Journal of Genetic Syndromes & Gene Therapy
Open Access
ISSN: ISSN: 2157-7412
ISSN: ISSN: 2157-7412
Ricki Lewis
Accepted Abstracts: J Genet Syndr Gene Ther
The idea for gene therapy was born in the minds of many in the years following Watson and Crick?s description of the genetic material and the unfolding of understanding gene action. The first gene therapy experiment was done in 1990, with some success, but the momentum suddenly ceased with the death of an 18-year-old in a clinical trial in 1999. Since then, despite other setbacks, the biotechnology that replaces a faulty gene has been reborn, thanks to results for several diseases that range from encouraging to spectacular. ?The Forever Fix: Gene Therapy and the Boy Who Saved It,? tells the tale of 8-year-old Corey Haas, who overcame certain blindness from Leber congenital amaurosis type 2 thanks to gene therapy, in the same city where the 18-year-old died, of a urea cycle disorder. The experiences of other families that gene therapy has touched provide the perspective to appreciate the technology?s comeback.
Ricki Lewis is a Ph.D. geneticist, journalist, professor and genetic counselor. The author of one of the most widely used college textbooks in the field (Human Genetics: Concepts and Applications, now in its 10th edition), she has also written hundreds of popular pieces on science and other topics for trade and specialized magazines, including Nature, Discover, and The Scientist.