Gene therapy, a revolution already underway
Long confined to basic research, gene therapy is now a clinical reality for a growing number of rare diseases, including certain forms of inherited blindness, spinal muscular atrophy, and blood disorders such as sickle cell disease.
The principle
The idea is simple to state, complex to achieve: correct or compensate for a faulty gene directly within the patient's cells, rather than only treating the symptoms it causes.
Increasingly precise tools
The arrival of genome-editing tools such as CRISPR-Cas9 has considerably accelerated the field, allowing a DNA sequence to be targeted with unprecedented precision. Several treatments based on this technology have since received regulatory approval in various countries.
Remaining challenges
- The cost of these treatments remains very high, raising questions of equitable access.
- Long-term follow-up of treated patients is still limited in duration.
- Not all genetic diseases lend themselves equally well to these approaches.
Gene therapy remains a fast-moving field, well worth following closely in the years ahead.