Genetic correction and analysis of induced pluripotent stem cells from a patient with gyrate atrophy
نویسندگان
چکیده
منابع مشابه
Genetic correction and analysis of induced pluripotent stem cells from a patient with gyrate atrophy.
Gene-corrected patient-specific induced pluripotent stem (iPS) cells offer a unique approach to gene therapy. Here, we begin to assess whether the mutational load acquired during gene correction of iPS cells is compatible with use in the treatment of genetic causes of retinal degenerative disease. We isolated iPS cells free of transgene sequences from a patient with gyrate atrophy caused by a p...
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Regenerative capacity of mammals is limited and can rarely regenerate a specific organ or tissue fully. Due to these limitations, regenerative medicine seeks efficient and safe cell sources for regeneration of damaged tissues and organs or treatment for incurable diseases. Human embryonic stem cells (HESCs) hold two important properties called self renewal and pluripotency. However, the use of ...
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HAX1 was identified as the gene responsible for the autosomal recessive type of severe congenital neutropenia. However, the connection between mutations in the HAX1 gene and defective granulopoiesis in this disease has remained unclear, mainly due to the lack of a useful experimental model for this disease. In this study, we generated induced pluripotent stem cell lines from a patient presentin...
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ژورنال
عنوان ژورنال: Proceedings of the National Academy of Sciences
سال: 2011
ISSN: 0027-8424,1091-6490
DOI: 10.1073/pnas.1103388108