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(2018) A blood‐Mind‐barrier‐penetrating anti‐human transferrin receptor antibody fusion protein for neuronopathic mucopolysaccharidosis II. Mol TherEqually CRISPR‐Cas9‐ and iPSC‐centered strategies offer you unparalleled chances to the era of mobile designs of LSDs. These can be utilized for the identification of latest pathways invol