Gene Regulation and Inheritance
The Gene Regulation and Inheritance Program focuses on revealing basic mechanisms governing the regulation of gene expression and cell inheritance at the DNA, RNA, and protein levels, and on discovering how these mechanisms are perturbed to influence the initiation and/or progression of cancer. A major strength of the Program is the innovative science that is yielding novel insights into non-coding RNA species, RNA splicing, chromatin biology, and cell-cycle control. Alterations in these processes are critical features of the transformed phenotype. Although fundamental research is the central to this Program, many discoveries are being translated toward the clinic, due in part to the strong strategic alliance with clinical partners.
Program Co-leaders
David Spector, Ph.D.
Leemor Joshua-Tor, Ph.D.
Members of the Gene Regulation and Inheritance Program share an interest in uncovering the mechanisms governing inheritance of cell state as well as mechanisms of transcriptional and post-transcriptional regulation, and on understanding how those mechanisms are altered in cancer cells. The Program has three main focus areas: (1) elucidating fundamental mechanisms governing the regulation of non-coding RNAs, transcription, and cell inheritance; (2) determining how transcriptional and post-transcriptional control are dysregulated in cancer; and (3) developing therapeutic agents and biological systems to target pro-tumorigenic alterations in transcriptional and post-transcriptional regulators. The Program also has expertise in computational analysis of gene expression patterns, mRNA splicing, and mutation identification which is being used to uncover alterations that drive aberrant gene regulation and impact all three focus areas. Program members combine cell, molecular biology, biochemical, structural biology, computational, and genetic approaches. The Program is enhanced by the excellent Cancer Center Shared Resources, especially the Animal, Sequencing Technologies & Analysis, Flow Cytometry, Microscopy, and Mass Spectrometry Shared Resources.
Krainer wins 2021 Wolf Prize in Medicine
February 17, 2021
CSHL Professor Adrian Krainer was awarded the 2021 Wolf Prize in Medicine for his work on RNA splicing.
AI researchers ask: What’s going on inside the black box?
February 8, 2021
Although researchers have figured out how to train computers to recognize things, they have yet to understand how machines make those predictions.
How to bury carbon? Let plants do the dirty work.
February 5, 2021
Carbon sequestration could slow or reverse human emissions—and nothing is better at sequestration than a green plant.
CSHL Association holds its annual meeting
January 29, 2021
The Cold Spring Harbor Laboratory Association welcomes four new directors and thanks three retiring ones.
Building a corn cob—cell by cell, gene by gene
January 26, 2021
CSHL scientists are piecing together the genes that control how corn develops.
How roundworms decide the time is right
December 22, 2020
Roundworm embryos calibrate the speed of their development to environmental conditions. A similar system may go wrong in some cancers.
Regulatory RNAs promote breast cancer metastasis
December 22, 2020
A gene-regulating bit of RNA promotes breast cancer metastasis. Agents that destroy that RNA provide hope for a new drug.
Patricia Churchland: Social Conscience
November 24, 2020
Patricia Churchland, founder of the field of neurophilosophy, discusses research on the origins of human morality and social bonding.
LIVE At the Lab with Patricia Churchland: Social Conscience
November 24, 2020
Scientist and philosopher Patricia Churchland discusses the evolutionary basis of morality and social bonding in humans.
Joshua-Tor wins Biophysical Society honor
November 16, 2020
CSHL Professor and HHMI Investigator Leemor Joshua-Tor was named a 2021 Fellow of the Biophysical Society for her work on RNAi and DNA replication.
Mary Ruchalski Foundation donates $60k for RMS research
November 13, 2020
The Mary Ruchalski Foundation donated $60,000 to CSHL Professor Chris Vakoc and his team for ongoing rhabdomyosarcoma research.
One experiment: Organoids as living laboratories
October 30, 2020
These tiny balls of cells are revolutionizing the research and treatment of pancreas and other types of cancers.
