Construction of a high efficiency retroviral vector for gene therapy of Hunter's syndrome
Y Hong, SS Yu, JM Kim, K Lee, YS Na… - The Journal of Gene …, 2003 - Wiley Online Library
Background As an alternative method to the conventional therapies for Hunter's syndrome,
which is a lethal lysosomal storage disorder, we have developed gene delivery vehicles …
which is a lethal lysosomal storage disorder, we have developed gene delivery vehicles …
Biodistribution and efficacy of donor T lymphocytes in a murine model of lysosomal storage disease
PP Young, C Vogler, AA Hofling, MS Sands - Molecular Therapy, 2003 - cell.com
Lymphocyte-directed gene transfer has been proposed as potential therapy to treat certain
congenital immunological deficiencies as well as other genetic diseases such as lysosomal …
congenital immunological deficiencies as well as other genetic diseases such as lysosomal …