In 2020, Jennifer Doudna and Emmanuelle Charpentier won the Nobel Prize in Chemistry for their revolutionary innovation - CRISPR Cas9. Previously, these gene editing tools were only used for research or drug discovery, but now they are being used to treat genetic disorders in humans.
With successful results in correcting mutations that cause single gene diseases like blindness and sickle cell disease, scientists are hopeful that more complex health issues like Alzheimer's and chronic pain can also be addressed.
This breakthrough has the potential to change lives and create a huge economic impact by 2023. However, there are ethical concerns that need to be addressed, requiring international dialogue and strict guidelines to ensure responsible and secure use by medical professionals.
What technologies are used in gene editing?
CRISPR technology came into being in 2012 as a breakthrough invention for gene editing that changed the course for scientific research upside down. The acronym stands for clustered regularly interspaced short palindromic repeats and was initially found within bacterial immunity systems, where it works against viral attacks by cutting their DNA structure.
CRISPR/Cas is a genetically centric engineering tool that can effectively cut distinct DNA sequences with an RNA template of the target sequence faster than any other available methods while also reducing costs drastically. This innovation has exhibited immense potential as a possible treatment for various genetic disorders such as blood disorders MND Huntingtons disease CFIDS blindness AIDS Covid 19 cancer or others.
Scientists are currently running tests on its efficiency levels with promising results already visible across different domains of science like genetics and biology. An example of this can be observed when scientists extracted immune T cells from an end stage lung cancer patient and using CRISPR technology removed the gene encoding the protein called PD 1 that some tumor cells bind to impair immune function.
CRISPR is currently undergoing testing as a potential one time treatment for sickle cell disease and transfusion dependent beta thalassemia.
CRISPR/Cas9 technology has opened up exciting new possibilities in the field of biology! Its an innovative approach that allows us to edit genes so as to treat various diseases effectively; however there's still much we need to learn about how it functions and what potential risks it could carry—as with all medical inventions. Thankfully, researchers are working hard to find ways around any obstacles they may encounter during their work testing out this novel therapeutic tool which promises great results for everything from mental illness causing genes down through treating various types of cancer.
Transcription Activator-Like Effector Nucleases
TALENs (transcription activator-like effector nucleases) are used in genome engineering, which involves the manipulation of genes in different organisms and cells. TALENs are formed by fusing a DNA-binding domain with a restriction enzyme, Fokl, and can produce double-stranded breaks in DNA for effective targeting of various gene sequences.
The adaptability and ease of TALENs techniques have led to the development of genetic engineering, and TALENs have various applications, including targeted insertion of DNA in potato plants, genome editing in plants and studying cell mutations in various organisms. TALENs can also be used to induce mutations in viruses like hepatitis B, HIV, and herpes, which are present in a latent state inside the body and are unaffected by treatment that inhibits viruses and their replications.
Zinc Finger Nucleases
Researchers at the University of Toronto and NYU Grossman School of Medicine have created an artificial intelligence technology called ZFDesign that can design zinc finger proteins to target any stretch of DNA in the human genome.
The researchers have fed data from billions of interactions between ZF proteins and DNA into a machine-learning model, which can then generate engineered zinc fingers that bind to the given DNA sequence.
This technology could help to develop gene therapies for a broader range of health conditions. Zinc fingers are a common class of human proteins that regulate gene expression, a process that transcribes genetic information into RNA molecules and proteins.
This technology carries a lot of exciting possibilities! It can be used to help fix genetic problems by replacing or repairing broken genes in gene therapy. It can also create new organisms with improved traits, like plants that are resistant to dry weather or crops that produce more food. And in agriculture, it can help create new types of plants that are better for us to consume.
Social Implications and Challenges
There are several medical dangers of gene editing, such as accidental unintended mutations in the DNA sequence, as well as immune responses that are the effects of different delivery methods of this form of theraphy.
Additionally, there are many ethical challenges that come with gene editing. Two experts, Sandy Sufian and Rosemarie Garland-Thomson, have written an opinion piece in Scientific American discussing the potential challenges of CRISPR-Cas9 gene-editing technology.
They note that the far-reaching promise of this technology is its ability to eliminate what medical science identifies as faulty or abnormal genes that cause differences in individual people, and that some scientists seem excited yet cautious about this.
The authors mention that the use of these “genetic scissors” will cut people like them, who have genetic differences, out of existence without others even noticing. While supporters of gene-editing may argue that editing out a gene-linked condition is different from editing out a person and curing disease is an indisputably good thing, the authors note that their genetic conditions are not simply entities that can be clipped away from them. They are whole beings, and their genetic conditions form a fundamental part of who they are.
They warn that the common belief that disease and anomaly from society is an incontrovertible good can lead quickly from the actual possibilities of science to fantasies of “improving” humanity where people become an aspirational, robotic version of themselves that is somehow better, stronger, smarter, and healthier. These technologies are also not accessible to everyone, which would lead to creation of a ‘genetic elite’ who has access to these types of genetic alterations, creating large societal inequalities.
Sufian and Garland-Thomson both have genetic conditions that many people consider serious enough to eliminate from the human gene pool, but they believe that their conditions have shaped their bodies and lives and that improved medical treatments, social progress, and political equality movements have raised their quality of life in ways that people like them in generations prior to theirs could not have imagined.