DNA Learning Center Nigeria opens to local students
October 16, 2020
A small university in Nigeria hosts Africa’s first DNA Learning Center and plans to serve hundreds of students with hands-on genetics instruction.
Combining chemistry and biology at CSHL
October 1, 2020
Professor John Moses joins the CSHL faculty, specializing in the field of click chemistry.
The “ORC” twists, pinches, and dances around DNA
September 16, 2020
The Origin Recognition Complex (ORC) is a key piece of cellular machinery, fundamental to life, yet so far mysterious.
Replicating a genome starts with a twist, a pinch, and a bit of a dance
September 16, 2020
Researchers have their first high resolution look at how “ORC,” a human protein complex essential to life, moves.
Martienssen named 2020 Royal Society winner
August 3, 2020
Professor and HHMI Investigator Rob Martienssen wins a 2020 Royal Society medal for his RNAi research.
How two CSHL programs adapted during the COVID-19 pandemic
July 16, 2020
Mikala Egeblad and David Micklos presented their work at the “Life Science Across the Globe” seminar series.
CSHL tops Bush Center’s Innovation Impact Productivity Score
July 13, 2020
Cold Spring Harbor Laboratory ranks #1 in Innovation Impact Productivity Score among Pure Research/Health Care Institutions by the Bush Center.
Krainer wins 2020 NYAS Innovators prize for SMA research
July 8, 2020
Professor Adrian Krainer is the 2020 Senior Scientist Winner of the Innovators in Science Award for his work on SPINRAZA®.
The world destroyer in your shampoo and ice cream
July 6, 2020
Palm oil is an environmental scourge. Genetics has a solution.
Nobelist Sir Richard Roberts talks GMOs at CSHL
June 26, 2020
Nobelist and CSHL alum Sir Richard Roberts spoke about GMOs and the future of agriculture with Pamela Ronald and Rob Martienssen.
Nobelist Sir Richard Roberts talks GMOs at CSHL hosted event
June 25, 2020
Nobelist and CSHL alum Sir Richard Roberts spoke about GMOs and the future of agriculture with Pamela Ronald and Rob Martienssen in this video.
CSHL President Bruce Stillman wins Heineken Prize
June 2, 2020
CSHL President and CEO Bruce Stillman wins the Heineken Prize for his work on eukaryotic DNA replication.
Why pancreatic ductal adenocarcinoma is so lethal
May 19, 2020
CSHL researchers discovered factors that allow a pancreatic cell to lose its identity and turn into an aggressive cancer cell.
Coronavirus research in plants
May 15, 2020
Purified coronavirus proteins are in short supply for COVID-19 researchers, so CSHL plant scientists are jumping in to make them.
What do these scientist moms do? Ask their kids.
May 8, 2020
We asked the children of three scientists to describe their mother’s work. See what they had to say.
Adrian Krainer elected to the National Academy of Sciences
April 28, 2020
Professor Adrian Krainer was elected to the National Academy of Science as part of its 2020 election.
CSHL featured in new Ken Burns documentary, The Gene
April 6, 2020
The documentary will feature CSHL Professor Adrian Krainer, materials from the CSHL Library & Archives, and glimpses of the CSHL campus.
Barbara McClintock: Free to discover
March 23, 2020
Nobel laureate Barbara McClintock spent half her life at CSHL, enjoying the freedom to explore new ideas and ignore criticism. This is her journey.
Adrian Krainer wins Ross Prize in Molecular Medicine
March 2, 2020
Professor Adrian Krainer wins the Ross Prize for his pioneering work on antisense therapy and its application to treating SMA.
Christina Renna Foundation donates $38k to study sarcoma
February 27, 2020
The Christina Renna Foundation donated $38,000 to the lab of Professor Chris Vakoc.
Event: Canceled Cocktails & Chromosomes
February 18, 2020
As a precautionary measure to ensure the safety of our employees and visitors during the current COVID-19 outbreak, Cold Spring Harbor Laboratory (CSHL) is canceling and/or postponing events for public audiences.
Cracking the case of the norovirus
February 6, 2020
A pervasive virus has evaded vaccine developers for decades. By getting a clear look at its protective shell, they may finally know how to defeat it.
Mutation’s role in blood cancers revealed by ideal team-up
January 31, 2020
