Advances in genetic science are opening new possibilities in how we understand and treat disease. Among the most talked-about developments are gene therapy and gene editing – two approaches that hold the potential to modify, and potentially even cure, diseases by targeting their genetic roots.
Although often discussed together, these technologies are not interchangeable. Each offers distinct mechanisms, applications, and implications – scientifically, ethically, and clinically. With gene therapy already in clinical use and gene editing rapidly evolving, it’s increasingly important to understand what sets them apart.
This article explores the core concepts behind each technology, highlights their current and emerging roles, and considers what their continued development could mean for patients, healthcare systems, and the future of medicine.
Understanding the approaches
Gene therapy
Gene therapy aims to treat or prevent disease by introducing genetic material – DNA – into a person’s cells. The goal is to compensate for faulty or missing genes, often by adding a working copy of the gene rather than fixing the original.
Most gene therapies today use viral vectors, which are modified viruses that act as delivery vehicles to transport healthy genes into target cells. Once inside, these genes can begin producing the proteins or enzymes that were missing or defective due to the genetic mutation. This approach is especially useful for conditions caused by a single faulty gene, such as spinal muscular atrophy (SMA) or beta-thalassaemia, where restoring a single biological function can have significant clinical impact.
Importantly, gene therapy does not typically alter the patient’s existing DNA. Instead, it introduces new genetic instructions that can exist alongside the original faulty code, often in non-dividing cells where long-term expression is possible.
Gene editing
Gene editing, in contrast, involves directly modifying the DNA sequence itself, by correcting a mutation, deleting a faulty section, or inserting new code at a specific gene location.
One of the most widely known tools for this is CRISPR-Cas9, a molecular system adapted from bacteria that works like a pair of genetic scissors. Guided by a synthetic RNA sequence, the system locates the target DNA sequence within the faulty gene and makes a cut, after which the cell’s own repair machinery can introduce the desired changes.
This precision enables the potential for one-time, permanent corrections to the genome, with possible applications in conditions ranging from cystic fibrosis to Huntington’s disease and even some cancers. Early clinical trials have shown promising results, particularly in ex vivo approaches where cells are edited outside the body and then reinfused.
While still largely in the experimental phase, gene editing is advancing rapidly and may become a cornerstone in the next generation of personalised medicine.
What sets them apart and where they’re headed
While gene therapy and gene editing both aim to address disease at the genetic level, they differ in how they work, what they treat, and stage of development.
Mechanism of action
- Gene therapy adds a new copy of a gene to support or restore a cellular function.
- Gene editing modifies the original DNA sequence by correcting, deleting, or rewriting the genetic code at specific locations.
Therapeutic goals
- Gene therapy is already being used to compensate for faulty genes in rare diseases.
- Gene editing holds promise for precise, potentially permanent corrections, especially in single-gene disorders.
Stage of maturity and outlook
- Gene therapy has reached a clinical milestone, with several approved treatments now in use. Efforts are underway to improve delivery, extend durability, and expand indications into more common diseases.
- Gene editing is still largely experimental, with most clinical activity limited to early-stage trials and ex vivo The next frontier is in vivo editing, where cells are modified directly within the body – offering transformative potential but requiring greater precision and regulatory oversight.
In the years ahead, these technologies may enable more personalised, durable, and potentially curative treatments across a wide range of conditions. But their success will depend not only on scientific advances, but also on robust regulatory frameworks, ethical safeguards, and accessible delivery models.
Safety and ethical considerations
Both approaches raise important questions, particularly around long-term safety, off-target effects, and equity of access. Gene editing, in particular, carries additional ethical weight when involving germline cells (sperm and egg cells), as changes could be inherited by future generations.
As these tools move closer to mainstream care, thoughtful engagement with patients, regulators, and the public will be essential.
Summary
Gene therapy and gene editing are reshaping what’s possible in modern medicine. Though often discussed together, they represent distinct scientific approaches: one supplements faulty genes, while the other rewrites them.
Gene therapy is already in clinical use, showing real-world impact in treating rare inherited conditions. Gene editing, while earlier in its journey, is advancing quickly with the potential to expand the reach of personalised medicine and redefine what ‘treatable’ means in genetic disease.
Understanding the differences, and the future potential, of these technologies is not just a matter of scientific interest. It’s a strategic imperative for those working at the intersection of biotech innovation, healthcare delivery, and patient outcomes.
If you’re looking to translate complex science into clear, engaging narratives that resonate with both expert and non-expert audiences, get in touch with us. We help biotech and pharma teams communicate with purpose, precision, and impact – at every stage of innovation.