Sample interview questions: Can you explain the concept of CRISPR-based gene therapy and its potential applications?
Sample answer:
Concept of CRISPR-based Gene Therapy
CRISPR-Cas9 is a revolutionary gene-editing tool that leverages a system originally found in bacteria to target specific DNA sequences. In gene therapy, CRISPR-Cas9 allows for precise alterations to defective genes, potentially correcting genetic disorders and treating diseases at the source.
Process:
- Design of guide RNA: A guide RNA (gRNA) is designed to match the target DNA sequence.
- Formation of Cas9-gRNA complex: The Cas9 protein combines with the gRNA to form a complex that recognizes the specific DNA target.
- DNA cleavage: The Cas9 complex locates the target DNA and introduces double-stranded breaks, creating an opportunity for gene editing.
- Gene editing: The broken DNA is repaired through cellular mechanisms, such as non-homologous end joining (NHEJ) or homology-directed repair (HDR).
Potential Applications:
- Treatment of genetic disorders: CRISPR-based gene therapy offers the potential to cure diseases caused by single-gene defects, such as sickle cell anemia, cystic fibrosis, and muscular dystrophy.
- Cancer immunotherapy: By modifying immune cells with CRISPR, researchers can enhance their ability to recognize and attack cancer cells.
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