Researchers from CSHL and MSKCC have determined how a mutation implicated in many blood cancers reduces the proteins that make healthy blood cells.
Mutation’s role in blood cancers revealed by ideal collaboration
January 31, 2020
Researchers from CSHL and MSKCC have determined how a mutation implicated in many blood cancers reduces the proteins that make healthy blood cells.
Vitamin B6, leukemia’s deadly addiction
January 13, 2020
Acute Myeloid Leukemia is addicted to vitamin B6. Now that researchers know this, they can pursue new treatment options for the deadly blood cancer.
Vitamin B6, leukemia’s deadly addiction
January 13, 2020
Acute Myeloid Leukemia is addicted to vitamin B6. Now that researchers know this, they can pursue new treatment options for the deadly blood cancer.
Finally, machine learning interprets gene regulation clearly
December 26, 2019
Machine learning and a new kind of easily-interpretable artificial neural network is helping scientists make sense of crucial gene regulation.
Bridge to education
December 15, 2019
CSHL’s DNA Learning Center builds new bridges between unique science education and diverse groups.
Researchers identify gene behind spread of deadly breast cancer
December 9, 2019
Jackson Laboratory and Cold Spring Harbor Laboratory researchers uncovered novel targets for therapies related to deadly triple negative breast cancers.
Adrian Krainer wins 2019 Peter Speiser Award
November 27, 2019
Professor Adrian Krainer was awarded the Peter Speiser Award for advances in the pharmaceutical sciences.
Scientists take action to prevent sexual harassment and bias
November 7, 2019
Scientists gathered at Banbury Center last year to discuss ways to prevent gender bias and sexual harassment in science.
High school senior wins big in Breakthrough Junior Challenge
November 5, 2019
Winning the 2019 Breakthrough Junior Challenge, student Jeffery Chen earned his high school a $100,000 science lab designed by the DNA Learning Center.
Mary Ruchalski Foundation donates $30k for cancer research
October 30, 2019
The Mary Ruchalski Foundation donated $30,000 for Professor Chris Vakoc’s lab for continued sarcoma research.
Historic building—groundbreaking science
October 29, 2019
The Demerec building has been monumental in scientific history. Now, a $75 million renovation of this celebrated labspace will define CSHL’s future.
One experiment: The shape of norovirus
October 25, 2019
Scientists at CSHL examine the structure of a norovirus under the cryo-electron microscope.
Adrian Krainer elected to National Academy of Medicine
October 23, 2019
Professor Adrian Krainer joins the National Academy of Medicine in honor of his groundbreaking work on SMA.
Friends of T.J. donate $50,000 for sarcoma research
October 23, 2019
The Friends of T.J. Foundation donated $50,000 for Professor Chris Vakoc’s lab for sarcoma research on October 14, 2019.
Adrian Krainer wins Zülch Prize for lifesaving SMA Treatment
October 8, 2019
Professor Adrian Krainer is awarded the K-J. Zülch Prize for the development and testing of a first-of-its-kind treatment for spinal muscular atrophy.
Mutant cells team up to make an even deadlier blood cancer
October 2, 2019
Two very different mutations have been identified as unexpected partners-in-crime for causing the deadly blood cancer acute myeloid leukemia.
Discovery could improve MDS cancer treatment
September 25, 2019
Researchers have discovered a promising new target for treating myelodysplastic syndrome (MDS), a common and lethal blood cancer.
Peter Koo wants to understand how machines learn biology
September 20, 2019
Dr. Peter Koo joins the CSHL faculty as an assistant professor. His focus is on exploring how artificial intelligence integrates with biology and genomics.
The world of Brianna Bibel, The Bumbling Biochemist
September 17, 2019
Brianna Bibel, a third-year graduate student at WSBS, is making ripples in the science community through her social media presence.
How one family secured the future of a laboratory
September 12, 2019
The Robertson family gifts to CSHL in the early 1970s were of extraordinary importance, for without them the Laboratory might not have survived.
CSHL graduate student wins HHMI fellowship
August 15, 2019
WSBS student David Johnson won a 2019 Gilliam Fellowships for Advanced Study for leadership and diversity in science.
Event: Public Lecture: Seeing With Sequencing
