Genetic engineeringholds enormous promise for combatinggenetic diseases. Here are some
ways it can be used:
1. Gene Editing Techniques:
oBase Editing: This technique allows precise changes in genes by altering a single
DNA letter.Researchers can correct specific mutations that cause diseases 1
.
oPrime Editing : It safely deletes or repairs long lengths of disease-causing DNA
or inserts DNA to fix dangerous mutations 1
.
2. Gene Therapy:
oIntroduction of Genetic Material: Gene therapy involves introducing genetic
material into a patient’s cells to treat or prevent disease.It has shown remarkable
success in clinical trials, especially for disorders with known genetic causes 2
.
oTargeting Specific Mutations : Researchers are developing therapeutics and
technologies that specifically target genetic mutations causing diseases like sickle
cell disease, Huntington’s disease, and heart disease 1
.
3. Specific Examples:
oHeart Disease: Interventions can help patients with a high genetic risk of heart
attack.For instance, a one-time intravenous drug is being tested to turn off a gene
in the liver, leading to lower LDL cholesterol levels and potentially preventing
heart attacks 1
.
oHuntington’s Disease : Research explores using a non-pathogenic virus (AAV) to
deliver gene therapy and mitigate the effects of the mutation causing
Huntington’s 1
.
In summary, genetic engineering offers hope for precise treatments and potential cures for a wide
range of genetic diseases.
Feldscher, K. (2022, November 30). Using gene editing to fight deadly genetic diseases. News.
https://www.hsph.harvard.edu/news/features/using-gene-editing-to-fight-deadly-genetic-
diseases/
Libretexts. (2021, December 23). 20.3: Genetic Engineering. Biology LibreTexts.
https://bio.libretexts.org/Bookshelves/Introductory_and_General_Biology/
Principles_of_Biology/02%3A_Chapter_2/20%3A_Biotechnology/
20.03%3A_Genetic_Engineering
Williamson, K. (2024, April 25). Revolutionizing health: The future of gene editing and genetic
engineering. Pharma Focus Europe.
https://www.pharmafocuseurope.com/articles/revolutionizing-health-the-future-of-gene-editing-
and-genetic-engineering
