%0 Journal Article
%T Musings on genome medicine: gene therapy
%A David G Nathan
%A Stuart H Orkin
%J Genome Medicine
%D 2009
%I BioMed Central
%R 10.1186/gm38
%X Discoveries of restriction enzymes by Hamilton Smith and Daniel Nathans [1], and reverse transcriptase by Howard Temin [2] and David Baltimore [3] in the 1970s set the stage for an explosion in molecular and clinical genetics. Armed with a sufficient amount of the mRNA product of a gene such as the beta globin gene, investigators could now produce a radioactive DNA copy (cDNA) of the mRNA and use it as a valuable probe of gene expression. Moreover, the globin cDNA was functional. Cloned into an expression plasmid [4] and transduced into an appropriate cell, the cDNA would produce mature beta globin mRNA, and cDNAs could be manipulated at will in what became the era of recombinant DNA technology.The new discoveries were greeted with enthusiasm by most biologists and clinical investigators but with mounting horror and suspicion by many members of the public and their elected officials, as well as some academics. Frankensteins were thought to be loose in biomedical laboratories; monsters would be created; plagues of vicious E. coli would be loosed on an innocent population; mad scientists would forever contaminate the food supply; the new genetics would lead to a resurgence of social Darwinism. The specter of Nazi medicine roiled some faculty meetings and communities in which the science was rapidly advancing. Rules and restrictions were demanded that would deliberately inhibit the work. To their credit, leaders of this new genetics revolution met in Asilomar California [5], where they established laboratory standards intended to reassure themselves, their colleagues, and the general population that gene manipulation could be rendered safe and useful.While eager clinical investigators hoped to apply the new genetics in the treatment of inherited diseases, cooler heads recognized that the technology was not sufficiently powerful or predictable. The more cautious advised the National Institutes of Health to be very wary of human application because the biological 'rules
%U http://genomemedicine.com/content/1/4/38