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Assignment sample solution of BIO401 - Advanced Genetic Engineering

CRISPR-Cas9 Gene Editing and Its Implications on Genetic Disorders

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Biology Assignment Sample

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CRISPR-Cas9 is a revolutionary tool in genetic engineering, allowing precise editing of genomes. Discovered as a bacterial immune system, CRISPR uses a guide RNA (gRNA) to direct the Cas9 enzyme to specific DNA sequences, creating double-stranded breaks. These breaks are repaired by the cell’s natural repair mechanisms, namely Non-Homologous End Joining (NHEJ) or Homology-Directed Repair (HDR).

The applications of CRISPR-Cas9 in genetic disorders are vast. For instance, in diseases like sickle cell anemia, CRISPR can target the HBB gene mutation and repair it, restoring normal hemoglobin function. Similarly, in cystic fibrosis, CRISPR can correct the defective CFTR gene. Beyond single-gene disorders, CRISPR holds promise in treating complex diseases like cancer by modifying immune cells (e.g., CAR-T therapy).

 

Despite its potential, CRISPR faces ethical concerns, including the risk of off-target effects and germline editing, which could alter human evolution. Regulatory frameworks in countries like the UK and Australia emphasize balancing innovation with ethical considerations. Research continues to refine CRISPR’s precision, making it a cornerstone in personalized medicine and a subject of intense academic and industrial focus.