August 8, 2019
Come hear from three quantitative biologists as they discuss how they see with sequencing to solve mysteries ranging from the genetics of evolution.
Cryo-EM illuminates intricate designs of noroviruses
June 11, 2019
Researchers reconstruct four different strains of the food-poisoning causing norovirus using the groundbreaking cryo-electron microscope.
Norovirus structures could help develop treatments for food poisoning
June 11, 2019
Researchers at CSHL used cryo-EM to reconstruct the structures of four different strains of human noroviruses, which cause food poisoning.
Leading discovery
May 20, 2019
Current discoveries about DNA and human genome position CSHL scientists to make life-changing breakthroughs that will improve the human condition.
An essay from the President: Biology for the planet
May 16, 2019
CSHL plant scientists are looking for solutions to the biggest questions in agriculture as environments are reshaped by climate change.
Andrea Schorn zooms in on small RNAs in the cell
April 29, 2019
Dr. Andrea Schorn joins CSHL faculty as research assistant professor. Her focus is exploring transposable elements in the genome of mammalian cells.
Seeing with sequencing—A public lecture with three CSHL experts
April 19, 2019
Quantitative biologists discuss how physics, modern computing power, and a new perspective on biology can make sense of our complex genomes.
Rob Martienssen wins Martin Gibbs Medal for plant research
April 15, 2019
CSHL Professor Rob Martienssen wins the 2019 Martin Gibbs Medal for his contributions to plant biology.
NIH grant awarded for interneuron research
April 4, 2019
CSHL postdoc Maggie Crow will use her NIH grant to pursue the quantification and analysis of specific types of neurons in the brain.
President Bruce Stillman wins prestigious Gairdner Award
April 2, 2019
Dr. Bruce Stillman, President & CEO of CSHL, has been awarded the 2019 Canada Gairdner International Award for his work on eukaryotic DNA replication.
CSHL President elected as AACR Academy Fellow
March 25, 2019
CSHL President and CEO Dr. Bruce Stillman has been elected to the AACR Academy as part of the 2019 Fellows class.
Adrian Krainer wins RNA Society’s Lifetime Achievement Award
March 8, 2019
Professor Adrian Krainer was awarded the RNA Society’s Lifetime Achievement Award for his life-saving research.
Genetic ‘usual suspects’ identified in researchers’ new list
March 4, 2019
An exhaustive ranked list of “usual suspect” genes involved in disease may prove invaluable for future research and drug discovery.
What does sight mean to a cancer researcher?
February 26, 2019
Postdoc Leah Banks discusses vision’s role in cancer research, and how nearly losing her sight gave her a new perspective on her work.
Christina Renna Foundation gives $35k for pediatric cancer research
February 7, 2019
The Christina Renna Foundation donated $35,000 to CSHL for continued work on Sarcoma Research Project and research into RMS.
Rob Martienssen special talk: Barbara McClintock’s Controlling Elements, Then and Now
January 25, 2019
Rob Martienssen's special lecture on Barbara McClintock's 'Controlling Elements, Then and Now,' given at the CSHL Library Szybalski Reading Room.
National Academy of Inventors Fellow, Adrian Krainer
December 11, 2018
Professor Adrian Krainer has been named a NAI 2018 Fellow for his work on the first FDA-approved treatment for spinal muscular atrophy.
Big bold dreamers
December 10, 2018
Dreamers drive discoveries. CSHL honors its many scientists making big impacts in their respective fields.
Breakthrough Prize laureates meet at CSHL
December 6, 2018
Breakthrough Prize winner Adrian Krainer & C. Frank Bennett reunite for the first time since their award ceremony at CSHL to discuss further project.
Women’s coalition donates $100k to breast cancer research
December 6, 2018
Members of the Manhasset Women’s Coalition Against Breast Cancer supported research through a donation from the Ladies Night Out fundraiser.
Adrian Krainer, ganador del Breakthough Prize
October 19, 2018
Profesor Adrian Krainer desarrolló un tratamiento para la Atrofia Muscular Espinal. Por su trabajo, recibió el prestigioso el Breakthrough Prize.
The big problem of small data: A new approach
October 18, 2018
