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M odern molecular genetics has revolutionized medical research by providing approaches to determine the location of genes that cause disease, even in the absence of knowledge about the underlying biological defects. These new approaches, when combined with innovative techniques derived from such traditional disciplines as physiology and biochemistry, will have far-reaching effects. Potential benefits include improved methods for presymptomatic diagnosis, carrier detection, primary prevention, and treatment. The National Heart, Lung, and Blood Institute (NHLBI) has an established record of encouraging and supporting the development of innovative genetic approaches to study monogenic disorders, including cystic fibrosis, familial hypertrophic cardiomyopathy, hemophilia, and sickle cell disease. While continuing its commitment to stimulate and support such efforts, the institute is placing a major emphasis on the use of existing knowledge to devise effective methods of genetic therapy for these diseases. One manifestation of that emphasis is the Working Group on Gene Therapy Approaches and Resources for Heart, Lung, and Blood Diseases, convened by the institute in 1992. Its charge was to identify new opportunities and chart a course for future research efforts. The working group recognized that several clinical protocols involving gene transfer were already approved and generating data, but they noted the existence of barriers to the further evolution of human gene therapy. Although genetic interventions for heart, lung, and blood diseases may differ in the specifics of the approach, the working group identified common themes in scientific issues and resources fundamental to facilitating development of gene therapy for these diseases. Two planned NHLBI programs already reflect the influence of the working group's report. Gene Therapy Approaches for Cystic Fibrosis aind Other Heart, Lung, and Blood Diseases will promote collaboration within and between research institutions, develop infrastructural resources, and foster the entry of both young and established investigators into the field of gene therapy research. The other, Gene Therapy for Sickle Cell Disease, is directed toward developing strategies to correct or replace the endogenous, defective gene in sickle cell anemia.

Molecular genetics of heart, lung, and blood diseases. The shape of things to come.