Test Tube Tuesday

Experimental Treatment Wards Off Blinding Autoimmune Disease

Drug Candidate Calms Overzealous Immune Response in the Eye

damage to the eye caused by hyperactive immune cells

Our immune cells don’t like strangers and attack many organisms and substances that they have never seen before, including harmless ones. In autoimmune diseases, this reaction gets out of hand and our own cells are caught in the crossfire. IRP scientists have found that a new therapeutic compound can curb this sort of autoimmune carnage in the eye.

Cutting-Edge Carriers Deliver Controllable Cancer Chemotherapy

New System Could Boost Treatment Effectiveness and Curb Side Effects

nanovesicles

Cancer kills more than half a million men, women, and children each year in the U.S, and chemotherapy is only slightly more discriminating than the disease it treats. As a result, many cancer treatments kill cells throughout the body and cause severe side effects. New IRP research could solve this problem by creating a way to release those toxic compounds only when and where doctors desire.

Inflammation Cuts Lifeline for Blood-Producing Stem Cells

Discovery Could Lead to New Approaches for Boosting Blood Cell Counts

red blood cell (left), platelet (middle), and white blood cell (right)

Much of human biology is a black box — scientists know the key players and the end results, but not how those outcomes come about. Consequently, it remains a mystery why some medications help patients. A new IRP study has cracked open the black box to reveal how high levels of an inflammatory molecule inhibit blood cell production in some individuals and why a particular medicine helps reverse this life-threatening condition.

Cutting-Edge Technique Simultaneously Edits Multiple Genetic Targets

Alternative to CRISPR/Cas9 May Cause Fewer Undesired Changes

diagram of DNA strand

IRP researchers have always worked on the cutting edge of biomedical science, from testing the first successful treatment for childhood schizophrenia to pioneering the first screening technique for HIV. In a new study, an IRP team recently achieved yet another first: simultaneously editing two genetic sites in mice using a brand-new approach called base editing that may prove to be more precise – and therefore safer – than other gene editing methods.

Lab-Designed Virus Shows Promise for Inner Ear Gene Therapy

Delivery Method Could Eventually Help Correct Mutations That Cause Hearing Loss

inner ear cells infected by a lab-designed virus with a gene that produces a glowing green protein

Most people probably think of viruses as villains that bring illnesses like measles, HIV, and the flu, but some viruses are proving to be valuable allies in the fight against genetic diseases. In a new study, a team of scientists from the NIH IRP and their colleagues showed the promise of a lab-designed virus for delivering gene therapies aimed at correcting hereditary hearing loss.

Cholesterol Molecule Yields Insights Into Distressed Lungs

Potential biomarker may contribute to personalized treatments

diagram of fluid buildup in the lungs' air sacs

Until recently, medical treatment has largely been one-size-fits-all, with doctors unable to separate patients into distinct groups that might benefit more or less from a particular approach. However, researchers are increasingly finding that individuals with the same disease can differ markedly in ways that might one day influence their care. A recent IRP study has identified a particular molecule that may have just such an impact for patients with damaged lungs.

Cutting Calories Combats Aging Arteries

red blood cells traveling through a blood vessel

While many people think staving off aging means drinking seaweed smoothies and swallowing fish oil supplements, the key to extending life increasingly appears to be not putting things in our mouths. In yet another example of the anti-aging powers of eating less — what scientists call caloric restriction — IRP researchers have identified the specific aging-induced cellular and molecular changes in arteries that are curbed by substantially reducing calorie intake.

Experimental Therapy Reverses Neurological Effects of Malaria

MRI brain scans of mice with (right) and without (left) cerebral malaria, indicating areas of damage to the blood-brain barrier

For Americans and others living outside the tropics, a mosquito bite is nothing more than an itchy inconvenience, but for billions of others, it can lead to a life-or-death battle with malaria. In some cases, the illness can wreak havoc on the brain. A new IRP study has used magnetic resonance imaging (MRI) technology to demonstrate that an investigational therapy can reverse that damage in mice.

Diabetes Drug Helps Patients by Modifying the Microbiome

bacteria in the digestive system

The more scientists have learned about the community of benign bacteria inside our bodies, known as the microbiome, the more effort they have put into recruiting it in the fight against disease. What’s more, scientists occasionally discover that treatments long thought to work completely independently of our native microbes also relieve symptoms by interacting with them. New IRP research into the most commonly used medication for type 2 diabetes has led to just such a revelation by demonstrating that its benefits stem in part from its ability to kill off a particular species of bacteria in the human digestive tract.

Smartphone Apps Show Promise for Assessing Multiple Sclerosis Symptoms

brain scan of a patient with multiple sclerosis

The three-quarters of Americans who own a smartphone use them not just for communicating but also keeping a calendar, playing games, scouring the Internet for funny cat memes, and — soon — maybe even evaluating their neurological health. A new study conducted by IRP and University of Maryland researchers has confirmed the potential of smartphone apps for gauging symptoms of the neurological disease multiple sclerosis.