Gene Therapy — Promise, Pitfalls, and Prognosis
- 28 September 1995
- journal article
- Published by Massachusetts Medical Society in New England Journal of Medicine
- Vol. 333 (13) , 871-873
- https://doi.org/10.1056/nejm199509283331310
Abstract
The application of molecular genetics to human biology has had a profound effect on our ability to understand, diagnose, and treat a variety of diseases. The identification and cloning of a number of disease-related genes has provided us with a powerful set of tools for identifying susceptibility to disease and, more important, understanding the molecular pathophysiology of many disorders. More recently, the revolution in molecular medicine has also generated considerable enthusiasm for gene therapy.Somatic gene therapy is the introduction and expression of recombinant genes in somatic cells for the purpose of treating a disease. Although barely five years old, . . .Keywords
This publication has 12 references indexed in Scilit:
- A Controlled Study of Adenoviral-Vector–Mediated Gene Transfer in the Nasal Epithelium of Patients with Cystic FibrosisNew England Journal of Medicine, 1995
- Cytostatic Gene Therapy for Vascular Proliferative Disorders with a Constitutively Active form of the Retinoblastoma Gene ProductScience, 1995
- In vivo suppression of injury-induced vascular smooth muscle cell accumulation using adenovirus-mediated transfer of the herpes simplex virus thymidine kinase gene.Proceedings of the National Academy of Sciences, 1994
- Inefficient gene transfer by adenovirus vector to cystic fibrosis airway epithelia of mice and humansNature, 1994
- Gene Therapy for Vascular Smooth Muscle Cell Proliferation After Arterial InjuryScience, 1994
- MHC class I-cestricted cytotoxic T lymphocytes to viral antigens destroy hepatocytes in mice infected with E1-deleted recombinant adenovirusesImmunity, 1994
- Inactivation of E2a in recombinant adenoviruses improves the prospect for gene therapy in cystic fibrosisNature Genetics, 1994
- Heterologous Protection Against Influenza by Injection of DNA Encoding a Viral ProteinScience, 1993
- Direct Gene Transfer into Mouse Muscle in VivoScience, 1990
- Conversion of mdx myofibres from dystrophin-negative to -positive by injection of normal myoblastsNature, 1989