Rocket Pharmaceuticals RCKT announced that the EMA accepted the marketing authorization application (MAA), seeking approval for RP-L102, an investigational gene therapy for fanconi anemia (FA). FA is ...
Mutations in FANCX appear to cause a lethal form of Fanconi anemia, a finding that sheds light on unexplained pregnancy loss and offers new avenues for genetic screening. Fanconi anemia is an ...
The ‘heart’ of the Fanconi anemia core complex, composed of FANCB-FANCL-FAAP100 (now FANCX) proteins, allows the rest of the core complex to assemble, and is necessary for downstream DNA repair.
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