DNA

Friendly Virus Could Deliver Gene Therapy Under Immune System’s Radar

IRP Research Points to New Tool for Alleviating Genetic Disorders

antibodies combatting viral particles

With all the dangerous viruses out there, from the seasonal flu to the novel coronavirus that causes COVID-19, people understandably want to make sure their immune systems are topped-up with disease-fighting antibodies that block viral invaders. However, when it comes to the viruses scientists are modifying to deliver gene therapy, having a robust immune response is actually an obstacle to getting healthy. In a new study, IRP researchers showed that most people’s immune systems don’t react to a particular harmless virus that can effectively deliver new genes to the liver and heart, making it a promising delivery vehicle for therapies designed to alleviate a life-threatening genetic condition.

Understanding the Foundations of Immune Defenses

IRP’s Mary Carrington Elected to the American Academy of Arts and Sciences for Insights Into Immune System Variations

Dr. Mary Carrington

Some people never seem to get sick, while others catch a new bug of some sort every other week. Humans are immensely variable both in their capacity to shrug off illness and in the ways their bodies respond to medical treatments. IRP senior investigator Mary Carrington, Ph.D., has spent her entire career exploring the biological roots of these differences, and the discoveries she has made earned her election to the American Academy of Arts and Sciences earlier this year.

Mosquitos With Human Gene Hinder Malaria Transmission

Genetically Modified Insects Could Help Curb Infections

transgenic mosquitos with blue, red, and green eyes

“Scientists create genetically modified mosquitos” sounds like the plot of a bad sci-fi movie, but it’s actually the reality in labs all around the world. Researchers are producing these ‘transgenic’ mosquitos in the hopes that the bugs could help combat the scourge of malaria, and in a recent study, IRP scientists demonstrated that their unique strategy in this realm has strong potential to accomplish that goal.

Postdoc Profile: From Bench to Bedside and Back Again

Dr. Stefan Barisic Turns Laboratory Discoveries into Kidney Cancer Treatments

Dr. Stefan Barisic

The Laboratory of Transplantation Immunotherapy sits at the heart of the NIH Clinical Center, just down the hallway from the Southeast inpatient unit. Here, IRP postdoctoral research fellow Stefan Barisic, M.D., labors at the bench with the goal of creating practical treatments for kidney cancer patients. Having such proximity to his patients was one of the chief attractions of working at NIH for Dr. Barisic.

“The NIH Clinical Center is an amazing place because it has all the resources you need to go from the bench to the bedside and back to the bench all in one building,” says Dr. Barisic.

Award Honors Promising Female Scientists

Women Scientists Advisors Select Three Young Researchers for Recognition

Dr. Sally Chang

While women have now overtaken men in terms of admission and enrollment in undergraduate education, they remain underrepresented in the sciences. This includes at NIH, where 74 percent of senior investigators and 54 percent of tenure-track investigators are male, according to the most recent statistics available. Consequently, NIH is putting considerable effort into supporting women scientists at all stages of their careers.

One NIH entity dedicated to this important work is the NIH Women Scientists Advisors (WSA), a group of women elected to represent the interests of women scientists in the IRP. Among its many initiatives, each year the WSA chooses several female postdoctoral fellows or graduate students in the IRP to receive the WSA Scholar Award in recognition of their outstanding scientific achievements. The awardees present their research at the annual WSA Scholars Symposium, which this year was held on April 25 and recognized young women leading efforts to better understand how disease-related genes evolved, an investigation of how a fatty liver can give rise to liver cancer, and the evaluation of a way to deliver gene therapy for a rare genetic disease. Read on to learn more about this year’s WSA Scholars and the impressive discoveries they have made during their time in the IRP.

New Lasker Scholars Begin Breaking New Ground

Early-Career Scientists Power Through Pandemic to Launch Labs

NIH’s 2021 Lasker Clinical Research Scholars

NIH has long prided itself on its ability to accelerate the careers of the brightest young physicians and scientists in the country. One of these many efforts is the Lasker Clinical Research Scholars Program, which provides a select group of individuals relatively early in their scientific careers with the funding and institutional support to start their own labs at NIH. After five to seven years of independent research in the IRP, Lasker Scholars are given the option to apply for three years of funding for work outside of NIH or to remain as investigators at NIH.

While launching a lab in the midst of a global pandemic is no easy task, five Lasker Scholars have done just that over the past year. Their research on cancer, Parkinson’s disease, childhood blindness, and inflammatory conditions is now well underway and promises to eventually improve the lives of many patients. Keep reading to learn more about how NIH’s newest Lasker Scholars are changing the way we treat those illnesses.

Older Cells Make for Riskier Transplants

Examining Molecular Markers of Aging Could Improve Patient Outcomes

old clock

In 2003, 92-year-old Fauja Singh ran the Toronto Waterfront Marathon in slightly under six hours, a feat that many people decades younger could not accomplish. Such examples reveal the problems with making assumptions about a person’s health based solely on age. Similarly, new IRP research suggests that assessing cellular characteristics associated with aging, rather than a person’s chronologic age in years, could improve outcomes for the more than 20,000 patients who receive bone marrow or blood stem cell transplants each year.

Tiny Molecules Have Big Potential for Treating Eye Diseases

Approach Could Protect or Even Regenerate Neurons in Eye and Spinal Cord

eye

At the end of Aesop’s fable The Lion and the Mouse, the titular rodent saves his much larger friend from a hunter’s trap. Just like Aesop, scientists know well that even something tiny and often overlooked can lend a helping hand. Extremely short strands of genetic material called microRNAs, for instance, could make for useful therapeutic targets for glaucoma and other degenerative eye ailments, according to new IRP research.

Global Scientists Come Together at the National Institutes of Health

Individuals From Around the World Drive IRP Breakthroughs

Dr. Flossie Wong-Staal

Come to NIH and you’ll hear many accents. Scientists from around the world have always contributed significantly to the NIH mission. The resulting diversity of backgrounds and perspectives makes the NIH Intramural Research Program an extremely stimulating and productive environment. Read on to learn about some of the many scientists of the past and present who brought their talents from abroad to one of the world’s leading institutions for biomedical research.

Dog Genome Yields Clues to Human Cancer

IRP Research Identifies Genetic Risk Factors for Highly Lethal Disease

flat-coated retriever

We may share our food and even our beds with them, but despite what many dog lovers might like to believe, our canine companions are not humans who just happen to walk on four legs. One thing we do have in common, though, is the array of genetic diseases that afflict both man and man’s best friend. As a result, scientists can learn a great deal about human illnesses by studying dogs. Using this approach, IRP researchers recently discovered genetic variants that likely play an important role in a rare and poorly understood form of cancer.