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Saturday,
April 29
3:15pm–5:15pm
2763—New
Approaches in Stem Cell Technology
PAS
Educational Workshop
Room
Golden Gate Hall A1, SF Marriott
Leader: Kathleen Sakamoto, Los Angeles, CA; Co-leaders: Ed
Horwitz, Harley Kornblum, and Punam Malik
Recent
advances in molecular and cellular techniques have provided
new approaches to studying the role of gene function in a
variety of cell types, including stem cells. It is critical
for pediatricians and pediatric subspecialists to understand
the basis and use of these emerging technologies. This
workshop will provide an overview of new methods that are
currently being used to study stem cells. The topics include
RNA interference (RNAi), isolation and purification of stem
cells, gene expression profiling, and gene therapy. Upon
completion of this workshop, participants will be able to
describe (a) uses of RNAi in stem cells, (b) approaches to
isolate and purify mesenchymal stem cells, (c)
characterization of neural stem cells using expression
profiling, and (d) applications of gene therapy and stem
cells.
3:15pm–5:15pm
2767—How
Do I Incorporate Proteomics Technology Into My Research?
PAS
Educational Workshop
Room
Golden Gate Hall A3, SF Marriott
Leader: Anne Murphy, Johns Hopkins University School of
Medicine, Baltimore, MD; and Co-leader: James Schilling,
Stanford University School of Medicine, Stanford, CA
Laboratory
scientists, physician-scientists and trainees as well as
clinical scientists interested in disease biomarkers.
Proteomics
is a technology driven field. Many investigators not currently
employing these technologies would love to incorporate them
into their research. This workshop will address some
strategies for investigators contemplating the use of the gel,
mass-spectrometry, and array based approaches. Discussions of
strategies to choose technologies to match the question,
strategies for assessing post-translational modifications, and
how to best work with a core facility will be addressed. The
workshop attendees should emerge with an understanding of the
strengths and weaknesses of various broad-based proteomic
technologies. This will permit them to match techniques with
an experimental question and to maximize interactions with
core facilities already in place within their institutions.
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Approaches
To Analyze The Phosphoproteome
Anne
M. Murphy, Johns Hopkins University School of Medicine,
Baltimore, MD
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Overview
Of Approaches
David
R. Graham, Johns Hopkins University-Bayview Campus,
Baltimore, MD
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Sample
preparation
James
Malone, Washington University School of Medicine, St.
Louis, MO
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Mass
Tagging Approaches to Biomarker Discovery
K.W.
Michael Siu, Centre for Research in Mass Spectrometry,
York University, Toronto, Ontario Canada
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Statistics
and Data Analysis
Robert
Tibshirani, Stanford University, Stanford, CA
Supported in part by an unrestricted educational grant from
Applied Biosystems and GE Healthcare
Sunday,
April 30
4:15pm–5:45pm
3810—RNA
Interference, Technological Development of siRNAs and
Potential Treatments for Childhood Diseases
PAS
State of the Art Plenary
Room
3016-3018, Moscone West
Chair: R. Alan B. Ezekowitz, Harvard Medical School,
Massachusetts General Hospital, Boston, MA
Target
Audience: Basic scientists studying a broad range of childhood
diseases, translational scientists of all disciplines studying
clinical implications of basic science research, clinical
scientists studying childhood and other diseases in need of
improved therapies and clinicians interested in cutting-edge
science and its medical implications.
RNA
interference is a recently discovered, naturally occurring
intracellular process that regulates gene expression through
the silencing of specific mRNAs. Methods of harnessing this
natural pathway are being developed that allow the catalytic
degradation of targeted mRNAs using specifically designed
complementary small inhibitory RNAs (siRNA). siRNAs are being
chemically modified to acquire drug-like properties. Numerous
recent high-profile publications have provided proofs of
concept that RNA interference may be useful therapeutically.
Much of the design of these siRNAs can be accomplished
bioinformatically, thus potentially expediting drug discovery
and opening new avenues of therapy for many childhood diseases
including uncommon pediatric and orphan diseases. A discussion
of the science behind RNA interference will be followed by a
presentation of the potential practical issues in applying
this technology to disease. The program then describes two
therapeutic programs currently under way with applications to
pediatric diseases. A question-and-answer time will follow
each discussion.
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The
Science of RNA Interference
John
J. Rossi, Beckman Research Institute of City of Hope,
Duarte, CA
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RNA
Interference and Its Potential Applications for
Controlling Disease
Judy
Lieberman, CBR Institute for Biomedical Research and
Harvard Medical School, Boston, MA
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Silencing
the VEGF Pathway with siRNAs and the Potential Application
to Retinopathy of Prematurity
Pamela
Pavco, Sirna Therapeutics, Boulder, CO
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siRNA
as Therapy for Respiratory Syncytial Virus
John
P. DeVincenzo, University of Tennessee School of Medicine,
Memphis, TN
4:15pm–6:15pm
3850—Human
Milk and Breastfeeding
PAS
Poster Symposium
Room
3001, Moscone West
Chairs: Sheela R. Geraghty and Ardythe L. Morrow
Monday,
May 1
8:15am–10:15am
4180A—Chronic
Organ Damage in Sickle Cell Disease: Diagnosis, Prevention and
Treatment
ASPHO
Symposium
Room
3016-3018, Moscone West
Chair: Russell E. Ware, St. Jude Children’s Hospital,
Memphis, TN
Chronic
organ damage is increasingly recognized as an important source
of morbidity and mortality for young persons with sickle cell
disease. This symposium will include descriptions of several
prospective multicenter clinical trials that focus on the
diagnosis, treatment and prevention of organ damage in this
common hematological disorder.
After
attending this session, it is expected that the learner will
be able to:
1.
Discuss ongoing prospective multicenter randomized clinical
trials that focus on brain damage in pediatric patients with
sickle cell anemia.
2. Describe prospective clinical trials that focus on spleen
and kidney damage in very young patients with sickle cell
anemia.
3. Describe new imaging modalities for transfusional iron
overload in the liver and heart, as well as studies using new
oral iron chelating agents for its therapeutic management.
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Introduction
and Overview
Russell
E. Ware, St. Jude Children's Research Hospital, Memphis,
TN
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Brain
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Silent
Infarct Transfusion (SIT) Trial
Michael
R. DeBaun, Washington University School of Medicine,
St. Louis, MO
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Stroke
with Transfusions Changing to Hydroxyurea (SWiTCH)
Trial
Russell
E. Ware, St. Jude Children's Research Hospital,
Memphis, TN
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Spleen
and Kidney
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Splenic
Function in Young Children: Lessons from BABY HUG
Zora
R. Rogers, The University of Texas Southwestern
Medical Center, Dallas, TX
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Renal
Function in Young Children: BABY HUG, Toddler HUG
Sherri
A. Zimmerman, Duke University Medical Center, Durham,
NC
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Liver
and Heart
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New
Imaging Techniques for Transfusional Iron Overload
Thomas
D. Coates, Children's Hospital of Los Angeles, Los
Angeles, CA
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Oral
Iron Chelators for the Management of Transfusional
Iron Overload
Alan
R. Cohen, Children's Hospital of Philadelphia,
Philadelphia, PA
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Questions
and Panel Discussion
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