According to a story from EurekAlert!, scientists from UCLA have developed a new method of delivery for gene therapies called nanospears. These structures could allow for more accurate and precise delivery of gene therapies, making them less costly, faster-acting, and safer for the patient.
The first description of nanospears was first published in the scientific journal ACS Nano. Gene therapy has been a common treatment method approach for a great variety of diseases that are affected by genetics, including a great many rare diseases. Some diseases for which gene therapies have been developed include certain types of cancer, hemophilia, immune deficiencies, and muscular dystrophy.
One of the greatest challenges of gene therapy medicine has been the development of delivery methods. A variety of useful solutions to this problem have been created, including the use of electrical fields, modified viruses, and chemicals. These methods have been used on many occasions in order to get the job done, but they all carry some significant disadvantages, such as significant cost, side effects and toxicity for the patient, and dubious efficiency.
Researchers the UCLA saw a need for a highly precise and inexpensive new delivery method that would not carry a great risk of side effects. This method is the nanospear, comprised of nickel, silicon, and gold. The tips of the microscopic nanspears are 5,000 times smaller that the diameter of a human hair, so they have little risk of disrupting normal cellular functions. They can also be mass produced for chump change and are biodegradable.
The common solution for precision gene therapy delivery has been the use of chemicals in order to drive the medicine into the cell, but the nickel coating eliminates this problem. Nickel is essential for the effectiveness of the nanospear because it is a magnetic metal. This allows for a magnet to be used in order to guide the nanospears on their journey to the patient’s cells.
The scientists from UCLA tested the effectiveness of the nanospear and achieved an 80 percent delivery success rate, and of that percentage, 90 percent of the cells that received the delivery protein survived. This rate of accuracy is demonstrably superior in comparison to currently available delivery vehicles. Clearly, the nanospear has the potential to dramatically improve the efficiency and effectiveness of gene therapy.