Abstract
Gene therapy is a modality being studied for many diseases. Although most of the clinical applications are being considered for malignant disease, the origins of gene therapy lie in the inherited disease of metabolism. Gaucher disease serves as a prototype for the study of gene therapy for genetic disorders.
| Original language | English (US) |
|---|---|
| Pages (from-to) | 5-9 |
| Number of pages | 5 |
| Journal | International Pediatrics |
| Volume | 10 |
| Issue number | 1 |
| State | Published - Jan 1 1995 |
| Externally published | Yes |