An Examination of Gene Editing and Gene Therapy in Genetic Diseases
DOI:
https://doi.org/10.58445/rars.834Keywords:
Gene Editing, Gene Therapy, Genetic DiseasesAbstract
The field of gene editing and therapy has helped the scientific community understand treatment
options related to different genetic diseases such as ADA-SCID, Huntington's disease, and
cystic fibrosis. Research has shown that CRISPR, a gene editing tool, has helped in treating
genetic diseases that currently do not have conventional treatments available. Although being a
greatly beneficial tool, CRISPR comes with its limitations such as leading to a possible risk of
cancer through off-target edits. Gene therapy is a direct approach to treating genetic diseases,
such as ADA-SCID, through either in-vivo or ex-vivo applications. This paper analyzes the
application of gene editing and gene therapy for ADA-SCID, Huntington's disease, and cystic
fibrosis, while also considering the ethical implications.
References
ALZForum. “Gene Editing for Huntington's Disease Shows Promise in Pigs | ALZFORUM.” Alzforum, 21 February 2023,
https://www.alzforum.org/news/research-news/gene-editing-huntingtons-disease-shows-promise-pigs. Accessed 26 December 2023.
Ayanoğlu, Fatma Betül, et al. “Bioethical issues in genome editing by CRISPR-Cas9 technology.” Turkish journal of biology = Turk biyoloji dergisi vol. 44,2 110-120. 2 Apr. 2020, doi:10.3906/biy-1912-52
Carroll, Dana. "Focus: Genome Editing: Genome Editing: Past, Present, and Future." The Yale Journal of Biology and Medicine, vol. 90, no. 4, 2017, pp. 653-659,
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5733845/. Accessed 24 Nov. 2023.
Chaney, Patricia, and Jodi Helmer. “Using Viral Vectors for Gene Therapy Treatments.” Pfizer, 17 December 2018,
https://www.pfizer.com/news/articles/customizing_viral_vectors_to_further_gene_therapy
_innovation. Accessed 26 December 2023.
“CRISPR-Cas Gene Editing Teaching Resources.” Bio-Rad,
https://www.bio-rad.com/en-us/applications-technologies/crispr-cas-gene-editing-teaching
-resources?ID=Q58I0DWDLBV5. Accessed 23 November 2023.
“Cystic Fibrosis.” CDC, 9 May 2022,
https://www.cdc.gov/genomics/disease/cystic_fibrosis.htm. Accessed 23 November 2023.
Cystic Fibrosis Foundation (CFF). “Gene Editing for Cystic Fibrosis.” Cystic Fibrosis Foundation, 2023, https://www.cff.org/research-clinical-trials/gene-editing-cystic-fibrosis. Accessed 27 December 2023.
Fliesler, Nancy. “A short history of gene therapy - Boston Children's Answers.” Boston Children's Answers, 22 December 2020, https://answers.childrenshospital.org/gene-therapy-history/. Accessed 23 November 2023.
“Huntington's disease.” MedlinePlus, 1 July 2020,
https://medlineplus.gov/genetics/condition/huntingtons-disease/. Accessed 23 November 2023.
MedlinePlus. “Adenosine deaminase deficiency.” MedlinePlus, 1 July 2013,
https://medlineplus.gov/genetics/condition/adenosine-deaminase-deficiency/#inheritance. Accessed 26 December 2023.
MedlinePlus. “How does gene therapy work?” MedlinePlus, 28 February 2022,
https://medlineplus.gov/genetics/understanding/therapy/procedures/. Accessed 26 December 2023.
MedlinePlus. “What are genome editing and CRISPR-Cas9.” MedlinePlus, 22 March 2022,
https://medlineplus.gov/genetics/understanding/genomicresearch/genomeediting/. Accessed 26 December 2023.
Moritz, Juliet, et al. “Ashanthi DeSilva's Story: A Look Back at the First Gene Therapy Trial.”
Premier Research, 14 November 2018,
https://premier-research.com/blog-ashanthi-desilva-gene-therapy-trial/. Accessed 26 December 2023.
National Cancer Institute (NCI). “How CRISPR Is Changing Cancer Research and
Treatment.” National Cancer Institute, 27 July 2020,
https://www.cancer.gov/news-events/cancer-currents-blog/2020/crispr-cancer-research-treatment. Accessed 27 December 2023.
National Heart, Lung, and Blood Institute (NHLBI). “Cystic Fibrosis - Causes.” NHLBI, 21
November 2023, https://www.nhlbi.nih.gov/health/cystic-fibrosis/causes. Accessed 25 December 2023.
National Institutes of Health (NIH). “Homologous Recombination.” National Human Genome
Research Institute, 19 December 2023,
https://www.genome.gov/genetics-glossary/homologous-recombination. Accessed 25 December 2023.
National Institutes of Health (NIH). “How Does Genome Editing Work?” National Human Genome Research Institute, 3 August 2017,
https://www.genome.gov/about-genomics/policy-issues/Genome-Editing/How-genome-editing-works. Accessed 25 December 2023.
NYU Grossman School of Medicine (NYU). “Gene Therapy Research & the Case of Jesse
Gelsinger.” NYU Langone Health, 2023,
https://med.nyu.edu/departments-institutes/population-health/divisions-sections-centers/medical-ethics/education/high-school-bioethics-project/learning-scenarios/jesse-gelsinger-case. Accessed 27 December 2023.
Sharman, Sarah, and Cathleen Shaw. “Huntington's Disease Research: Shutting down the mutant gene.” HudsonAlpha Institute for Biotechnology, 10 June 2021,
https://www.hudsonalpha.org/huntingtons-disease-research-shutting-down-the-mutant-gene/. Accessed 23 November 2023.
Solomon, Benjamin. “Gene Therapy.” National Human Genome Research Institute, 2023,
https://www.genome.gov/genetics-glossary/Gene-Therapy. Accessed 23 November 2023.
Westermann, Lukas, et al. "Nobel Prize 2020 in Chemistry Honors CRISPR: A Tool for
Rewriting the Code of Life." Pflugers Archiv, vol. 473, no. 1, 2021, pp. 1-2, https://doi.org/10.1007/s00424-020-02497-9. Accessed 26 Dec. 2023.
Wu, Xuebing, et al. "Target Specificity of the CRISPR-Cas9 System." Quantitative Biology, vol. 2, no. 2, 2014, p. 59, https://doi.org/10.1007/s40484-014-0030-x. Accessed 26 Dec. 2023.
Yan, Sen, et al. “A Huntingtin Knockin Pig Model Recapitulates Features of Selective Neurodegeneration in Huntington's Disease.” Cell vol. 173,4 (2018): 989-1002.e13. doi:10.1016/j.cell.2018.03.005
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