You’ve heard of “big data” but what about small? Researches have crafted a modern approach that could solve a decades-old problem in statistics.
The hope of a Breakthough Prize Winner
October 17, 2018
Professor Adrian Krainer describes the hope he fulfilled with the development of the first effective therapy for the neurodegenerative disease SMA.
Breakthrough Prize to CSHL professor for SMA research
October 17, 2018
Professor Adrian Krainer recently received the 2019 Breakthrough Prize in Life Sciences for the development of a life-saving treatment for SMA.
Breakthrough Prize to CSHL professor for SMA research
October 17, 2018
Professor Adrian Krainer has received a Breakthrough Prize in Life Sciences for his work on the first FDA-approved treatment of SMA.
Friends of T.J. donate $50k for sarcoma research
October 9, 2018
Associate Professor Chris Vakoc and his team receive a donation to further their rhabdomyosarcoma research.
Path to cancer in the brain set by protein CHD5
September 14, 2018
Researchers find that protein keeps neural stem cells from activating too soon and hindering brain development
From workshops to lab work—right next door
September 13, 2018
College student James Korossy’s journey from taking workshops at the DNA Learning Center to working at the bench in CSHL Fellow Lingbo Zhang’s lab.
Predicting how splicing errors impact disease risk
August 30, 2018
New research helps correlate genetic mutations with errors in RNA splicing that can cause serious illness
CSHL helps ring opening bell at Nasdaq
August 21, 2018
CSHL Professor Adrian Krainer and his team join the Spinal Muscular Atrophy (SMA) Foundation in ringing the Nasdaq opening bell
Undergrad symposium highlights lessons in the lab
August 20, 2018
The 2018 Undergraduate Research Program class presents their projects after working for ten weeks in various labs across campus.
What does it mean to be an URP?
August 20, 2018
A quick chat with a student and his mentor reveals how much the work of our undergraduate researchers mean to CSHL. Hint: A whole lot.
Base Pairs Episode 16.5: Fuels of the fuels
August 16, 2018
Rob Martienssen explains how genetic modification and advances in technology factor into the future of fuel as well as cinematic sci-fi fuels.
Elisabeth R. Woods Foundation donates to cancer research
July 17, 2018
The Elisabeth R. Woods Foundation donated $30,000 for lung cancer research at their donor gathering, hosted at CSHL.
Base Pairs Episode 16: Big plans for a tiny plant
July 16, 2018
As temperatures around the globe continue to rise, scientists are working hard to develop solutions that deal with the consequences of climate change.
Big plans for a tiny plant
July 15, 2018
On this episode of Base Pairs, Rob Martienssen discusses how duckweed could be the next biofuel and help combat climate change
Energy from thin air: Basic research to biofuels
June 12, 2018
A plant scientist and an industry innovator discuss how simple duckweed may help address climate change and energy needs.
The case for open and inclusive science
June 8, 2018
America must continue to a place where the most talented people in the world want to live, study and work.
Nation’s cancer centers endorse HPV vaccination
June 8, 2018
Dr. Stillman and Dr. Tuveson joined with the leaders of other institutions nationwide in endorsing a statement regarding the HPV vaccine
A science writer’s quest to understand heredity
May 30, 2018
LabDish spoke with science writer Carl Zimmer about what he learned about heredity as he zig-zagged through CSHL while writing his new book.
A promising new strategy to combat familial dysautonomia
May 17, 2018
Scientists determine how to correct the error in RNA splicing that causes familial dysautonomia, a rare inherited neurodegenerative disorder.
Nature’s masterpiece: the brain
May 11, 2018
A message from President and CEO Dr. Bruce Stillman from the Spring 2018 Harbor Transcript on the innovative research happening at CSHL
Therapeutic RNA corrects splicing defect that causes familial dysautonomia
April 30, 2018
A team provides proof of concept for an RNA drug correcting an error in RNA splicing that causes familial dysautonomia (FD)
Portrait of a Neuroscience Powerhouse
April 27, 2018
A relatively small neuroscience group at CSHL is having an outsized impact on a dynamic and highly competitive field


