Article
BTK gene targeting by homologous recombination using a helper-dependent adenovirus/adeno-associated virus hybrid vector.
Gene therapy - 1 Feb 2016
Yamamoto H, Ishimura M, Ochiai M, Takada H, Kusuhara K, Nakatsu Y, Tsuzuki T, Mitani K, Hara T
Abstract excerpt
X-linked agammaglobulinemia (XLA) is one of the most common humoral immunodeficiencies, which is caused by mutations in Bruton's tyrosine kinase (BTK) gene. To examine the possibility of using gene therapy for XLA, we constructed a helper-dependent adenovirus/adeno-associated virus BTK targeting vector (HD-Ad.AAV BTK vector) composed of a genomic sequence containing BTK exons 6-19 and a green fluorescence...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.