scholarly journals Mutation analysis and clinical characterization of Iranian patients with mucopolysaccharidosis type I

2019 ◽  
Vol 33 (8) ◽  
Author(s):  
Mohammad Taghikhani ◽  
Shohreh Khatami ◽  
Mohammad Abdi ◽  
Mohammad Said Hakhamaneshi ◽  
Mohammad Reza Alaei ◽  
...  
Keyword(s):  
Mutation Analysis ◽  
Type I ◽  
Mucopolysaccharidosis Type ◽  
Mucopolysaccharidosis Type I ◽  
Iranian Patients

scholarly journals Characterization of an immunodeficient mouse model of mucopolysaccharidosis type I suitable for preclinical testing of human stem cell and gene therapy

2007 ◽  
Vol 74 (6) ◽  
pp. 429-438 ◽  
Author(s):  
Mayra F. Garcia-Rivera ◽  
Leah E. Colvin-Wanshura ◽  
Matthew S. Nelson ◽  
Zhenhong Nan ◽  
Shaukat A. Khan ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Mouse Model ◽  
Type I ◽  
Mucopolysaccharidosis Type ◽  
Mucopolysaccharidosis Type I ◽  
Human Stem Cell ◽  
Preclinical Testing ◽  
Cell And Gene Therapy

Mutation analysis of 19 North American mucopolysaccharidosis type I patients: Identification of two additional frequent mutations

1994 ◽  
Vol 3 (3) ◽  
pp. 275-282 ◽  
Author(s):  
Lorne A. Clarke ◽  
Paul V. Nelson ◽  
Cara L. Warrington ◽  
C. Phillip Morris ◽  
John J. Hopwood ◽  
...  
Keyword(s):  
Mutation Analysis ◽  
North American ◽  
Type I ◽  
Mucopolysaccharidosis Type ◽  
Mucopolysaccharidosis Type I ◽  
Frequent Mutations
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