Anti-tfr:acid sphingomyelinase for treatment of acid sphingomyelinase deficiency

Abstract

Multidomain therapeutic proteins comprising a TfR-binding delivery domain fused to an acid sphingomyelinase polypeptide and nucleic acid constructs and compositions that allow insertion of a multidomain therapeutic protein coding sequence into a target genomic locus such as an endogenous ALB locus and/or expression of the multidomain therapeutic protein coding sequence are provided. The multidomain therapeutic proteins and nucleic acid constructs and compositions can be administered to cells, populations of cells, or subjects and can be used in methods of integration of a multidomain therapeutic protein nucleic acid into a target genomic locus, methods of expression of a multidomain therapeutic protein in a cell, methods of treating acid sphingomyelinase deficiency in a subject, and methods of preventing or reducing the onset of a sign or symptom of acid sphingomyelinase deficiency in a subject.

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