Major Scientific Achievements
Researchers at TSRI have been responsible for a number
of seminal studies into the basic biology of molecules and cells that
are at the root of all life. Among the more significant advances of
the past decade are the following:
Developed and successfully tested the anti-leukemia drug 2-Chlorodeoxyadenosine
(2-CdA, trade name: Leustatin) Demonstrated that rheumatoid factor
is a product of an antibody gene that has maintained its "germlike"
arrangement, explaining why so many rheumatoid factors are so similar.
Developed a new and efficient method to produce disease-fighting proteins,
called monoclonal antibodies, an advance that is expected too have
a profound impact on pharmaceuticals and the treatment of disease.
Determined the complete, three-dimensional, atomic structure of the
poliovirus.
Pioneered the concept that small, synthetic peptides -- the building
blocks of protein structures -- could replace larger peptide chains
of bacteria and viruses for the purpose of making vaccines.
Cloned the gene for the enzyme that is deficient in people with Gaucher's
disease and developed a method to predict the severity of the disease,
a potentially fatal inherited disorder.
Purified the antihemophilic Factor VIII, a coagulation protein lacking
in people with hemophilia A. Monoclate, the purified concentrate of
Factor VIII, enables hemophiliacs to receive blood plasma that is
free of contamination with viruses from blood donors.
Synthesized surfactant, a lung material that keeps air sacs open and
prevents respiratory distress syndrome, a major killer of premature
babies and adults.
Pioneered the development of catalytic antibodies -- antibodies designed
to function as enzymes in catalyzing specified chemical reactions
-- opening new possibilities for protein synthesis and the rational
design of new drugs.
Mapped the prohormone for somatostatin in the brain and associated
it with the primary neuropathic signs of Alzheimer's disease.
Discovered a cell receptor for allergy-inducing IgE antibodies on
lymphocytes, a finding that redirected research on the control of
allergic diseases.
Designed and synthesized a new class of molecules, known as enediynes,
that represent some of the most potent anti-cancer agents ever tested
and demonstrate unusual selectivity in their ability to destroy cancer
cells while leaving healthy cells intact.
Solved the three-dimensional structure of the enzyme superoxide dismutase
(SOD), thereby establishing a direct link between mutations in the
gene for SOD that lead to an unstable, less active enzyme and can
cause amyotrophic lateral sclerosis (ALS), or Lou Gehrig's disease.
Completed the total chemical synthesis of the anti-cancer drug, taxol,
approved by the Food and Drug Administration for the treatment of
ovarian cancer.
Solved the three-dimensional structure of the T cell receptor, a key component of the immune system. Understanding its structure and function may enable scientists to enhance the effectiveness of the immune system through the development of new, highly-targeted therapeutics.
Elucidated the x-ray crystal structure of a membrane transporter protein, a finding that could be useful for improving cancer therapy and fighting antibiotic resistant bacteria.
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