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Lentiviral Gene Therapy for Mucopolysaccharidosis II with Tagged Iduronate 2-Sulfatase Prevents Life-Threatening Pathology in Peripheral Tissues But Fails to Correct Cartilage.
Hum Gene Ther. 2024 Apr;35(7-8):256-268. doi: 10.1089/hum.2023.177. Epub 2024 Feb 2.
Hum Gene Ther. 2024.
PMID: 38085235
Free PMC article.
Tagged IDS causes efficient and engraftment-independent prevention of brain pathology during lentiviral gene therapy for Mucopolysaccharidosis type II.
Catalano F, Vlaar EC, Katsavelis D, Dammou Z, Huizer TF, van den Bosch JC, Hoogeveen-Westerveld M, van den Hout HJMP, Oussoren E, Ruijter GJG, Schaaf G, Pike-Overzet K, Staal FJT, van der Ploeg AT, Pijnappel WWMP.
Catalano F, et al. Among authors: van den hout hjmp.
Mol Ther Methods Clin Dev. 2023 Nov 2;31:101149. doi: 10.1016/j.omtm.2023.101149. eCollection 2023 Dec 14.
Mol Ther Methods Clin Dev. 2023.
PMID: 38033460
Free PMC article.
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Broad variation in phenotypes for common GAA genotypes in Pompe disease.
Niño MY, In't Groen SLM, de Faria DOS, Hoogeveen-Westerveld M, van den Hout HJMP, van der Ploeg AT, Bergsma AJ, Pijnappel WWMP.
Niño MY, et al. Among authors: van den hout hjmp.
Hum Mutat. 2021 Nov;42(11):1461-1472. doi: 10.1002/humu.24272. Epub 2021 Sep 8.
Hum Mutat. 2021.
PMID: 34405923
Free PMC article.
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A Generic Assay to Detect Aberrant ARSB Splicing and mRNA Degradation for the Molecular Diagnosis of MPS VI.
Broeders M, Smits K, Goynuk B, Oussoren E, van den Hout HJMP, Bergsma AJ, van der Ploeg AT, Pijnappel WWMP.
Broeders M, et al. Among authors: van den hout hjmp.
Mol Ther Methods Clin Dev. 2020 Sep 16;19:174-185. doi: 10.1016/j.omtm.2020.09.004. eCollection 2020 Dec 11.
Mol Ther Methods Clin Dev. 2020.
PMID: 33209960
Free PMC article.
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A genetic modifier of symptom onset in Pompe disease.
Bergsma AJ, In 't Groen SLM, van den Dorpel JJA, van den Hout HJMP, van der Beek NAME, Schoser B, Toscano A, Musumeci O, Bembi B, Dardis A, Morrone A, Tummolo A, Pasquini E, van der Ploeg AT, Pijnappel WWMP.
Bergsma AJ, et al. Among authors: van den hout hjmp.
EBioMedicine. 2019 May;43:553-561. doi: 10.1016/j.ebiom.2019.03.048. Epub 2019 Mar 25.
EBioMedicine. 2019.
PMID: 30922962
Free PMC article.
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Morphological changes in muscle tissue of patients with infantile Pompe's disease receiving enzyme replacement therapy.
Winkel LP, Kamphoven JH, van den Hout HJ, Severijnen LA, van Doorn PA, Reuser AJ, van der Ploeg AT.
Winkel LP, et al.
Muscle Nerve. 2003 Jun;27(6):743-51. doi: 10.1002/mus.10381.
Muscle Nerve. 2003.
PMID: 12766987
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Anesthesiological approach to the floppy child.
Tesoro S, DE Robertis E, Marturano F, VAN DEN Hout HJ, DE Graaff JC.
Tesoro S, et al.
Minerva Anestesiol. 2021 Aug;87(8):940-949. doi: 10.23736/S0375-9393.20.15011-9. Epub 2021 Jan 12.
Minerva Anestesiol. 2021.
PMID: 33432795
Free article.
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