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Hemophilia
- ... or inherited, disorders such as cystic fibrosis , thalassemia , hemophilia , and sickle cell disease . Genetic therapies are approaches ...
- ... Loss GM1 Gangliosidosis Tay-Sachs and Sandhoff Disease Hemophilia Huntington's Disease Inherited Retinal Diseases Mucopolysaccharidoses (MPS) Muscular ...