The Future of Gene Editing: Curing Disease Without Limits
▼ Summary
– David Liu’s lab plans to report a “disease-agnostic therapeutic gene editing” strategy later this year, aiming to treat many unrelated diseases with one approach.
– Current gene-editing treatments target specific mutations for rare diseases, such as Casgevy for sickle cell and beta thalassemia, and a customized treatment for baby KJ’s CPS1 deficiency.
– These existing treatments are costly to develop and designed for small patient populations, limiting their practicality for numerous rare conditions.
– Liu argues that a streamlined, universal approach is necessary because genetic diseases collectively affect over 400 million people worldwide across at least 10,000 rare diseases.
– He emphasizes the urgency of directly treating genetic diseases, noting they are more prevalent than cancer or HIV/AIDS and require addressing root causes.
At the recent WIRED Health summit, Harvard biochemist and gene-editing innovator David Liu announced that his laboratory intends to reveal a groundbreaking single gene-editing strategy later this year. This approach, which he terms disease-agnostic therapeutic gene editing, holds the potential to address numerous unrelated illnesses through a unified molecular framework.
Liu captivated the Boston audience by explaining that while the concept may sound ambitious, it rests on solid molecular biological principles. He chose not to disclose full details at this stage, but the implications are profound for the future of genetic medicine.
Current gene-editing therapies focus on targeting specific mutations responsible for particular rare or inherited conditions. For instance, Casgevy has received commercial approval for treating sickle cell disease and beta thalassemia. In another landmark case earlier this year, an infant named KJ Muldoon received a customized gene-editing intervention for CPS1 deficiency, a typically fatal disorder causing toxic ammonia accumulation. His successful treatment marked a medical milestone.
However, developing these therapies is often expensive and tailored to narrow patient groups. Conditions like KJ’s affect only about one in 1.3 million births, making individualized treatments economically and logistically challenging.
Liu’s vision involves a universal editing platform applicable across diverse diseases, irrespective of the affected organ or genetic origin. He emphasizes the necessity of such a streamlined approach, noting that rare diseases collectively impact over 400 million people globally. According to advocacy group Global Genes, more than 10,000 rare diseases exist, a number that underscores the impracticality of creating bespoke therapies for each one.
Liu pointed out that genetic diseases as a category are far from uncommon. In fact, they affect significantly more people worldwide than cancer or HIV/AIDS. The urgent need, he stressed, is for strategies that directly tackle the root genetic causes rather than merely managing symptoms. His work aims to provide exactly that, a versatile, scalable solution capable of rewriting the future of genetic healthcare.
(Source: Wired)
