CRISPR is one of this generation’s most profound innovations, with the potential to fundamentally alter the realm of modern day diagnostics and medical treatment. Scientists can use the tool to make changes to DNA at very specific locations to thereby alter the genome permanently, leading to modified traits and manifestations in organisms.
There are many potential applications. In crops, for example, this could mean enhancing specific traits to improve yield or resist disease. For humans, this can entail altering the fundamental building blocks of life in the genome to eliminate an individual’s potential for negative health outcomes.
One of the most profound breakthroughs using this technology was in the arena of sickle cell disease (SCD). Scientists were able to use CRISPR technology to modify blood stem cells to increase production of fetal hemoglobin, thereby decreasing levels of sickling in red blood cells. This was the first gene therapy to treat patients with SCD, and was approved by the FDA in 2023. Since then, steady progress has continued to be made in this field.
Another novel treatment succeeded in 2025, when scientists used CRISPR gene editing therapy to help an infant with a rare metabolic disorder, carbamoyl phosphate synthetase 1 (CPS1) deficiency. After just two infusions, the infant was found to be thriving significantly compared to baseline; as the authors noted in the study that was published in the New England Journal of Medicine,”In the 7 weeks after the initial infusion, the patient was able to receive an increased amount of dietary protein and a reduced dose of a nitrogen-scavenger medication to half the starting dose, without unacceptable adverse events and despite viral illnesses.” As Dr. Rebecca Ahrens-Nicklas, MD, PhD, director of the Gene Therapy for Inherited Metabolic Disorders Frontier Program (GTIMD) at Children’s Hospital of Philadelphia explains,”Years and years of progress in gene editing and collaboration between researchers and clinicians made this moment possible, and while [this] is just one patient, we hope he is the first of many to benefit from a methodology that can be scaled to fit an individual patient’s needs.”
Even as recent as last month, the Cleveland Clinic published results of a novel study which found that a newly designed CRISPR therapy reduced “bad” cholesterol and triglycerides by half in patients for nearly one year. As heart disease and hyperlipidemia remain one of the most fundamental causes of death and chronic disease burden both in the nation and worldwide, this creates significant opportunities for the decades ahead.
Nevertheless, the technology remains relatively niche, and fundamental questions still remain regarding its scalability.
For one, pricing is a major challenge. Given the significant amount of resources it takes to create a sustainable and safe therapy using the technology, price points remain high. Accounts indicate that some treatments can range upto $2.2 million per course, meaning that cost-effectiveness is a key consideration.
Another aspect is the infrastructure needed to actually deliver the treatments. This technology is not a one-size-fits-all approach or a simple pill that a pharmaceutical company has developed which can be mass produced. Rather, it is an intricate process combining biology, wet-lab sciences and hardware to be able to redesign and edit a human’s genome. This requires access to an entire healthcare and scientific network to undergo therapy and monitor the course of treatment.
Finally, awareness is paramount. Despite being a part of the mainstream scientific news for nearly a decade, CRISPR still remains largely unknown to a broad percent of the population. Why is this important? Because conversation drives movement. With more awareness among patients, more interest, funding and incentive to continue research in this arena is generated. If the technology is truly as valuable as its promise, then there is significant potential to fundamentally improve the lives of millions of people in an affordable and sustainable manner. Thus, the time to push this conversation forward is now.
This article was originally published onForbes.com