Jesse Gillis
There has been a growing appreciation in recent years that gene function is frequently context-dependent, with a large part of that context provided by the activities of other genes. The Gillis lab focuses on characterizing these shared patterns of gene activity through co-expression networks and showing how they can lead to changes in cell function, particularly in single cell expression data.

Christopher Hammell
As organisms develop, genes turn on and off with a precise order and timing, much like the order and duration of notes in a song. My group uses model organisms to understand the molecules that control the tempo of development. We also study how changes in the timing of gene expression contribute to diseases like cancer.

Leemor Joshua-Tor
Our cells depend on thousands of proteins and nucleic acids that function as tiny machines: molecules that build, fold, cut, destroy, and transport all of the molecules essential for life. My group is discovering how these molecular machines work, looking at interactions between individual atoms to understand how they activate gene expression, DNA replication, and small RNA biology.

Justin Kinney
Research in the Kinney Lab combines mathematical theory, machine learning, and experiments in an effort to illuminate how cells control their genes. These efforts are advancing the fundamental understanding of biology and biophysics, as well as accelerating the discovery of new treatments for cancer and other diseases.

Peter Koo
Deep learning has the potential to make a significant impact in basic biology and cancer, but a major challenge is understanding the reasons behind their predictions. My research develops methods to interpret this powerful class of black box models, with a goal of elucidating data-driven insights into the underlying mechanisms of sequence-function relationships.

Adrian R. Krainer
Our DNA carries the instructions to manufacture all the molecules needed by a cell. After each gene is copied from DNA into RNA, the RNA message is "spliced" - an editing process involving precise cutting and pasting. I am interested in how splicing normally works, how it is altered in genetic diseases and cancer, and how we can correct these defects for therapy.

Rob Martienssen
Chromosomes are covered with chemical modifications that help control gene expression. I study this secondary genetic code - the epigenome - and how it is guided by small mobile RNAs in plants and fission yeast. Our discoveries impact plant breeding and human health, and we use this and other genomic information to improve aquatic plants as a source of bioenergy.

Alea A. Mills
Cells employ stringent controls to ensure that genes are turned on and off at the correct time and place. Accurate gene expression relies on several levels of regulation, including how DNA and its associated molecules are packed together. I study the diseases arising from defects in these control systems, such as aging and cancer.

John Moses
My group uses click chemistry to study biological systems at the molecular level. We develop and exploit powerful bond-forming click reactions that enable the rapid synthesis of small functional molecules, including cancer drugs and chemical probes. We apply these novel molecular tools in multidisciplinary discovery projects spanning the fields of biology and chemistry.

Jon Preall
Developing single-cell genomics technologies for applications related to cancer progression, immune surveillance, and discovery of rare novel cell types and transcriptional programs.

Andrea Schorn
Transposable elements make up half of our DNA. They control gene expression and have been a major evolutionary force in all organisms. The Schorn lab investigates how small RNAs identify and silence transposable elements when they become active during development and cancer.

David L. Spector
The immense amount of DNA, RNA and proteins that contribute to our genetic programs are precisely organized inside the cell's nucleus. My group studies how nuclear organization impacts gene regulation, and how misregulation of non-coding RNAs contributes to human diseases such as cancer.

Bruce Stillman
Every time a cell divides, it must accurately copy its DNA. With 3 billion “letters” in the human genome, this is no small task. My studies reveal the many steps and molecular actors involved, as well as how errors in DNA replication are involved in diseases that range from cancer to rare genetic disorders.

Lingbo Zhang
Proper balancing of self-renewal and differentiation in hematopoietic stem and progenitor cells is a central question in hematopoiesis. My laboratory investigates how growth signal and nutrient coordinate to regulate this key process and aims to develop novel therapeutic strategies for hematological diseases and malignancies.