Gene Therapy Disease Targets
The articles in this category describe the gene therapy approaches that have been adopted in an effort to treat genetic disorders. Genetic diseases are those that are passed down by our parents and which are the result of mutations in our genes. If a gene contains a mutation then the protein that it encodes will not form properly and will be unable to perform its allocated function thus resulting in a pthological situation and the formation of the disease.
In this section we discuss all the gene therapy strategies that have been conceived with a view to correcting these genetic defects in diseased cells.
Gene Therapy for Phenylketonuria
- Details
- Category: Genetic
- Published on Monday, 27 December 2010 17:09
- Written by Orestis Argyros and Suet-Ping Wong
- Hits: 4122
PKU is an autosomal recessive disorder resulting from a deficiency of the hepatic enzyme phenylalanine hydroxylase (PAH), which converts phenylalanine to tyrosine (figure 1) (1). PAH deficiency is the most common cause of the accumulation of phenylalanine (Phe), called hyperphenylalanemia (HPA), with an incidence of roughly 1:10000 Caucasian live births, with a higher incidence in the populations of Turkey, Ireland and Norway (http://emedicine.medscape.com/article/947781-overview).
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