Below are the most recent publications written about "Lysosomal Storage Diseases" by people in Profiles.
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Leal AF, Benincore-Fl?rez E, Rintz E, Herre?o-Pach?n AM, Celik B, Ago Y, Alm?ciga-D?az CJ, Tomatsu S. Mucopolysaccharidoses: Cellular Consequences of Glycosaminoglycans Accumulation and Potential Targets. Int J Mol Sci. 2022 Dec 28; 24(1).
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Cao M, Luo X, Wu K, He X. Targeting lysosomes in human disease: from basic research to clinical applications. Signal Transduct Target Ther. 2021 11 08; 6(1):379.
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Arunkumar N, Langan TJ, Stapleton M, Kubaski F, Mason RW, Singh R, Kobayashi H, Yamaguchi S, Suzuki Y, Orii K, Orii T, Fukao T, Tomatsu S. Newborn screening of mucopolysaccharidoses: past, present, and future. J Hum Genet. 2020 Jul; 65(7):557-567.
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Chen HH, Sawamoto K, Mason RW, Kobayashi H, Yamaguchi S, Suzuki Y, Orii K, Orii T, Tomatsu S. Enzyme replacement therapy for mucopolysaccharidoses; past, present, and future. J Hum Genet. 2019 Nov; 64(11):1153-1171.
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Safary A, Akbarzadeh Khiavi M, Omidi Y, Rafi MA. Targeted enzyme delivery systems in lysosomal disorders: an innovative form of therapy for mucopolysaccharidosis. Cell Mol Life Sci. 2019 Sep; 76(17):3363-3381.
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Min SH, Suzuki A, Stalker TJ, Zhao L, Wang Y, McKennan C, Riese MJ, Guzman JF, Zhang S, Lian L, Joshi R, Meng R, Seeholzer SH, Choi JK, Koretzky G, Marks MS, Abrams CS. Loss of PIKfyve in platelets causes a lysosomal disease leading to inflammation and thrombosis in mice. Nat Commun. 2014 Sep 02; 5:4691.
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Malatack JJ, Consolini DM, Bayever E. The status of hematopoietic stem cell transplantation in lysosomal storage disease. Pediatr Neurol. 2003 Nov; 29(5):391-403.
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Wenger DA, Coppola S, Liu SL. Insights into the diagnosis and treatment of lysosomal storage diseases. Arch Neurol. 2003 Mar; 60(3):322-8.
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Wenger DA, Coppola S, Liu SL. Lysosomal storage disorders: diagnostic dilemmas and prospects for therapy. Genet Med. 2002 Nov-Dec; 4(6):412-9.
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Leimig T, Mann L, Martin Mdel P, Bonten E, Persons D, Knowles J, Allay JA, Cunningham J, Nienhuis AW, Smeyne R, d'Azzo A. Functional amelioration of murine galactosialidosis by genetically modified bone marrow hematopoietic progenitor cells. Blood. 2002 May 1; 99(9):3169-78.