A Promising Step Forward: Gene Therapy Progress for NF1

Researchers supported by NTAP have announced an exciting breakthrough in gene therapy for NF1. For the first time, scientists have created a smaller, working version of the NF1 gene, called a “mini-NF1,” and packaged it into a specially engineered viral vector that can target tumor cells more effectively.

In early studies using mice, this approach significantly slowed tumor growth and showed far better targeting of tumor tissue than previous attempts. The team is now working through safety and dosing studies, with plans to move toward larger-animal testing and eventually human clinical trials.

While this is still in the early stages, it represents a bold and hopeful step toward treatments that address NF1 at its genetic core, not just managing symptoms but potentially correcting the underlying cause of tumor growth.

For the NF community, this research signals meaningful momentum toward more precise, long-term therapies. There is still a long road ahead, but the direction is promising and the progress is real.