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Page 1
Mucopolysaccharidosis IVA: Diagnosis, Treatment, and Management.
Int J Mol Sci. 2020 Feb 23;21(4):1517. doi: 10.3390/ijms21041517.
Int J Mol Sci. 2020.
PMID: 32102177
Free PMC article.
Review.
Evading the AAV Immune Response in Mucopolysaccharidoses.
Piechnik M, Sawamoto K, Ohnishi H, Kawamoto N, Ago Y, Tomatsu S.
Piechnik M, et al.
Int J Mol Sci. 2020 May 13;21(10):3433. doi: 10.3390/ijms21103433.
Int J Mol Sci. 2020.
PMID: 32414007
Free PMC article.
Review.
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Sex Difference Leads to Differential Gene Expression Patterns and Therapeutic Efficacy in Mucopolysaccharidosis IVA Murine Model Receiving AAV8 Gene Therapy.
Piechnik M, Amendum PC, Sawamoto K, Stapleton M, Khan S, Fnu N, Álvarez V, Pachon AMH, Danos O, Bruder JT, Karumuthil-Melethil S, Tomatsu S.
Piechnik M, et al.
Int J Mol Sci. 2022 Oct 21;23(20):12693. doi: 10.3390/ijms232012693.
Int J Mol Sci. 2022.
PMID: 36293546
Free PMC article.
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Effect of enzyme replacement therapy on the growth of patients with Morquio A.
Doherty C, Stapleton M, Piechnik M, Mason RW, Mackenzie WG, Yamaguchi S, Kobayashi H, Suzuki Y, Tomatsu S.
Doherty C, et al. Among authors: piechnik m.
J Hum Genet. 2019 Jul;64(7):625-635. doi: 10.1038/s10038-019-0604-6. Epub 2019 Apr 24.
J Hum Genet. 2019.
PMID: 31019230
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