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Research Highlights

Research Highlights By Year (select year to download pdf)
2008 | 2009 | 2010 | 2011
Clinical trials began for a drug to treat a fatal childhood disease, Spinal Muscular Atrophy (SMA).  The drug is the result of Adrian Krainer’s work on the mechanism-based design of antisense compounds that he found could correct splicing defects in target genes. The first such compound, ISIS-SMNRx, developed in close collaboration with Isis Pharmaceuticals, efficiently corrects defective splicing of SMN2 in preclinical models, and is now being tested in pediatric patients with spinal muscular atrophy (SMA), a severe motor-neuron disease for which no treatment currently exists. In March 2013, Isis reported positive data from a single-dose, open-label Phase 1 clinical study evaluating ISIS-SMNRx in children with SMA. Similar strategies may be applied for other splicing-related diseases. 
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Chris Vakoc used an unconventional approach to cancer drug discovery to identify a new potential treatment for acute myeloid leukemia (AML) which is being developed for therapeutic use for cancer patients by Tensha Therapeutics.  In collaboration with 5 other institutions, Vakoc pinpointed a protein called Brd4 as a novel drug target for AML, an aggressive blood cancer that is currently incurable in 70% of patients. Using a drug compound that inhibits the activity of Brd4, the scientists were able to suppress the disease in experimental models. This work represents an early success in CSHL's Cancer Therapeutics Initiative, which aims to rapidly identify new therapeutic targets and validate them in mouse models that closely mimic the behavior of specific human cancers. In addition to offering insights into cancers molecular mechanisms, this pre-clinical testing of drug candidates within a living animal allows researchers to evaluate cancer's response to new therapies and glean information that can be used to modify treatment strategies and increase the success rate of drugs that eventually enter the clinic.
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Zach Lippman and colleagues in Israel identify a single gene that dramatically boosts yield and sweetness in tomato hybrids.The yield-boosting power of this gene, which controls when plants make flowers, works in different varieties of tomato, and crucially, across a range of environmental conditions. The discovery has the potential to significantly change agricultural practices designed to get the most yield from flowering crops.
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Elizabeth Murchison, Gregory Hannon and colleagues discover a genetic marker for the facial cancer that is decimating Australia’s Tasmanian devil population.  The finding, which suggests that the cancers probably originated in Schwann cells, a type of tissue that cushions and protects nerve fibers, indicates new avenues for research into treatments and vaccines to save the endangered marsupials.
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A multi-institutional effort co-led by Doreen Ware, Richard McCombie and Robert Martienssen completes a reference genome for maize, one of the world’s most important crops.  The genome is a crucial starting point for scientists to understand the genetic differences between individual lines of maize and the function of every gene, especially those that affect important agricultural traits such as the ability to tolerate drought or dampness, size of seeds, etc.
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Scott Lowe’s laboratory develops new mouse models for human acute myeloid leukemia (AML) that accurately predict human response to chemotherapy.
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Yi Zhong identifies a protein that determines how long resting intervals between learning sessions need to last so that long-term memories can form.

A collaboration between five different research groups integrates genomic studies on human liver cancers with RNAi-based screening in a mouse model of the disease to identify 13 new tumor suppressor genes.

Adrian Krainer and colleagues induce cells to replenish a protein that, when deficient, leads to damage in growing nerve cells and muscles causing spinal muscular atrophy (SMA). These results hold out hope for one day successfully treating this often-fatal disease. 

Jonathan Sebat and Michael Wigler find a genetic distinction between sporadic and heritable forms of autism. 

Gregory Hannon, Scott Lowe, Robert Lucito, Scott Powers and Michael Wigler identify two genes on chromosome 11 that are likely to have a role in liver cancer. 

James Hicks shows that individual breast cancers can be diagnosed at the DNA level through a technique developed by Michael Wigler, which will allow physicians to make more accurate prognoses for patients. 

Richard McCombie and other CSHL scientists are part of an international team that deciphers the genome of humanity’s most important food source, rice. 

Gregory Hannon, Scott Lowe and Scott Powers discover microRNAs as new targets for improved diagnosis and treatment of lymphoma, colon cancer and other tumors. 

Leemor Joshua-Tor uncovers valuable clues for rational anti-malarial drug design and vaccine development by determining the structure of a protein that enables malaria parasites to invade red blood cells.

Michael Wigler and colleagues detect a surprising degree of large-scale variation in the copy number of genes in otherwise normal human genomes, by using a technique that they initially developed to detect differences between normal cells and cancer cells.

Gregory Hannon creates the first full-scale library of human RNAi clones. The library enables users in industry and academia to rapidly identify and validate target genes involved in a wide variety of diseases. 

Scott Lowe establishes a promising combination therapy for treating many cancers that do not respond to traditional chemotherapy. 

Timothy Tully and Joshua Dubnau identify a large group of candidate memory genes that are potential targets for the development of therapies for treating human memory disorders. 

Bruce Stillman discovers many important cellular proteins that duplicate chromosomal DNA before cell division. 

Edward Harlow and his colleagues establish a crucial functional link between the two general classes of cancer-causing genes (oncogenes and tumor-suppressor genes). 

Michael Wigler discovers the first cancer-causing gene in a human tumor. 

Joseph Sambrook, Phillip Sharp and William Sugden develop a technique for separating and visualizing DNA fragments that, even today, underpins all of molecular biology. 

Milislav Demerec greatly increases wartime penicillin production by isolating a high-yielding strain of the filamentous fungus Penicillium chrysogenum

George Shull forms the basis of modern agricultural genetics by crossing inbred lines of corn, thus discovering heterosis, or "hybrid vigor.

Carol Greider is named one of the first CSHL Fellows and continues her research on telomeres, which protect the greider_labends of chromosomes from degradation during cell division. She wins the Nobel Prize in 2009 with Elizabeth Blackburn and Jack Szostak.

Richard Roberts discovers the phenomenon of RNA splicing richards_nobel
and thus reveals that the same DNA region can code for many different proteins. He wins the Nobel Prize in 1993 with Phillip Sharp.

James Watson gives the first public description of the newly discovered DNA double helix at the CSHL Symposium.watson_nobel The discovery influences virtually all subsequent biological and medical research. Nine years later, Watson wins the Nobel Prize with Francis
Crick and Maurice Wilkins.

Experiments by Alfred
hershey_nobelHershey show that DNA, not protein, is the genetic material of bacterial viruses. He wins the Nobel Prize in 1969 for this and other work. 

Barbara McClintock discovers mobile genetic elements mcclintock_nobel
(“jumping genes”), a finding that has had profound importance for understanding chromosome structure and function. She wins the Nobel Prize in 1983.