Cystic Fibrosis, the CRISPR Solution, and Ethical Considerations.

Abstract

The world of genetics is complex and increasingly astounding as scientists continue to uncover layers of its depths. DNA, the genetic coding design, is responsible for the successful functioning of the human body. Yet a miscoding of this critical blueprint can be detrimental. Cystic Fibrosis (CF), an autosomal recessive genetic disorder, is just one example of the impact a genetic defect can have on the entire functioning of the human body. Stemming from a flaw in the Cystic Fibrosis Transmembrane Conductance Regulator gene, the mutation causes salt and water to be imbalanced in the body, resulting in thick mucus that inhibits primarily the respiratory and digestive systems. A myriad of treatment methods have been developed to downstream the effects of the disease. Medications are being researched in the area of gene therapy in an effort to successfully inject healthy genes to replace defective ones. Most recently scientists are working to correct mutations at their DNA core, through remarkable gene editing technology; the most popular of which is the CRISPR/ Cas 9 system. By cutting a DNA sequence at a specific locus, a mutation can be cut out and potentially corrected for a lifetime. Prospects are exciting while ethical concerns are daunting. But the possibility of correcting previously believed incurable genetic disorders continues to propel research and complex discussions in both the scientific and Jewish religious communities.