoters only function in certain environmental conditions. However, the hormone is only functional in the presence of food. Scientists at AquaBounty strategically raise the salmon to feed whenever they like, as much as they please. Overall, the FDA approved GM salmon can grow to adult size in half the time as normal salmon and has reduced the amount of biomass required for food supplies. Even better, these fish are now not limited to the spring and summer season as they can be raised year-round. AquAdvantage is available in Canada, and by the end of 2020, it is set to be sold in the U.S. for families to enjoy!
While originally engineered by Taiwanese scientists to glow in and detect toxic environments, GloFish® has used the same methods to create their bio-fluorescent pet fish. By integrating the green fluorescent protein (GFP) gene from jellyfish into the DNA of zebrafish embryos, scientists at GloFish® have been able to sell these beautiful, glow-in-the-dark fish to millions of families in the U.S. looking for a little extra brightness in their homes!
AquAdvantage Salmon
Love salmon? These fish have been engineered to not only grow quicker, but also year-round!
Enviropig ™
While no longer available for use by farms, this pig held a possible solution to reduce harmful phosphorus levels in the environment!
GloFish®
Brighten up your home aquarium with these bioluminescent zebrafish!
Agriculture
In an effort to supply for the rapidly-growing population, scientists at Purdue University and the Chinese Academy of Sciences have used CRISPR editing tools to genetically modify rice for increased yields of the quintessential staple food of various cultures. Their modifications of 13 genes associated with phytohormone abscisic acid--an acid involved in “plant stress tolerance and suppression of growth”--were the basis of their experiments (In.C.16). Mutations of these genes proved to be immensely beneficial, with a significant 25-31% grain yield increase from traditional rice. Additionally, the teams collaborated to silence, or “knock-out” domains of PYL genes--known to “enhance tolerance to abiotic stresses” and stunt growth (In.C.16). The struggle here lies in the fact that plant genomes tend to have several repeated genes serving similar roles, so when one is knocked out, another could easily resume its function. The scientists worked to carefully “knock-out” the perfect combination of PYL genes to ensure stress tolerance while allowing maximum growth.
Bayer Crop Sciences and Pairwise have begun collaboration on several projects aiming to improve and refine agriculture using CRISPR. Their focus lies on five crucial crops: corn, wheat, soybeans, cotton, and canola. Seeking to rearrange these botanic genomes, Bayer and Pairwise are using Harvard science technologies to bring forth traits crucial to successful harvests, and silence those that limit these yields. We are a step closer to a more efficient food supply with CEO of Pairwise, Tom Adams, delightedly reporting that
“‘[t]he breadth of the gene-editing that we have developed unlocks tremendous potential to explore a variety of traits that will bring exciting changes to the produce aisle’” (In.C.17).
"Driven by the belief that food should be healthy, delicious, convenient, and sustainable, Pairwise brings together leaders in agriculture, technology, and consumer food to harness the transformative potential of new genomics technologies to create innovative new products" -Pairwise
As is, 70-80% of all foods in the U.S. contain some trace of GMO, and thus, it is harder to find more 'natural' products, like organics and non-GMOs, on grocery store shelves. While there is much reasoning as to why these products are healthier for consumption than their genetically modified counterparts, one thing holds true: the increased regulations for labeling GMO products drive prices up for consumers. Consumers are often left to choose their goods based on preference, since, in the end, the products boil out to about the same price: GMO prices are driven by regulatory processes and expensive manufacturing/development, while organic/non-GMO food costs are influenced by strict, labor-intensive production guidelines.
Economic Impact
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