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Long Non-Coding RNAs as Novel Biomarkers and Therapeutic Targets in Pediatric Leukemia

New York University School of Medicine
Iannis Aifantis, PhD
Grant Type: 
Innovation Grants
Year Awarded: 
Project Description: 

Acute lymphoblastic leukemia (ALL) is the most common type of childhood cancer with more than 3,000 children/adolescents under the age of 20 diagnosed with ALL each year in the United States. It mostly afflicts children between the ages 3 and 5. ALL is a disease that affects a type of white blood cells called lymphocytes that help the body fight infection and disease. ALL is characterized by the presence of a large number of immature/non-functional lymphocytes which accumulate in the liver, spleen, bone marrow and lymph nodes. The current treatments of ALL include chemotherapy, radiation therapy, stem cell transplant, targeted therapy and, more recently, immunotherapy available only for B cell ALL (B-ALL). In spite of these treatment options, children with ALL continue to have dismal prognosis, especially the ones that relapse, due to the absence of targeted therapies. Better targeted therapies that target leukemic cells without harming normal cells are the need of the hour. 

Project Goal
We aim to evaluate a new class of target molecules, called long non-coding RNA, as both prognostic and therapeutic candidates in ALL. Our initial analysis predicts that these long non-coding RNA molecules show exquisite specificity amongst the distinct types of ALL and this aspect could be exploited for better diagnosis and targeted therapy of children affected by ALL.