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    Human Genome Editing To Prevent Genetic Diseases

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    Nicola Ferron
    This document discusses how genome editing technologies like CRISPR-Cas9 can be used to prevent genetic diseases. CRISPR-Cas9 allows scientists to modify, delete, or correct mutations in DNA that cause diseases. It works by using an enzyme called Cas9 and a guide RNA to cut the DNA at a targeted location, which can then be repaired in a way that fixes the mutation. This technology could potentially cure diseases caused by single mutations like sickle cell disease. It may also help treat cancers and neurological diseases by preventing certain disease-causing proteins. More research is still needed before applying genome editing to humans.

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    Human Genome Editing To Prevent Genetic Diseases

    Uploaded by

    Nicola Ferron
    31 views5 pages
    This document discusses how genome editing technologies like CRISPR-Cas9 can be used to prevent genetic diseases. CRISPR-Cas9 allows scientists to modify, delete, or correct mutations in DNA that cause diseases. It works by using an enzyme called Cas9 and a guide RNA to cut the DNA at a targeted location, which can then be repaired in a way that fixes the mutation. This technology could potentially cure diseases caused by single mutations like sickle cell disease. It may also help treat cancers and neurological diseases by preventing certain disease-causing proteins. More research is still needed before applying genome editing to humans.

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    Human Genome Editing to Prevent Genetic Diseases

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    This document discusses how genome editing technologies like CRISPR-Cas9 can be used to prevent genetic diseases. CRISPR-Cas9 allows scientists to modify, delete, or correct mutations in DNA that cause diseases. It works by using an enzyme called Cas9 and a guide RNA to cut the DNA at a targeted location, which can then be repaired in a way that fixes the mutation. This technology could potentially cure diseases caused by single mutations like sickle cell disease. It may also help treat cancers and neurological diseases by preventing certain disease-causing proteins. More research is still needed before applying genome editing to humans.

    Copyright:

    © All Rights Reserved
    Download as PDF, TXT or read online from Scribd
    Download as pdf or txt
    31 views5 pages

    Human Genome Editing To Prevent Genetic Diseases

    Uploaded by

    Nicola Ferron
    This document discusses how genome editing technologies like CRISPR-Cas9 can be used to prevent genetic diseases. CRISPR-Cas9 allows scientists to modify, delete, or correct mutations in DNA that cause diseases. It works by using an enzyme called Cas9 and a guide RNA to cut the DNA at a targeted location, which can then be repaired in a way that fixes the mutation. This technology could potentially cure diseases caused by single mutations like sickle cell disease. It may also help treat cancers and neurological diseases by preventing certain disease-causing proteins. More research is still needed before applying genome editing to humans.

    Copyright:

    © All Rights Reserved
    Download as PDF, TXT or read online from Scribd
    Download as pdf or txt
    of 5

    Human Genome Editing to Prevent Genetic Diseases

    Nic Ferron

    Courtice Secondary School

    SBI 4U1

    Ms. Buchner

    January 18, 2023


    HUMAN GENOME EDITING TO PREVENT GENETIC DISEASES 1

    Human Genome Editing to Prevent Genetic Diseases

    The revolutionary advancements in the world of human genome editing have allowed for

    the emergence of the CRISPR-Cas9 technologies, which permit for the prevention of genetic

    diseases. These developing technologies have the potential to put an end to many genetic

    diseases and disorders as well as ease their symptoms through therapy. Deoxyribonucleic acid

    (DNA) carries the entire genetic information for an organism; the idea behind human genome

    editing is that if scientists can find a way to split DNA during genetic replication they can then

    modify the genetic material (U.S. National Library of Medicine, 2022).

    All living organisms contain a unique genome which contains the complete set of an

    organism’s hereditary information. Over the past 20 years, scientists have mapped out 92% of all

    23 homologous chromosome pairs found in humans (Mooney, 2020). This accounts for almost 3

    billion base pairs which each code for the different creation of amino acids which build up

    polypeptide chains which altogether make up proteins. These proteins are the building blocks in

    every cell in the body and allow for life. As soon as a single base pair has undergone an error,

    known as a mutation, a different protein will be created. This is where genetic diseases can

    occur, known as mutations. Mutations are often looked at as a negative, however genome editing

    works through creating “positive” mutations to reverse the negative effects of mutations (U.S.

    National Library of Medicine, 2022).

