Discoveries
Genetics
Genetics
We are rapidly demystifying cancers, exposing the molecular mechanisms underlying tumors and leading the search for the next generation of targeted cancer therapies. We see a future where every cancer and every patient has a cure.

Discoveries

Nature Communications
06/2022

RNA processing goes awry in rare immune disease

Professor Juan Carlos Izpisua Belmonte and colleagues from King Abdullah University of Science and Technology (KAUST) in Saudi Arabia have discovered a new underlying cause of Wiskott-Aldrich syndrome, a rare genetic disease that leads to bleeding and immune deficiencies in babies. Their findings involve the way cells cut and paste strands of RNA in a process called RNA splicing. The genetic mutations associated with Wiskott-Aldrich syndrome, they found, disrupt this process, ultimately preventing numerous immune and anti-inflammatory proteins from being made correctly.

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Nature Immunology
06/2022

Hair-raising research: The fascinating link between the immune system and hair growth

Associate Professor Ye Zheng, first author Zhi Liu, and colleagues have uncovered an unexpected molecular target of a common treatment for alopecia, a condition in which a person’s immune system attacks their own hair follicles, causing hair loss. Their findings describe how immune cells interact with skin cells using a hormone as a messenger to generate new hair follicles and hair growth.

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Science Advances
07/2022

Imaging solves mystery of how large HIV protein functions to form infectious virus

Assistant Professor Dmitry Lyumkis, co-first author Dario Passos, and colleagues from Rutgers University have determined the molecular structure of HIV Pol, a protein that plays a key role in the late stages of HIV replication—the process through which the virus propagates itself and spreads through the body. The molecule’s structure helps answer long-standing questions about how the protein breaks itself apart to advance the replication process. The discovery reveals a new vulnerability in the virus that could be targeted with drugs.

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Nature
07/2022

Making a memory positive or negative

Professor Kay Tye, co-first author Hao Li, and team have discovered a molecule in the brain responsible for associating good or bad feelings with a memory. Their finding paves the way for a better understanding of why some people are more likely to retain negative emotions than positive ones—as can occur with anxiety, depression, or post-traumatic stress disorder (PTSD).

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PNAS
07/2022

The best offense is a great defense for some carnivorous plants

Insect-eating plants have fascinated biologists for more than a century, but how plants evolved the ability to capture and consume live prey has largely remained a mystery. Professor Joanne Chory, Staff Scientist Carl Procko, and colleagues from Washington University in St. Louis have found evidence that plant carnivory evolved from mechanisms plants use to defend themselves. The findings broaden scientists’ understanding of how plants interact with their environments.

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Molecular Therapy – Methods & Clinical Development
07/2022

Discovery advances the potential of gene therapy to restore hearing loss

Sensory hair cells in the inner ear use long, hair-like structures called stereocilia to transduce sound. In the absence of the protein EPS8, stereocilia are too short to function, leading to deafness. Assistant Research Professor Uri Manor and colleagues from the University of Sheffield found that delivery of EPS8 can rescue stereocilia elongation and function in mice affected by the loss of EPS8. Their study shows promise for the development of gene therapies to repair hearing loss.

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Immunity
08/2022

Surprising link between mitochondrial DNA and increased atherosclerosis risk

Mitochondria are known as cells’ powerhouses, but mounting evidence suggests they also play a role in inflammation. Professor Gerald Shadel and colleagues from UC San Diego examined human blood cells and discovered a surprising link between mitochondria, inflammation, and DNMT3A and TET2—two genes that normally help regulate blood cell growth but, when mutated, are associated with an increased risk of atherosclerosis.

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Science Translational Medicine
08/2022

New target identified for treatment of premature aging disease

Professor Juan Carlos Izpisua Belmonte, co-first author Pradeep Reddy, and colleagues from KAUST in Saudi Arabia have discovered that a stretch of DNA that hops around the human genome plays a role in premature aging disorders. In people with early aging, or progeria, RNA encoded by this mobile DNA builds up inside cells. What’s more, the scientists found that blocking this RNA reverses the disease in mice.

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Nature Communications
08/2022

Light and temperature work together to affect plant growth

Plants lengthen and bend to secure access to sunlight, yet scientists do not fully understand this process. Professor Joanne Chory, first author Yogev Burko, and colleagues have discovered that two plant factors—the protein PIF7 and the growth hormone auxin—are the triggers that accelerate growth when plants are shaded by canopy and exposed to warm temperatures at the same time. The findings will help scientists increase crop productivity despite the yield-harming global temperature rise.

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Nature Neuroscience
08/2022

Beyond neurons: How cells called astrocytes contribute to brain disorders

Neurons often get most of the credit for keeping our brains sharp and functioning—as well as most of the blame when it comes to brain diseases. But star-shaped cells called astrocytes, another abundant cell in the human brain, may bear the brunt of the responsibility for exacerbating the symptoms of some neurodevelopmental disorders. Associate Professor Nicola Allen and colleagues have now identified a molecule produced by astrocytes that interferes with normal neuron development in Rett, fragile X, and Down syndromes.

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Cell Reports
08/2022

How the brain gathers threat cues and turns them into fear

Assistant Professor Sung Han, co-first authors Sukjae Joshua Kang and Shijia Liu, and colleagues have uncovered a molecular pathway that distills threatening sights, sounds, and smells into a single message: Be afraid. A molecule called CGRP enables neurons in two separate areas of the brain to bundle threatening sensory cues into a unified signal, tag it as negative, and convey it to the amygdala, which translates the signal into fear. The research may lead to new therapies for fear-related disorders such as PTSD or hypersensitivity disorders such as autism, migraines, and fibromyalgia.

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Science Advances
09/2022

Aggression de-escalation gene identified in fruit flies

The brain mechanisms that cause aggressive behavior have been well studied. Far less understood are the processes that tell the body when it’s time to stop fighting. Associate Professor Kenta Asahina and colleagues have now identified a gene and a group of cells in the brain that play a critical role in suppressing aggression in fruit flies. The findings have implications for disorders such as Parkinson’s disease, which can sometimes cause behavioral changes like increased aggression and combativeness.

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