UCLA scientists store materials in cells' natural vaults

Method may offer safer way to target drugs to living cells -


BACKGROUND: In the realm of nanotechnology, or study of the tiny, scientists often aim to safely deliver and leave
material in the human body without causing harm. A big challenge is how to design a package for this biomaterial that will be
compatible with living cells and will not provoke an immune reaction. Previous efforts have relied upon viruses or artificial
chemicals to house and deliver drugs or other substances to targeted cells.


RESEARCH: Scientists at the David Geffen School of Medicine at UCLA have exploited thousands of tiny capsules in
cells to store vast amounts of biomaterial. Dubbed vaults - for their high arches reminiscent of cathedral ceilings - these
naturally occurring capsules may prove less likely to elicit an immune response than foreign carriers like viruses.


IMPACT: The UCLA discovery will enable many potential applications, including:


-- Therapeutic delivery, such as homing cancer drugs directly to a tumor cell without harming healthy tissue


-- Enzyme delivery to replace missing or defective enzymes, such as those that cause Tay Sachs disease


-- DNA delivery to correct genetic mutations


-- Timed release of drugs, enzymes and DNA


-- Extracting and imprisoning cellular toxins into the vault


-- Stabilizing proteins in vaults to increase their lifespans


AUTHORS: Leonard Rome, professor of biological chemistry and senior associate dean of research; Bruce Dunn, professor
of material science and engineering; Jeffrey Zink, professor of chemistry and biochemistry and Valerie Kickhoefer, first
author and associate research biochemist.


JOURNAL: The Proceedings of the
National Academy of Sciences


FUNDING: The National Science Foundation supported the research.


(NOTE: Full manuscript and color images of vaults available upon request.)


Contact: Elaine Schmidt

eschmidtmednet.ucla

310-794-2272

University of California - Los Angeles

ucla