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Top Translational Researchers of 2014

Posted by Richard Tsai on Nov 12, 2015

Larry h Bernstein, MD, FCAP, Curator

LPBI

Nature recently released its list of the top 20 translational researchers for 2014, highlighting scientists whose work and patents facilitate new discoveries and advances in medicine and healthcare. The publication ranked biotech’s top translational researchers according to patenting activity, using information supplied by the patent analytics firm, IP Checkups. This research directly impacts the role of personalized medicine and will help to identify the future wave of biotherapeutics and diagnostic tools in our industry. In this blog we’ll review some of the research from five of these top researchers, and learn how their research is shaping our future.

Carlo M. Croce, MD
Carlo Croce is an Italian medical doctor who specializes in oncology, specifically the pathogenesis and genetic mechanisms of cancer. His research revealed genetic links to a variety of cancers, such as acute leukemia, T-Cell lymphoma, and Burkitt’s lymphoma. Additionally, his research has demonstrated that translocations and other chromosomal abnormalities can contribute to the initiation and progression of cancer.

Croce secured 29 patents in 2014. His 2010 patent US7670840B2, microRNA expression abnormalities of pancreatic, endocrine and acinar tumors, offers new ways for doctors to diagnose pancreatic cancer and aid physicians in creating a prognosis and treatment plan for patients with this disease. The research showed that particular microRNAs have the potential to exhibit either tumor-suppressive or oncogenic properties. The invention also provides methods of identifying anti-pancreatic cancer agents.

George A Calin, M.D., Ph.D.
George Calin is the first author of patent US7670840B2. Calin became a postdoctoral fellow in Dr. Carlo Croce’s laboratory at Kimmel Cancer Center in Philadelphia, Pennsylvania, in 2000. He currently serves as a professor in the Department of Experimental Therapeutics, Division of Cancer Medicine, and as co-director of The RNA Interference and non-coding RNA Center at the University of Texas MD Anderson Cancer Center, Houston, TX.

In his laboratory at MD Anderson Cancer Center, Dr. Calin focuses on the involvement of non-coding RNAs in human diseases with a particular interest in microRNAs in human cancers. He also researches genetic predisposition to cancer, identification of ncRNA biomarkers in body fluids, and is developing novel RNA-based therapies for the treatment of cancer.

Thomas H. Tuschl, PhD
Tuschl works as an investigator for Howard Hughes Medical Institute and professor at the Laboratory of RNA Molecular Biology, Rockefeller University. He and a team of researchers received patent US7772389B2 for their invention relating to isolated anti-microRNA molecules. MicroRNA is a small, non-coding RNA molecule found in plants, animals and certain viruses. MicroRNA participates in RNA-silencing and regulation of gene expression. Research revealed a connection between microRNA and lymphocytic leukemia and other types of cancer. Anti-microRNA inhibit the actions of endogenous microRNA.

At Rockefeller University, Dr. Tuschl works towards the development of novel therapeutic treatments for genetic diseases by investigating the different gene regulatory mechanisms triggered by doublestranded RNA and RNA-binding proteins.

Dr. Tuschl is widely recognized for his molecular characterizations of small interfering RNAs (siRNAs) that guide gene silencing. Tuschl was the first to show that these siRNAs are capable of “knocking down” gene expression, which prevents the genes from reaching their full cancer-causing potential. This work will help other researchers gain a better understanding of the roles siRNAs and other regulatory RNAs play in the onset of disease.

Feng Zhang, PhD
The invention named in patent US8697359 B1, CRISPR-Cas systems and methods for altering expression of gene products, provides for systems, methods, and compositions that alter the expression of target gene sequences and other gene products. Used by many bacteria as protection from viruses, plasmids and other foreign nucleic acids, CRISPR systems are adaptable immune mechanisms. Zhang uses the patented CRISPR-Cas system and other methodologies to study the role genetic and epigenetic mechanisms play in underlying diseases, with a specific focus on nervous system diseases. Zhang makes these tools widely available to other researchers in hopes of accelerating biomedical research.

Bert Vogelstein, MD
Dr. Vogelstein focuses on identifying and characterizing the genes that cause cancer, and creating new ways to apply this knowledge to the management of patients with cancer. He is a pioneer in the field of cancer genomics and have established the tumorigenesis model, which has been widely accepted and used by many researchers globally during the last two decades.

Many research and clinical applications rely on the accurate identification of predefined mutation that a scientist would expect to be present in a minor fraction of a cell population. Dr. Vogelstein’s patent US7824889B2, digital amplification, transforms the exponential, analog nature of the polymerase chain reaction into a linear, digital signal that researchers can use to identify those predefined mutations. The process created by this invention helps researchers create a reliable quantitative measurement of the proportion of variant sequences within a given DNA sample.

Interested in reading more? I encourage you to explore the research of the other 15 scientists featured in the Nature publication