Sunday, May 2, 2010

Editorial in Molecular Therapy regarding Gene Therapy

The American Society for Gene & Cell Therapy publishes the journal Molecular Therapy under the auspices of Nature Publishing Group.
The Editorial in May 2010 issue by Dr. Derek Persons is titled "Lentiviral Vector Gene Therapy: Effective and Safe?"
The editor writes about the relative safety issues associated with γ-retroviral or lentiviral vector therapy to treat certain clinical disporders.
He also draws attention to the successful treatment of two X-ALD patients reported by Cartier et al. in Science last year "Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy". X-linked adrenoleukodystrophy is a genetic disorder involving defects in ABCD1 gene which results into deficiency of ATP binding cassette transporter in peroxisomal membrane. This paper was not only of significance to peroxisome field but in general a breakthrough treatment using gene therapy.

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