Gene Therapy

Research from Massachusetts General Hospital provides new data on gene therapy

Published in Gene Therapy Weekly, March 26th, 2009

According to recent research from the United States, "Familial dysautonomia (FD), a devastating hereditary sensory and autonomic neuropathy, results from an intronic mutation in the IKBKAP gene that disrupts normal mRNA splicing and leads to tissue-specific reduction of IKBKAP protein (IKAP) in the nervous system. To better understand the roles of IKAP in vivo, an Ikbkap knockout mouse model was created."

"Results from our study show that ablating Ikbkap leads to embryonic lethality, with no homozygous Ikbkap knockout (Ikbkap(-/-)) embryos surviving beyond 12.5 days postcoitum. Morphological analyses of the Ikbkap(-/-) conceptus at different stages revealed...

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Source: Gene Therapy Weekly (2009-03-26)

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