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1.
Usefulness of antibody-drug conjugate as preconditioning for hematopoietic stem cell-targeted gene therapy in wild-type and Fabry disease mouse models.
Mol Genet Metab
; 142(3): 108494, 2024 May 15.
Artículo
en Inglés
| MEDLINE | ID: mdl-38820907
2.
A novel preclinical model of mucopolysaccharidosis type II for developing human hematopoietic stem cell gene therapy.
Gene Ther
; 30(3-4): 288-296, 2023 04.
Artículo
en Inglés
| MEDLINE | ID: mdl-35835952
3.
Suppression of lysosomal acid alpha-glucosidase impacts the modulation of transcription factor EB translocation in pancreatic cancer.
Cancer Sci
; 112(6): 2335-2348, 2021 Jun.
Artículo
en Inglés
| MEDLINE | ID: mdl-33931930
4.
Inhibition of acid ceramidase elicits mitochondrial dysfunction and oxidative stress in pancreatic cancer cells.
Cancer Sci
; 112(11): 4570-4579, 2021 Nov.
Artículo
en Inglés
| MEDLINE | ID: mdl-34459070
5.
Efficient engraftment of genetically modified cells is necessary to ameliorate central nervous system involvement of murine model of mucopolysaccharidosis type II by hematopoietic stem cell targeted gene therapy.
Mol Genet Metab
; 130(4): 262-273, 2020 08.
Artículo
en Inglés
| MEDLINE | ID: mdl-32631737
6.
Long-term efficacy and safety of migalastat treatment in Fabry disease: 30-month results from the open-label extension of the randomized, phase 3 ATTRACT study.
Mol Genet Metab
; 131(1-2): 219-228, 2020.
Artículo
en Inglés
| MEDLINE | ID: mdl-33012654
7.
Efficacy and safety of migalastat in a Japanese population: a subgroup analysis of the ATTRACT study.
Clin Exp Nephrol
; 24(2): 157-166, 2020 Feb.
Artículo
en Inglés
| MEDLINE | ID: mdl-31889231
8.
Biallelic IARS Mutations Cause Growth Retardation with Prenatal Onset, Intellectual Disability, Muscular Hypotonia, and Infantile Hepatopathy.
Am J Hum Genet
; 99(2): 414-22, 2016 Aug 04.
Artículo
en Inglés
| MEDLINE | ID: mdl-27426735
9.
Gene therapy for lysosomal storage diseases and peroxisomal diseases.
J Hum Genet
; 64(2): 139-143, 2019 Feb.
Artículo
en Inglés
| MEDLINE | ID: mdl-30498239
10.
Mutation spectrum of α-Galactosidase gene in Japanese patients with Fabry disease.
J Hum Genet
; 64(7): 695-699, 2019 Jul.
Artículo
en Inglés
| MEDLINE | ID: mdl-30988410
11.
Chaperone effect of sulfated disaccharide from heparin on mutant iduronate-2-sulfatase in mucopolysaccharidosis type II.
Mol Genet Metab
; 123(2): 118-122, 2018 02.
Artículo
en Inglés
| MEDLINE | ID: mdl-29289480
12.
The beneficial effects of long-term enzyme replacement therapy on cardiac involvement in Japanese Fabry patients.
Mol Genet Metab
; 124(2): 143-151, 2018 06.
Artículo
en Inglés
| MEDLINE | ID: mdl-29747997
13.
Oral pharmacological chaperone migalastat compared with enzyme replacement therapy in Fabry disease: 18-month results from the randomised phase III ATTRACT study.
J Med Genet
; 54(4): 288-296, 2017 04.
Artículo
en Inglés
| MEDLINE | ID: mdl-27834756
14.
Anti-BlyS antibody reduces the immune reaction against enzyme and enhances the efficacy of enzyme replacement therapy in Fabry disease model mice.
Clin Immunol
; 178: 56-63, 2017 05.
Artículo
en Inglés
| MEDLINE | ID: mdl-28161408
15.
The production of human glucocerebrosidase in glyco-engineered Nicotiana benthamiana plants.
Plant Biotechnol J
; 14(8): 1682-94, 2016 08.
Artículo
en Inglés
| MEDLINE | ID: mdl-26868756
16.
A method for measuring disease-specific iduronic acid from the non-reducing end of glycosaminoglycan in mucopolysaccharidosis type II mice.
Mol Genet Metab
; 117(2): 140-3, 2016 Feb.
Artículo
en Inglés
| MEDLINE | ID: mdl-26051019
17.
Non-myeloablative preconditioning with ACK2 (anti-c-kit antibody) is efficient in bone marrow transplantation for murine models of mucopolysaccharidosis type II.
Mol Genet Metab
; 119(3): 232-238, 2016 11.
Artículo
en Inglés
| MEDLINE | ID: mdl-27590924
18.
New treatment strategy with nuclear factor-κB inhibitor for pancreatic cancer.
J Surg Res
; 206(1): 1-8, 2016 11.
Artículo
en Inglés
| MEDLINE | ID: mdl-27916347
19.
Hematopoietic stem cell transplantation for inborn errors of metabolism: A report from the Research Committee on Transplantation for Inborn Errors of Metabolism of the Japanese Ministry of Health, Labour and Welfare and the Working Group of the Japan Society for Hematopoietic Cell Transplantation.
Pediatr Transplant
; 20(2): 203-14, 2016 Mar.
Artículo
en Inglés
| MEDLINE | ID: mdl-26806759
20.
Characteristics of Cerebral Microbleeds in Patients with Fabry Disease.
J Stroke Cerebrovasc Dis
; 25(6): 1320-5, 2016 Jun.
Artículo
en Inglés
| MEDLINE | ID: mdl-26987491