Results 121 to 130 of about 12,127 (209)

Genotype-Phenotype Correlations and Shifting Diagnosis Age in Turkish Mucopolysaccharidosis Type II Patients: A Multicenter Retrospective Study. [PDF]

Diagnostics (Basel)
Yazıcı H, Kara E, Bulut FD, Çelik MY, Köşeci B, Erdem F, Burgaç E, Yanbolu AY, Kaplan İ, Durmaz A, Aykut A, Canda E, Kor D, Uçar SK, Sözmen E, Çoker M, Mungan HNÖ.   +16 more
europepmc   +1 more source

Nebulized and intravenous enzyme replacement therapy in mice with mucopolysaccharidosis type II. [PDF]

PLoS One
Shamoun AJ, Reis G, Ashley M, Labilloy A, Ferreira LF.   +4 more
europepmc   +1 more source

Gene Therapy for Mucopolysaccharidosis Type II-A Review of the Current Possibilities. [PDF]

Int J Mol Sci, 2021
Zapolnik P, Pyrkosz A.
europepmc   +1 more source

Phenotypic Correction of Murine Mucopolysaccharidosis Type II by Engraftment of Ex Vivo Lentiviral Vector-Transduced Hematopoietic Stem and Progenitor Cells. [PDF]

Hum Gene Ther, 2022
Smith MC, Belur LR, Karlen AD, Erlanson O, Podetz-Pedersen KM, McKenzie J, Detellis J, Gagnidze K, Parsons G, Robinson N, Labarre S, Shah S, Furcich J, Lund TC, Tsai HC, McIvor RS, Bonner M.   +16 more
europepmc   +1 more source

Epidemiology of Mucopolysaccharidosis Type II According to the Register of the Russian Federation. [PDF]

Turk Arch Pediatr
Buchinskaia NV, Zakharova EY, Yulia S K, Anastasia O V, Skitchenko RK, Aleksandr M N, Kurilova VI, Maximova YV, Aksyanova KF, Bakulina EG, Kononenko NI, Osipova EV, Kostik MM, Kutsev SI.   +13 more
europepmc   +1 more source

Detailed pedigree analyses and prenatal diagnosis for a family with mucopolysaccharidosis type II. [PDF]

BMC Med Genomics, 2021
Zhang C, Hao S, Meng Z, Hui L, Wang Y, Xuan F, Chen X, Wang X, Zheng F, Zheng L, Zhou B, Wu X, Zhang Q, Cao Z.   +13 more
europepmc   +1 more source

Rescue of neurologic disease in mucopolysaccharidosis type II mice via AAV-mediated liver delivery of brain-penetrating iduronate-2-sulfatase. [PDF]

Neurotherapeutics
Jin X, Ye Q, Wu X, Su J, Song L, Fu J, Xu Q, Luo M, Liu F, Ren C, Hu M, Liu M, An Y, Li Q, Li M, Yang Y.   +15 more
europepmc   +1 more source

iPS-derived neural stem cells for disease modeling and evaluation of therapeutics for mucopolysaccharidosis type II. [PDF]

Exp Cell Res, 2022
Hong J, Cheng YS, Yang S, Swaroop M, Xu M, Beers J, Zou J, Huang W, Marugan JJ, Cai X, Zheng W.   +10 more
europepmc   +1 more source

A review of gene therapy in canine and feline models of lysosomal storage disorders [PDF]

, 2015
Bradbury, Allison M, Casal, Margret L, Gurda, Brittney L, Haskins, Mark E, Ponder, Katherine P, Vite, Charles H   +5 more
core   +2 more sources

Novel Phenotypic Insights into the <i>IDS</i> c.817C>T Variant in Mucopolysaccharidosis Type II from Newborn Screening Cohorts. [PDF]

Int J Neonatal Screen
Beauregard-Lacroix É, Menello C, Steffensen M, Lin HY, Chuang CK, Lin SP, Ficicioglu C.   +6 more
europepmc   +1 more source