Gene Therapy May Finally Reach The Right Cells

InnovationHealthcareGene Therapy May Finally Reach The Right CellsByWilliam A. Haseltine,Contributor.Forbes contributors publish independent expert analyses and insights. William A. Haseltine, Ph.D., covers genomics and regenerative medicineFollow AuthorMay 12, 2026, 10:55am EDTGene-silencing therapies may finally be able to reach hard-to-target organs like the brain and kidney by hitching a ride on the body’s own delivery vesicles, which naturally home to specific cell types and can deliver RNA drugs at far lower, safer doses.ACCESS Health InternationalThe promise of turning off the genetic drivers of devastating diseases is becoming an attainable reality, assuming these therapies can effectively reach the targeted cells. In controlled lab settings, delivering gene-silencing drugs into cells is relatively straightforward. Inside the human body is another story.The drugs must travel through the bloodstream, evade immune defenses and enter only the intended cell types while bypassing all others. Most do not; instead, they accumulate in the liver, leaving patients whose diseases originate in other organs without effective options. A new method offers a compelling way forward: harnessing the body’s own delivery system.The Delivery ProblemGene-silencing therapies use short strands of RNA to intercept and destroy the molecular instructions that produce harmful proteins. Regulatory agencies have already approved several such drugs, but nearly all succeed only in the liver. This occurs because the liver is easily accessible. It readily absorbs particles from the bloodstream, making it the default destination for most injected therapies.Reaching other organs proves far more difficult. Often higher doses are needed. These increase the risk of toxicity and immune reactions. The brain poses an even greater challenge because a tightly regulated barrier prevents most circulating molecules from entering. Therefore, a gap persists between what works in vitro and what works in vivo...