New Institute Within TSRI Focuses on Molecular Genetics
of Disease
Researchers Target Illnesses of Children and Afflictions
of People in Developing Nations
By Mika Ono
With the opening of a brand-new, 54,000-square-foot building
on the east side of The Scripps Research Institute (TSRI)
campus last month, the idea of the Institute for Childhood
and Neglected Diseases (ICND) became a reality. The ICND will
act as an umbrella group within TSRI for young scientists
working in areas relevant to childhood diseases (such as mental
disorders and cystic fibrosis) and diseases (such as malaria
and schistosomiasis) that primarily afflict people in developing
countries.
“The ICND represents a whole new effort for TSRI in
the molecular genetics of disease,” says Cell Biology
Professor Steve Kay, PhD, who has been organizing the initiative.
"The ICND will focus on combining genetic analysis with the
new tools of genomics under development by private industry.
The potential for synergy is phenomenal."
Six faculty, four new to TSRI, will open the ICND:
•Assistant Professor Colin Fletcher (AB, Dartmouth
College; PhD, The Rockefeller University), who investigates
the genetics of the locomotor disorders—work relevant
to childhood epilepsy and familial forms of migraines and
ataxia. Fletcher arrives at TSRI from the National Cancer
Institute's Frederick Cancer Research and Development Center.
• Associate Professor Mark Mayford (BS, PhD, University
of Wisconsin, Madison), whose work focuses on the role of
single genes and specific cells in the formation of memories—work
relevant to mental retardation and learning disabilities.
Mayford most recently headed a lab at the University of California,
San Diego.
•Assistant Professor Ardem Patapoutian (BA, University
of California, Los Angeles, PhD, California Institute of Technology),
who works on pain and pain perception—a field relevant
to most diseases. Patapoutian arrives at TSRI after a postdoctoral
fellowship at the University of California, San Francisco.
• Assistant Professor Elizabeth Winzeler (BA, Lewis and
Clark College; MS, Oregon State University; PhD, Stanford
University), who is finding ways to select promising proteins
to target in the malaria parasite. Winzeler comes to TSRI
after a postdoctoral fellowship at Stanford University.
• Professor William Balch (BSc, Portland State University;
PhD, University of Illinois), who works transport through
the secretory pathway with disease models of cystic fibrosis
and hereditary emphysema. Balch, who has been at TSRI since
1990, will continue to run his laboratory outside the ICND
building.
•Professor Steve Kay (BSc, PhD, University of Bristol,
United Kingdom), who studies circadian rhythms—a field
relevant to sleep disorders, depression, and bipolar disorder.
Kay has been at TSRI since 1996.
The concept of the ICND grew out of conversations in 1996
and 1997 among TSRI President Richard Lerner, John Moores,
who was interested in supporting research on illnesses affecting
people in developing countries, and the brothers Bernard and
Marc Chase, who were interested in supporting research on
childhood diseases. John and Rebecca Moores, Bill Bauce, and
other automobile enthusiasts donated a number of vintage automobiles,
which were auctioned to support the initiative. The Moores
went on to contribute a valuable coin collection, as well
as pledging $5 million over five years. The Chase brothers
also made a donation to the effort.
In 1999, Lerner, Kay, and Professor Peter Schultz met to
begin detailing the scientific aspects of the ICND, which
was conceived in three phases:
• Phase I, now essentially complete, puts in place investigators
who explore the physiologic functions of genes;
• Phase II, currently underway, focuses on recruiting
scientists with experience working with direct models of disease;
• Phase III, which is to launch in a year or more, brings
in genomics-based human geneticists interested in working
on both basic and clinical research.
Throughout all phases, collaborations with the The Genomics
Institute of the Novartis Research Foundation (GNF) and other
private institutions will be set up as needed.
"The ICND is a far-reaching project that is immensely appropriate
for an institution of TSRI’s stature," comments Kay.
"With the ICND, TSRI is making a significant commitment to
translating the letters of the Human Genome Project into prose
that will benefit mankind."
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Assistant Professor Elizabeth Winzeler,
whose research focuses on finding ways to select promising
proteins to target in the malaria parasite, is one of six
faculty to open the ICND.
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