What Is Genetic Engineering?
Genetic engineering is defined as the deliberate modification of an organism's DNA to achieve specific traits or purposes commonly related to health issues. The field began in the mid-20th century through crops and other foods which has now paved the way to editing human genomes as well. In opposition to enhancing the genome of fruits, for example, scientists aim to cure genetic diseases across the world to eliminate certain illnesses.
What's The Point?
The purpose of genetic engineering in humans lies in its potential to revolutionize healthcare and improve lives. Some examples of this are:
-Gene therapy could provide treatments for diseases like cystic fibrosis or sickle cell anemia by correcting defective genes.
-Cancer research uses genetic engineering to develop therapies that target specific mutations in cancer cells.
-Personalized medicine tailors' treatments to an individual's genetic makeup for greater efficacy and fewer side effects.
How Does It Get Into The Genome?
Retroviruses are modified to carry therapeutic genes instead of their original viral genes. These engineered viruses can deliver the desired genetic material into target cells. They convert their RNA genome into DNA using reverse transcriptase. This DNA is then integrated into the host cell's genome, leading to long-term expression of the introduced gene.
Adeno-associated viruses (AAVs) are engineered to carry therapeutic genes into target cells. They are non-pathogenic and have low immunogenicity, making them ideal for gene therapy applications. In most cases, AAVs remain as episomes (circular DNA) in the nucleus without integrating into the host genome. This is the primary mode of persistence for AAVs and is considered safer for gene therapy applications.
CRISPR-Cas9 uses a guide RNA (gRNA) to identify and bind to a specific DNA sequence in the genome. The gRNA is designed to match the target sequence, ensuring accuracy. The Cas9 enzyme acts as molecular scissors, cutting the DNA at the target site. This creates a double-strand break in the DNA. The cell's natural repair mechanisms then take over to completely work this into the host's genome.