Obviously, she said, the efficiency of the technique would need to be improved for it to have practical use in people. Even so, albinism is a rare disease. As for using the same approach to repair a gene involved with hair loss, Yoon said the common cases of age-related baldness involved many factors. The genetic role in typical cases, for example, of male-pattern baldness has not be worked out, she said. In the work, researchers inserted a sort of genetic Band-Aid to cells of the rats' hair follicles either by injection or by applying to the skin.
Hair follicles are clusters of cells that produce hair.
Traditional gene therapy aims to introduce a healthy gene sequence to supplement the function of a defective gene. Yoon and her colleagues have been developing another type of gene therapy, called gene repair, which corrects the original mutation in the gene sequence rather than introducing an entirely new gene. Gene repair uses short hairpin-shaped molecules made up of a corrective DNA sequence flanked by other genetic material to bind to a faulty DNA base in a gene. The repair insert fools cellular repair enzymes into fixing it into the defective gene. She said the repaired gene would be better regulated than genes that are added, thus preventing possible overproduction of gene products.