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Cell type-selective targeted delivery of a recombinant lysosomal enzyme for enzyme therapies.
Mol Ther. 2021 Dec 1;29(12):3512-3524. doi: 10.1016/j.ymthe.2021.08.020. Epub 2021 Aug 14.
Mol Ther. 2021.
PMID: 34400331
Free PMC article.
Skeletal phenotype amelioration in mucopolysaccharidosis VI requires intervention at the earliest stages of postnatal development.
Hwang-Wong E, Amar G, Das N, Zhang X, Aaron N, Gale K, Rothman N, Fante M, Baik A, Bhargava A, Fricker A, McAlister M, Rabinowitz J, Lees-Shepard J, Nannuru K, Economides AN, Cygnar KD.
Hwang-Wong E, et al.
JCI Insight. 2023 Nov 8;8(21):e171312. doi: 10.1172/jci.insight.171312.
JCI Insight. 2023.
PMID: 37751300
Free PMC article.
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