EXECUTIVE SUMMARY 

• On December 08, 2023, the U.S. Food and Drug Administration (FDA) approved Casgevy and Lyfgenia, the first CRISPR-based treatment for sickle cell disease.  

• Gene editing marks a major milestone in the potential to cure genetic disorders but, the process has raised many ethical concerns.

• As gene editing is a relatively recent advancement, there is room for extensive policy work about its technicalities and applications.

Introduction 

Gene editing has recently evolved into one of the most revolutionary medical advancements in history. The technology at the vanguard of gene editing is known as CRISPR, which is short for clustered regularly interspaced short palindromic repeats. CRISPR was originally discovered as a bacterial defense system. It is comparatively more technical and cost efficient than other gene editing technologies. Now, it holds incredible potential for groundbreaking applications in medicine, agriculture, and genetics. 

Potential for Cross-Sector Advancements: 

Gene editing has created more effective and targeted treatment methods of rare diseases that once had limited options. For instance, in the case of blood disorders like sickle cell disease and beta thalassemia, CRISPR showed very promising results in a clinical trial held in October 2023. There are more ongoing trials and clinical research for treatment of other complex diseases such as cystic fibrosis, Huntington’s disease, and muscular dystrophy. A notable success story would be in 2019 with the case of Victoria Gray, sickle cell patient who was the first person to receive CRISPR gene editing treatment in the U.S. Furthermore, CRISPR could potentially lead to major developments in cancer treatment through targeting specific gene mutations. Promising studies in organ transplantation indicate the possibility of preventing organ rejection, thus, making transplants more viable and accessible. 

In agriculture, gene editing holds incredible potential to yield more nutritious crops, tolerance to extreme weather and many others. As the frequency of natural disasters are on the rise, gene editing technology could be a useful tool in positively impacting the durability of plants and produce. It also lessens the need for chemical inputs and avenues of encroachment on biodiverse ecosystems while providing protection against crop losses from pests. 

Ethical Questions Raised by Gene Editing: 

The potential of gene editing also brings about complex ethical challenges. In 2014, once a team in China had altered the DNA of a monkey to introduce customized mutations, though it seemed that wasn’t sufficient to cross the ethical line of medical conduct. Although, the concept of ‘designer babies’ made through germline therapy – when changes are made to the DNA of reproductive cells and are passed down from generation to generation – is a cause for concern in the medical community as it could lead us down a slippery slope using gene editing for non-therapeutic and trait enhancement purposes. As with many technological advancements, there is concern that if genetic enhancements become available, they will be limited to the wealthier population due to its high cost thereby increasing healthcare disparities between social classes and ultimately, germline editing could result in classes of individuals defined by the quality of their edited genome.  

Additionally, an area of worry in gene editing is privacy. If gene editing in fact becomes developed and widely practiced there is an increased risk of gene terrorists or even the government using our genetic information without our knowledge or permission. 

Public Perception and Societal Impact 

Public opinion on gene editing varies and is dependent on societal norms and ethical values. The consensus on the use of CRISPR is supportive when used for treating serious diseases, but against the use of gene editing for trait-enhancing purposes. According to a Pew Research Center Survey of 10,260 U.S. adults in 2021, 30 percent of Americans each think that using gene editing to lower the occurrence of severe diseases is good and bad idea while 39 percent are not sure. This prompt among many others in the survey seems to highlight the societal hesitance towards the potential of gene editing to change the general trends of human identity and reduce variance in social dynamics. 

According to the National Human Genome Research Institute, although the United States doesn’t explicitly ban germline editing, the Appropriations Act of 2016 passed in December 2015, prohibits the Food and Drug Administration (FDA) from reviewing any research around embryonic germline editing. Furthermore, the NIH publicly stated its refusal to fund any germline genome editing projects involving human embryos due to ethical and safety concerns. Despite this, in 2017 through private funding, scientists at the Oregon Health and Science University in Portland were able to successfully perform the first gene editing on embryos.  

Regulatory Initiatives 

With the complexities that gene editing practices face, the presence of regulations makes the ethical boundaries easier to navigate. Most regulations are focused on Germline editing which results in heritable genetic changes rather than Somatic editing which yields non-heritable changes as it is focused on targeting cells which make up nonreproductive organs and tissues. In 2017, the U.S. Food and Drug Administration (FDA) issued the Chemistry, Manufacturing, and Control Information for Human Gene Therapy Investigational New Drug Applications to clarify the guidelines for editing human cells, including safety and efficacy protocols. Following the CRSIPR gene editing scandal involving Chinese scientist He Jiankui in 2018, after deeming germline editing illegal, the Chinese government created the Measures for Ethical Review of Life Sciences and Medical Research Involving Humans. The challenge with this is that the rules do not apply to the private sector which creates an imbalance in fully regulating gene editing practices in various countries. These are only a few examples of regulations on germline editing, as other countries are trying to navigate balancing the strive for medical development with ethical boundaries.   

Conclusion: 

The potential of CRISPR and other gene editing programs is very promising. However, it is burdened with significant ethical concerns and regulatory inconsistencies so, it is essential to continue establishing clear frameworks and regulations. Public discourse and dissemination of information must also be encouraged to guarantee the progression of gene editing alongside societal values.