Breaching the blood-brain barrier to deliver the medicine
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RNA-based drugs have the potential to change the standard of care for many diseases. So far, these drugs haven't been very useful in getting through to the well-protected brain to treat tumors or other maladies. A team of researchers has figured out a way: using ultrasound and RNA-loaded nanoparticles to get through the protective blood-brain barrier and deliver potent medicine to brain tumors.

Investigators have examined the delivery of siRNA, packaged in 50-nm cationic lipid-polymer hybrid nanoparticles (LPHs:siRNA), combined with microbubble-enhanced focused ultrasound (MB-FUS) in pediatric and adult preclinical brain tumor models.

Using single-cell image analysis, researchers show that MB-FUS in combination with LPHs:siRNA leads to more than 10-fold improvement in siRNA delivery into brain tumor microenvironments of the two models. MB-FUS delivery of Smoothened (SMO) targeting siRNAs reduces SMO protein production and markedly increases tumor cell death in the SMO-activated medulloblastoma model.

Moreover, the analysis reveals that MB-FUS and nanoparticle properties can be optimized to maximize delivery in the brain tumor microenvironment, thereby serving as a platform for developing next-generation tunable delivery systems for RNA-based therapy in brain tumors.

Science Advances
Source: https://advances.sciencemag.org/content/7/18/eabf7390
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