the National Basic Research Program of China (973 Program) (No 2004CB518806);the National High-tech R&D Program (863 Program) (No 2007AA021206);the National Natural Science Foundation of China (No 30571777);the Chinese National and Shanghai Leading Academic Disci-pline Project (No B204)
Large amounts of aberrantly spliced mRNA from the β^654 allele was present in erythroid cells, which might impair the erythropoiesis. A therapeutic strategy for β-thalassemia was explored by knocking down the aberra...