Nanostructures created by UCLA scientists could make gene therapies safer, faster and more affordable

UCLA scientists have developed a new method that utilizes microscopic splinter-like structures called “nanospears” for the targeted delivery of biomolecules such as genes straight to patient cells. These magnetically guided nanostructures could enable gene therapies that are safer, faster and more cost-effective.The research waspublished in the journal ACS Nano by senior author Paul Weiss, UC Presidential Chair and distinguished professor of chemistry and biochemistry, materials science and engineering, and member of theEli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA.Gene therapy, the process of adding or replacing missing or defective genes in patient cells, has shown great promise as a treatment for a host of diseases, including hemophilia, muscular dystrophy, immune deficiencies and certain types of cancer.Current gene therapy approaches rely on modified viruses, external electrical fields or harsh chemicals to penetrate cell membranes and deliver genes straight to patient cells. Each of these methods has its own shortcomings; they can be costly, inefficient or cause undesirable stress and toxicity to cells.To overcome these barriers, Weiss and Dr. Steven Jonas, a clinical fellow in the UCLA Broad Stem Cell Research Center Training Program, led a research team that designed nanospears composed of silicon, nickel and gold. These nanospears are biodegradable, can be mass-produced inexpensively and efficiently, and, because of their inf...
Source: UCLA Newsroom: Health Sciences - Category: Universities & Medical Training Source Type: news