Leonard H. Rome

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Leonard H. Rome is a cell biologist and biochemist who has been a faculty member of the David Geffen School of Medicine at UCLA since he joined the Department of Biological Chemistry in 1979. He became a full professor in 1988 and has also served as the Senior Associate Dean for Research in the School of Medicine from 1997 to 2012. He is the Associate Director of the California NanoSystems Institute (CNSI) since 2004, and was Interim Director from 2007-2009. In addition, he served from 2001 to 2005 as UCLA Associate Vice Chancellor for Research for the Life and Health Sciences.

Rome earned his B.S. in Chemistry and M.S. and Ph.D. in Biological Chemistry at the University of Michigan with William E.M. Lands. He was a postdoctoral fellow at the National Institutes of Health, where he worked with Elizabeth Neufeld on lysosome biogenesis and lysosomal storage diseases. His laboratory research at UCLA centers on a novel cellular organelle called a "vault" which he and a former postdoc, Nancy Kedersha, discovered in 1986.[1] Rome is the past chair of the Association of American Medical Colleges (AAMC) Group on Research Advancement and Development (GRAND).


Rome’s lab is using molecular engineering of a naturally occurring cellular structure called a vault to develop a flexible, targetable nano-scale capsule. Vaults are abundant cellular particles of unknown function found in nearly all eukaryotes (cells containing a nucleus). In 1986, Rome and his then Postdoctoral Associate Nancy Kedersha were the first investigators to isolate and describe the vault particle.[1] Cryo-electron microscopy combined with single particle reconstruction has provided overall dimensions of the vault at 42 x 75 nanometers (a nanometer is a millionth of a meter). These measurements indicate that the vault is larger in mass and size than many viruses. The overall structure of the intact vault is like a hollow barrel with two protruding caps and an indented waist with a very thin shell surrounding an internal cavity large enough to encompass several hundred proteins. Thus, the vault particle is a nanocapsule with incredible potential for compound encapsulation, protection, and delivery. Using a well-characterized insect virus into which a cloned gene can be easily inserted, it is possible to produce large quantities of a given protein in cultured insect cells.

The Rome lab has collaborated with a number of groups to use the baculovirus system to produce large quantities of vaults. When the major vault protein (MVP) is expressed in insect cells, vault particles are assembled on polyribosomes in the cytoplasm.[2] By using molecular genetic techniques to modify the gene encoding the major vault protein, vault particles have been produced with chemically active peptides attached to their sequence. These modified proteins are incorporated into the inside of the vault particle without altering its basic structure.

In 2003 Rome co-founded a company called Vault Pharma Inc. (http://www.vaultpharma.com) to move the first vault therapeutic into a phase I clinical trial. This vault is packaged with a chemokine and will be used to activate the immune system to attack lung cancer.[3][4] Vault Pharma is currently working with Protein Sciences Corp. to develop the GLP/cGMP manufacture of this vault-based therapeutic


  1. ^ a b "Isolation and characterization of a novel ribonucleoprotein particle: large structures contain a single species of small RNA". The Journal of Cell Biology 103 (3): 699–709. 1986-09-01. ISSN 0021-9525. PMC 2114306. PMID 2943744. 
  2. ^ Mrazek, Jan; Toso, Daniel; Ryazantsev, Sergey; Zhang, Xing; Zhou, Z. Hong; Fernandez, Beatriz Campo; Kickhoefer, Valerie A.; Rome, Leonard H. (2014-11-25). "Polyribosomes are molecular 3D nanoprinters that orchestrate the assembly of vault particles". ACS Nano 8 (11): 11552–11559. doi:10.1021/nn504778h. ISSN 1936-086X. PMC 4245718. PMID 25354757. 
  3. ^ Sharma, Sherven; Zhu, Li; Srivastava, Minu K.; Harris-White, Marni; Huang, Min; Lee, Jay M.; Rosen, Fran; Lee, Gina; Wang, Gerald (2013-01-01). "CCL21 Chemokine Therapy for Lung Cancer". International Trends in Immunity 1 (1): 10–15. ISSN 2326-3121. PMC 4175527. PMID 25264541. 
  4. ^ Kar, Upendra K.; Srivastava, Minu K.; Andersson, Asa; Baratelli, Felicita; Huang, Min; Kickhoefer, Valerie A.; Dubinett, Steven M.; Rome, Leonard H.; Sharma, Sherven (2011-01-01). "Novel CCL21-vault nanocapsule intratumoral delivery inhibits lung cancer growth". PloS One 6 (5): e18758. doi:10.1371/journal.pone.0018758. ISSN 1932-6203. PMC 3086906. PMID 21559281. 

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