Research Highlights - Autumn 2016 - St. Jude Children’s Research Hospital

CRISPR gene editing reveals new therapeutic approach for blood disorders

Researchers found a way to use genome editing to help fix sickle cell disease and beta-thalassemia in blood cells isolated from patients.
Researchers identify master flu assassin

Molecular "master assassin" identified that recognizes flu-infected cells and triggers cell death, raises hope for drugs to prevent flu complications.
Modeling and simulation help optimize chemotherapy to combat brain tumor

Pharmacologic modeling and simulation help translate preclinical data into phase I trial of the drug 5-FU for treatment of brain tumor ependymoma.
St. Jude researchers reveal how two types of immune cells can arise from one

Lopsided distribution of the signaling protein c-Myc plays a critical role in determining T cell destiny during asymmetric cell division.
Targeted antibiotic is easier on the gut microbiome

Pathogen-specific antibiotic is less likely to kill good bacteria in gut, raising hopes for reduced side effects caused by disruption of microbiome.
Rare genetic variations may solve porphyria mystery

Variations in a porphyrin transport protein may affect symptom severity in patients with porphyria, a rare disorder of skin, liver and nervous system.
Newly discovered immune cell type protects against lung infections during chemotherapy

Newly recognized lung macrophage protects chemotherapy-treated mice with a depleted immune system from lethal bacterial pneumonia
Some adolescent cancer survivors may require more comprehensive mental health screening

Study shows that most teen cancer survivors do well psychologically, but cancer treatment may influence some survivors’ symptoms