Gene Therapy and The Rise of CRISPR Technology in Modern Medicine

Amreen 1
Sheikh Fabiha Amreen
Mirpur Cantonment Public School and College
[email protected]
5 October 2024
Gene Therapy and the Rise of CRISPR Technology in Modern Medicine
Introduction
We sometimes lack knowledge on the truly wondersome ability of modern medicine
as we are submerged in a blindfolded loop to current advancements in recent years, one such
would be the promising developments of Gene Therapy. The development of CRISPR-Cas9
technology has almost skyrocketed its productivity and efficiency for editing the human
genome. This essay will briefly discuss and demonstrate the magic-like wonder of this tool
and its application in genetic disorders, what challenges it is still bound by and the ethical
implications of its use. CRISPR/Cas9 technology has the potential to revolutionize the
treatment of many pediatric conditions.
CRISPR: A Revolutionary Gene-Editing Tool
CRISPR/Cas9 is a gene-editing technology which involves two essential components:
a guide RNA to match a desired target gene, and Cas9 (CRISPR-associated protein 9)—an
endonuclease which causes a double-stranded DNA break, allowing modifications to the
genome. One of the most exciting applications of CRISPR/Cas9 is its potential use to treat
genetic disorders caused by single gene mutations. Examples of such diseases include cystic
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fibrosis (CF), Duchenne's muscular dystrophy (DMD) and haemoglobinopathies. Preclinical
validations is already readily available yet there is still some steps to be taken to ensure
clinical practice.
Schwank et al used CRISPR/Cas9 to investigate the treatment of CF. They obtained adult
intestinal stem cells from two patients with CF and modified the most common mutation
causing the illness. Once the mutation had been corrected, the function of the CF
transmembrane conductor receptor (CFTR) was restored.
Therapeutic Applications of CRISPR
CRISPR/Cas9 has the potential to cure genetic illnesses resulting from single gene
mutations. These illnesses include hemoglobinopathies, Duchenne's muscular dystrophy
(DMD), and cystic fibrosis (CF). Even though it hasn’t faced clinical trials the preclinical
data are promising. In a recent study, researchers successfully corrected the gene mutation
associated with Duchenne muscular dystrophy in mice, leading to improved muscle function
(Long et al., 2021).
Beyond genetic disorders, CRISPR is also being investigated for its potential to
combat complex diseases like cancer and HIV. By editing the immune cells of patients,
CRISPR can enhance their ability to target and destroy cancer cells or resist HIV infection.
The versatility of CRISPR positions it as a powerful tool with far-reaching implications for a
wide range of diseases.
Challenges and Ethical Considerations

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The use of CRISPR-Cas9 and genome therapy in the germline can be classified into
two main topics depending on the success and failure of its editing technologies (Ormond et
al., 2017; Greely, 2019).
A different dilemma of the technology is patenting. As is known, transgenic
organisms of industrial use and also some human gene sequences for clinical purposes have
been patented (Rodriguez, 2016; Sherkow, 2018). Ever Evolving technologies as such will
attract patent related issues in the upcoming years. Zhang and Doudna and Charpentier has
both filed for the therapeutic use of CRISPR-Cas9 in human cells. In the end it was decided
to grant the patent to Caribou Biosciences, which Doudna was the founder of (Donohoue et
al., 2018).
Conclusion
After decades of research and development, CRISPR/Cas is no longer restricted to
cleaving DNA strands. Instead, it has given rise to a broad range of transcriptional regulation,
RNA strand cutting, and single-base gene editing techniques. As a result, the majority of
human diseases, including cancer, chronic illnesses, and hereditary disorders brought on by a
single gene, may be treated using these methods
As we continue to explore the potential of this groundbreaking technology, it is
essential to prioritize safety, efficacy, and the well-being of patients. By addressing these
concerns, we can harness the power of CRISPR to improve human health and unlock new
frontiers in medicine.
Works Cited
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Long, C., et al. (2021). CRISPR-Cas9 corrects Duchenne muscular dystrophy in mice.
Science, 373(6559), 870-875.
Vakulskas, C. A., et al. (2020). CRISPR-Cas9 gene editing in human cells: Off-target effects
and their implications. Nature Medicine, 26(1), 25-34.
Frangoul, H., et al. (2020). CRISPR-Cas9 gene editing for sickle cell disease and β-
thalassemia. New England Journal of Medicine, 384(3), 252-260.
Schwank G, Koo BK, Sasselli V, et al.. Functional repair of CFTR by CRISPR/Cas9 in
intestinal stem cell organoids of cystic fibrosis patients. Cell Stem Cell 2013;13:653–
8. 10.1016/j.stem.2013.11.002

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Amreen 1

Sheikh Fabiha Amreen

Mirpur Cantonment Public School and College

Gene Therapy and the Rise of CRISPR Technology in Modern Medicine

We sometimes lack knowledge on the truly wondersome ability of modern medicine

would be the promising developments of Gene Therapy. The development of CRISPR-Cas9

treatment of many pediatric conditions.

CRISPR: A Revolutionary Gene-Editing Tool

CRISPR/Cas9 is a gene-editing technology which involves two essential components:

endonuclease which causes a double-stranded DNA break, allowing modifications to the

fibrosis (CF), Duchenne's muscular dystrophy (DMD) and haemoglobinopathies. Preclinical

transmembrane conductor receptor (CFTR) was restored.

Therapeutic Applications of CRISPR

mutations. These illnesses include hemoglobinopathies, Duchenne's muscular dystrophy

(Long et al., 2021).

wide range of diseases.

Challenges and Ethical Considerations

al., 2017; Greely, 2019).

A different dilemma of the technology is patenting. As is known, transgenic

After decades of research and development, CRISPR/Cas is no longer restricted to

single gene, may be treated using these methods

As we continue to explore the potential of this groundbreaking technology, it is

Science, 373(6559), 870-875.

and their implications. Nature Medicine, 26(1), 25-34.

thalassemia. New England Journal of Medicine, 384(3), 252-260.

Schwank G, Koo BK, Sasselli V, et al.. Functional repair of CFTR by CRISPR/Cas9 in

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