Gene Editing & CRISPR Technologies – The Emerging Legal Questions
The therapeutic, ethical and legal implications of gene editing and CRISPR technologies are both profound and complex. However, before we delve into the potential panacea of gene editing technologies, a basic framework and understanding of how it all works is necessary. In its simplest terms, the human genome is the DNA (deoxyribonucleacacid), or set of instructions or assembly manual for an organism. The human genome is packaged into 46 chromosomes, one pair from each parent.
DNA contains 6 billion letters of a four (4) digit code known as “bases” or alphabets – Adenine (A); Cytosine (C); Guanine (G); and Thymine (T). This “genetic code” provides essential instructions which are read and translated into specific types of proteins. Moreover, these specific bases bind together to form “base pairs” “A-T” and “C-G”. Human DNA is composed of over three (3) billion base pairs. These bases are divided into triplets known as “codons” – code words for amino acids, which tell the cells which products to make, among other functions. “Transcription” is the process of reading the DNA and making a complimentary RNA copy. “Translation” is the process of reading the RNA and making proteins. Small changes in codons can have tremendous consequences. Mutations in coding regions can change a gene’s expression or function. Only about 2% of the genome consists of genes (i.e., coded regions).
There have been incredible developments in gene editing technologies such as Clustered Regularly Interspaced Short Palindromic Repeats (“CRISPR”), a family of DNA sequences founds in bacteria, are used with “Cas9” (an associated protein) to guide and “cleave” specific strands of DNA. In essence, CRISPR-Cas9 has been utilized to edit genes within various organisms, including humans. However, it can also be used as a valuable diagnostic tool as well. Also, gene editing technologies have lead to unprecedented advancements in food availability, safety and engineering.
Recent news has proven that CRISPR-Cas9, and other gene editing technologies can be both powerful and dangerous. For instance, in November 2018, there was a widely reported incident in China, where a researcher edited embryos to create twin baby girls with a deliberate mutation designed to prevent the HIV virus. This incident was especially troubling as the researcher in question moved forward with the experiment with no adherence to clinical safety procedures (for instance, there were no animal trials or previous testing).
However, while there have been calls within the scientific community for a voluntary moratorium on the clinical uses of gene editing (especially at the “Germline” level – or genetic code that can be passed down to offspring or “heritable DNA”), there is no worldwide regulatory framework that has been adopted. As of today, 40 countries across the world have banned germ-line gene editing procedures. In the US, the FDA regulates gene editing through the Center for Biologics Evaluation and Research (the “CBER”). In January 2020, the CBER released several “guidance” documents for the biologic industries related to recommendations for patient “follow-ups” after the administration of gene editing based therapeutics. However, the regulations have struggled to keep pace with the rapid pace of gene editing technologies. Likewise, the barriers to entry in the gene editing world are relatively low, resulting in easy access to these technologies outside a professional clinical setting.
As gene editing technology continue to advance, legislatures at the state and federal levels will have to keep up an establish a flexible approach to regulation that permits innovation and discovery, while holding preventing dangerous or inappropriate application of gene editing technologies.