Critical assessment of lifelong phenotype correction in hyperbilirubinemic Gunn rats after retroviral mediated gene transfer
نویسندگان
چکیده
منابع مشابه
Therapeutic lentivirus-mediated neonatal in vivo gene therapy in hyperbilirubinemic Gunn rats.
Crigler-Najjar type 1 disease (CN-1) is a genetic disorder characterized by high levels of unconjugated bilirubin due to the absence of hepatic UDPglucuronosyltransferase (UGT1) activity. Here we show that in vivo neonatal hepatocyte transduction with a lentiviral vector expressing the defective enzyme resulted in long-term correction in Gunn rats, a model of CN-1. Lentiviral vectors harboring ...
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Gunn rats, a mutant strain of rats, suffer from autosomal recessive hyperbilirubinemia. The homozygotes (j/j) develop jaundice soon after birth and often exhibit kernicterus and cerebellar hypoplasia that are due to bilirubin. Therefore, j/j Gunn rats have been used as an animal model of bilirubin encephalopathy, as well as of neonatal hyperbilirubinemia. In this review, we discuss morphologica...
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Gene transfer with vectors derived from murine retroviruses is restricted to cells which are proliferating and synthesizing DNA at the time of infection. This suggests that retroviral-mediated gene transfer might permit targeting of gene integration into malignant cells in organs composed mainly of quiescent nonproliferating cells, such as in the brain. Accordingly, selective introduction of ge...
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Gene transfer with vectors derived from murine retroviruses is re stricted to cells which are proliferating and synthesizing DNA at the time of infection. This suggests that retroviral-mediated gene transfer might permit targeting of gene integration into malignant cells in organs com posed mainly of quiescent nonproliferating cells, such as in the brain. Accordingly, selective introduction of ...
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X-linked severe combined immunodeficiency (XSCID) is a lethal disease caused by a defect in the gene encoding the common gamma chain (gamma-c) of the receptor for interleukin-2 (IL-2), IL-4, IL-7, IL-9, and IL-15. Allogeneic bone marrow transplantation, the current therapy of choice for this defect, is often complicated by graft-versus-host disease and/or incomplete reconstitution of B-lymphocy...
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ژورنال
عنوان ژورنال: Gene Therapy
سال: 2007
ISSN: 0969-7128,1476-5462
DOI: 10.1038/sj.gt.3302993