What are we going to see from gene and cell therapy in the near future?

According to Bulaklak and Gersbach (2020), advances in gene therapy will control the immune system response since about 50% of patients are excluded from treatment due to pre-existing immunity to the viral capsids. One example is the engineering of modified adeno-associated viruses (AAV) capsids that evade pre-existing neutralizing antibodies and methods for temporary clearing of antibodies from the circulation. Immunosuppression techniques can also help to avoid adaptive immunity to the vector. 

Another advancement is the use of non-viral for gene delivery. For example, Nanoparticles have the potential to circumvent detection by the immune system that limits viral delivery. These formulations can offer better tissue targeting, which is essential for the success of in vivo gene transfer outside of the retina and liver. 

Finally, researchers will likely turn their attention to non-coding DNA non-coding DNA that harbors epigenetic regulators responsible for >90% of common disease susceptibility, uncovering new therapeutic targets. 

References

Bulaklak, K., Gersbach, C.A. The once and future gene therapy. Nat Commun 11, 5820 (2020). https://doi.org/10.1038/s41467-020-19505-2