(Funded by the National Institutes of Health)
A child diagnosed with a rare genetic disorder has been successfully treated with a customized CRISPR gene editing therapy by a team of researchers at Children’s Hospital of Philadelphia and Penn Medicine. The researchers targeted the infant’s specific variant of a gene that codes for an enzyme in the liver that converts ammonia to urea (which is later excreted in urine). The researchers designed and manufactured a gene-editing therapy delivered via lipid nanoparticles to the liver in order to correct the infant’s faulty enzyme. As of April, the infant had received three doses of the therapy with no serious side effects.

(Funded by the National Institutes of Health)
Northwestern Medicine investigators have developed first-of-its-kind eyedrops that use synthetic nanoparticles to help the eye regenerate cells that have been damaged by exposure to mustard gas, which has been historically used during wartime. These nanoparticles were designed to mimic some properties of high-density lipoproteins, which are naturally found in the bloodstream and can help the body regulate inflammation. The investigators tested the eyedrops on mice and discovered that the eyedrops not only reduced inflammation in the eyes of the mice but also restored cells that are responsible for maintaining and regenerating the cornea’s epithelium.

(Funded by the U.S. National Science Foundation and the National Institutes of Health)
Researchers at the University of Pittsburgh have developed silk iron microparticles and magnetic iron oxide nanoparticles and then chemically bonded the silk microparticles with the nanoparticles. The microparticles were designed to deliver drugs to sites in the body, and the drugs were towed by the microparticles like a trailer is towed by a car. “You can think of it like towing cargo – we created the [micro]particles to carry drugs, and the nanoparticles are the tow hook,” said Mostafa Bedewy, associate professor at the University of Pittsburgh. Now that the researchers have found a way to magnetically guide the silk microparticles with the nanoparticles, the next step will be to load them with therapeutic cargo. This research opens the door to a wide range of future applications – from targeted cancer therapies to regenerative treatments for cardiovascular disease.

(Funded by the National Institutes of Health)
Historically, the vast majority of pharmaceutical drugs have been designed down to the atomic level, so that the specific location of each atom within the drug molecule determines how well it works and how safe it is. Now, Northwestern University and Mass General Brigham scientists argue that this precise structural control should be applied to optimize new nanomedicines. The scientists cite three examples of trailblazing structural nanomedicines: spherical nucleic acids (globular form of DNA that can easily enter cells and bind to targets), chemoflares (smart nanostructures that release chemotherapeutic drugs in response to cues in cancer cells) and megamolecules (precisely assembled protein structures that mimic antibodies).

(Funded by the U.S. Department of Defense, the U.S. National science Foundation and the National Institutes of Health)
In 2023, researchers at Caltech developed a smart bandage that can provide real-time data about chronic wounds and accelerate the healing process by applying medication or electrical fields to stimulate tissue growth. Now, the researchers have shown that an improved version of their bandage can continually sample fluid, which the body sends to wound sites as part of the inflammatory response. The bandage is composed of a flexible, biocompatible polymer strip that integrates a nanoengineered biomarker sensor array, which is disposable for hygiene and single-use applications. The system also includes a reusable printed circuit board that handles signal processing and wireless data transmission to a user interface, such as a smartphone.