Results 111 to 120 of about 11,933,855 (190)

Skewed X-Chromosome Inactivation in a Korean Girl with Severe Mucopolysaccharidosis Type II. [PDF]

Ann Lab Med, 2022
Kim HY, Kim MJ, Seong MW, Ko JM.
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

Corrigendum to "iPS-derived neural stem cells for disease modeling and evaluation of therapeutics for mucopolysaccharidosis type II" [Exp. Cell Res. 412, Issue 1, 1 March 2022, 113007]. [PDF]

Exp Cell Res, 2023
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

Early axon guidance and synapse maturation defects in a zebrafish model of Mucopolysaccharidosis type II [PDF]

, 2022
Enrico Moro, Lorenzo Badenetti, Rosella Tomanin, Rosa Manzoli   +3 more
core  

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

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

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

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

Newborn Screening Program for Mucopolysaccharidosis Type II and Long-Term Follow-Up of the Screen-Positive Subjects in Taiwan. [PDF]

J Pers Med, 2022
Lin HY, Chang YH, Lee CL, Tu YR, Lo YT, Hung PW, Niu DM, Liu MY, Liu HY, Chen HJ, Kao SM, Wang LY, Ho HJ, Chuang CK, Lin SP.   +14 more
europepmc   +1 more source

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