Biotechnology: Gene Editing Techniques

Over the last half century after post-DNA helical structure discovery, the world has seen a continuous staircase outburst of various molecular technologies, which are now heading forward toward translation into clinical and laboratory practice. Given the availability of sequencing platforms, acquired wisdom about the micro-mechanics at work within the genetic apparatus, and the introduction of user-friendly nanotechnologies, it was possible for next-generation scientists to manipulate the genetic codes at various levels. Principally, gene editing techniques can be interpreted as methods where DNA sequences are changed by deletions, mRNA processing, and post-transcriptional modifications to result in altered gene expression, leading to functional behavior of proteinsGene editing is performed using enzymes, particularly nucleases that have been engineered to target a specific DNA sequence, where they introduce cuts into the DNA strands, enabling the removal of existing DNA and the insertion of replacement DNA.

The enormous knowledge and ongoing research have now been able to demonstrate methodologies that can alter DNA coding. The gene editing techniques evolved from the earlier attempts like nuclease technologies, homologous recombination, and certain chemical methods (peptide nucleic acid). Molecular techniques like meganuclease, transcription activator-like effector nucleases (TALENs), and zinc-finger nucleases (ZFNs) initially emerged as genome-editing technologies.

These initial technologies suffer from lower specificity due to their off-targets side effects. Moreover, from biotechnology's perspective, the main obstacle was to develop simple but effective delivery methods for host cell entry. The latest discovery of CRISPR/Cas9 technology seems more encouraging by providing better efficiency, feasibility, and multi-role clinical application.

The significant leap in gene editing techniques brought new urgency to long standing discussions about the ethical concerns surrounding the genetic engineering. Many questions, such as whether genetic engineering should be used to treat human disease or to alter traits such as beauty or intelligence, had been asked in one form or another for decades. This course generally discusses the various gene editing techniques in terms of the mechanisms of action, advantages, and side effects.