Gene Therapy

Scientists at Semmelweis University, Medical Department release new data on gene therapy

Published in Gene Therapy Weekly, June 26th, 2008

According to a study from Budapest, Hungary, "Von Hippel-Lindau disease (VHL) is a rare autosomal dominant disease characterized by development of cystic and tumorous lesions at multiple sites, including the brain, spinal cord, kidneys, adrenals, pancreas, epididymis and eyes. The clinical phenotype results from molecular abnormalities of the VHL tumor suppressor gene, mapped to human chromosome 3p25-26."

"The VHL gene encodes two functionally active VHL proteins due to the presence of two translational initiation sites separated by 53 codons. The majority of disease-causing mutations have been detected downstream of the second translational initiation site, but there...

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Source: Gene Therapy Weekly (2008-06-26)

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