    CRISPR-Cas9 is a technology that allows for scientists and doctors to modify, delete or

    correct the root cause of diseases and disorders found in specific strands of DNA. The gene

    editing system is made from two key pieces: Cas9 and a guide RNA. Cas9 is the enzyme that

    makes the cutting of DNA possible. The guide RNA binds to the Cas9 and guides it to a specific

    location in the DNA where the modification will occur. Also a part of the guide RNA is a target
    HUMAN GENOME EDITING TO PREVENT GENETIC DISEASES 2

    specific sequence which will be used to change the base pairings. Once the two pieces arrive at

    the site, the Cas9 binds and begins to unwind the DNA helix, allowing for the target specific

    sequence to bind to the single strand of DNA. Now that the two strands have paired together, the

    Cas9 enzyme cuts the strand off and the DNA undergoes its normal repair system. Once repaired,

    this results in a different strand of DNA being made that was not previously in the original

    genome, thus fixing the mutation. Through this splicing technique, scientists can attempt

    different variations to attack different types of mutations. These techniques are disruptions,

    deletions and corrections which all serve their own unique purpose. Disruptions occur when only

    a single site is modified and new base pairs are added in. Deletions are when two sites are

    modified and the DNA strand between the two is completely removed. Corrections are when two

    sites are modified, but rather than removing the old strand, it is replaced with another strand that

    is placed by a Cas9 enzyme (Charpentier, 2022).

    With the ability to interrupt genetic codes, scientists can locate specific gene sequences

    that code for genetic diseases and then modify them to treat the issue. When it comes to

    treatment, there are several different approaches. Diseases can be treated through disruptions,

    deletions and corrections to the gene depending on the disease's structure. Another approach is to

    modify the disease's DNA. Certain symptoms tied to diseases could be significantly reduced

    through the creation of a new protein due to the new genetic coding. For example, the effects of

    colour blindness could be reduced by introducing the protein to activate colour interpreting cones

    (Fernandez, 2021).

    Scientists believe that the first diseases to be cured by CRISPR will be small scale point

    mutations like sickle cell disease. This is the case because diseases like this are caused by a

    single mutation within a gene. Thus a simple deletion with CRISPR technologies would resolve
    HUMAN GENOME EDITING TO PREVENT GENETIC DISEASES 3

    the mutation. Unfortunately not all diseases are this simple and are far more intricate. Another

    application for this technology would be to treat cancer. Currently certain proteins bind to tumor

    cells and prevent an immune response against cancer. Using the CRISPR technology, scientists

    could significantly reduce the chances of certain types of cancer by preventing these proteins

    from being made by removing their coding (Kuruvilla et al., 2018). This is also the case for

    many neurodegenerative diseases like Alzhiemer’s or ALS. Currently, any genome editing that

    can be inherited by future generations is illegal on humans. For now, it is important to improve

    and develop the technology through tests on animals, similar to our gene structure like tilapia,

    before applying it on humans to avoid creating more issues than what scientists are already

    trying to cure (Li et al., 2021).


    HUMAN GENOME EDITING TO PREVENT GENETIC DISEASES 4

    References

    Mooney, C. (2020). The Human Genome : Mapping the Blueprint of Human Life. : Nomad Press.

    eBook Collection (EBSCOhost).

    Charpentier, E. (2022). CRISPR/Cas9. CRISPR Therapeutics.

    https://crisprtx.com/gene-editing/crispr-cas9

    Fernandez, C. R. (2021). Eight diseases CRISPR technology could cure. Labiotech.EU.

    https://www.labiotech.eu/best-biotech/crispr-technology-cure-disease/

    Kuruvilla, J., Sasmita, A. O., & Ling, A. P. K. (2018). Therapeutic potential of combined viral

    transduction and CRISPR/Cas9 gene editing in treating neurodegenerative diseases.

    Neurological Sciences, 39(11), 1827–1835. https://doi.org/10.1007/s10072-018-3521-0

    Li, M., Dai, S., Liu, X., Xiao, H., & Wang, D. (2021). A detailed procedure for

    CRISPR/Cas9-mediated gene editing in tilapia. Hydrobiologia, 848(16), 3865–3881.

    https://doi.org/10.1007/s10750-020-04414-8

    What are genome editing and CRISPR-Cas9?. (2022). U.S. National Library of Medicine.

    https://medlineplus.gov/genetics/understanding/genomicresearch/genomeediting/

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