![]() ![]() This could eventually become an effective therapy for the lungs of patients, regardless of the mutation type. Yet this study, Suk notes, has demonstrated that delivering normal copies of CF-related genes or corrective genes via the mucus-penetrating DNA-loaded nanoparticles could mediate production of normal, “functional” proteins long term. A couple of recently approved drugs designed to target the underlying cause of CF require daily treatment for the entire lifetime and can benefit only a subpopulation of patients with specific types of mutations. Most of the existing drugs for CF help clear infections but do not solve the disease’s underlying problems. This pathogenic process not only worsens patients’ quality of life but it also makes the airway mucus harder to overcome by inhaled therapeutic nanoparticles. In patients with CF, for instance, they experience a buildup of excess mucus caused by impaired ciliary beating, resulting in an ideal breeding ground for chronic bacterial infection and inflammation. Suk says their work with nanoparticles grew out of failed efforts to deliver treatments to people with lung diseases. What’s more, because a single dose might theoretically last for several months, patients would experience fewer side effects common to drugs that must be taken regularly over long stretches of time. His team’s experiments with human airway mucus and small animals, Suk adds, were designed as a proof-of-concept study demonstrating that placing corrective or replacement genes or drugs inside a man-made biodegradable nanoparticle “wrapper” that patients inhale could penetrate the mucus barrier and one day be used to treat serious lung disorders. ![]() Genes or drugs could be placed inside the biodegradable nanoparticle wrapper ![]() Unfortunately, Suk notes, this essential protective mechanism also prevents many inhaled therapeutics, including gene-based medicine, from reaching their target. In healthy lungs, inhaled matter is typically trapped in airway mucus and subsequently swept away from the lungs via beating activities of cilia, or small, hairlike strands, to the stomach to be eventually degraded. The mucus barrier protects foreign materials and bacteria from entering and/or infecting lungs. ![]()
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