Gene Therapy

Research results from University of Modena update knowledge of gene therapy

Published in Gene Therapy Weekly, February 12th, 2009

"Deficiency of the basement membrane component laminin-5 (LAM5) causes junctional epidermolysis bullosa (JEB), a severe and often fatal skin adhesion defect. Autologous transplantation of epidermal stem cells genetically corrected with a Moloney leukemia virus (MLV)-derived retroviral vector reconstitutes LAM5 synthesis, and corrects the adhesion defect in JEB patients," researchers in Modena, Italy report.

"However, MLV-derived vectors have genotoxic characteristics, and are unable to reproduce the physiological, basal layer -restricted expression of LAM5 chains. We have developed an alternative gene transfer strategy based on self-inactivating (SIN) or long terminal...

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Source: Gene Therapy Weekly (2009-02-12)

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