International Journal of Nanomedicine

Nanoparticle Applications in Brain Disorders: Gene Therapy and Enzyme Replacement Therapy

Brain disorders are one of the most challenging targets for novel therapies due to their location and the presence of numerous barriers that limit the efficacy of treatments. Nanotechnologies, such as nanoparticles and nanomedicines, are promising tools to overcome the limitations of traditional treatments thanks to their ability to protect sensitive molecules from degradation and reduce off-target drug toxicity in vivo by using targeting ligands to cross the blood brain barrier, selectively accumulate at the target site, and release their payload in the brain parenchyma. In fact, the use of complex molecules such as DNA, RNA, siRNA, mRNA, enzymes, or proteins has shown promising results in hard-to-treat pathologies such as neurodegenerative disorders and brain cancer. This Article Collection collects the most recent innovations in the field of nanoparticle-based gene and enzyme replacement therapy for brain diseases.