Better Way to Deliver Fetal Therapy for Serious Genetic Disorders
Delivering medicine through amniotic fluid is as effective as delivering it to the fetal brain via cerebrospinal fluid to treat serious disordrs such as Angelman syndrome.
University of California San Francisco
Can a New Drug Candidate Cure Pancreatic Cancer?
A new drug candidate permanently modifies a wily cancer-causing mutation, paving the way for making pancreatic cancer treatable, or perhaps even curable.
Targeted Treatment of Rare Childhood Leukemia
Juvenile myelomonocytic leukemia (JMML) is a rare but aggressive childhood leukemia. While hematopoietic stem cell transplantation is curative for some patients, approximately half of all patients see their leukemia return following a transplant.