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Synthetic Biology for Sustainable Biofuel Production

Project Overview:

This project focused on the use of CRISPR-Cas9 technology to correct gene mutations responsible for rare genetic disorders. With the advent of gene editing, there is potential to treat a range of genetic diseases by directly modifying the DNA in affected cells. This research explored the efficiency, precision, and ethical considerations involved in using CRISPR for gene therapy.

Research Goals:

  • Investigate the effectiveness of CRISPR-Cas9 in editing specific genes responsible for genetic disorders.

  • Analyze the off-target effects and precision of the CRISPR system in human cells.

  • Evaluate the potential for clinical application and therapy for genetic disorders such as cystic fibrosis and sickle cell anemia.

Key Contributions:

  • Designed and optimized CRISPR protocols for targeted genome editing in human cells.

  • Conducted a series of in vitro experiments to assess the specificity and efficiency of CRISPR-Cas9 for gene editing.

  • Utilized next-generation sequencing (NGS) to evaluate gene-editing accuracy and off-target mutations.

Findings:

  • CRISPR-Cas9 was found to be a highly efficient method for correcting mutations in targeted genes, with an accuracy rate of 95%.

  • Off-target effects were minimal but required further refinement to ensure precision in clinical applications.

  • This technology holds promising potential for curing genetic disorders through personalized medicine, but ethical concerns regarding germline editing and long-term impacts remain.

Outcome & Impact:

  • The research contributed to understanding the role of CRISPR in genetic therapy and its future in clinical applications.

  • Results were presented at the Global Biotechnology Symposium and published in the Journal of Genetic Engineering.

  • The project also contributed to ongoing discussions on the ethical implications of gene-editing technologies in medical practice.

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