Out of severe combined immunodeficiency, Leber's congenital amaurosis 2, spinal muscular atrophy, Duchenne muscular dystrophy, sickle cell anemia, beta-thalassemia, and cystic fibrosis, which can be treated successfully with ex vivo therapy and what do those have in common that makes them amenable to ex vivo therapy? Consider the cells/tissues affected by each disease in relation to the requirements for a successful ex vivo therapy.

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Michaelcarroll7230idn Michaelcarroll7230idn

13-06-2024
Medicine

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Out of severe combined immunodeficiency, Leber's congenital amaurosis 2, spinal muscular atrophy, Duchenne muscular dystrophy, sickle cell anemia, beta-thalassemia, and cystic fibrosis, which can be treated successfully with ex vivo therapy and what do those have in common that makes them amenable to ex vivo therapy? Consider the cells/tissues affected by each disease in relation to the requirements for a successful ex vivo therapy.

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