Somatic cell gene therapy

Abstract

Abstract The present invention is a somatic cell gene therapy method that is especially useful for the treatment of certain diseases that are caused by gene defects. According to the invention, fibroblast cells are transduced so that they express a 'replacement' gene of interest. These transduced fibroblasts are preferably fixed in vitro in an extracellular matrix, and then implanted in the loose connective tissue of the skin of an individual or animal to be treated. Because the fibroblasts are implanted in a highly vascularized compartment of the skin i.e., loose connective tissue of the dermis, the transduced cells, and thus their 'replacement' gene products, have direct access to the circulatory system. As a result the needed replacement gene products can easily and efficiently be distributed to other parts of the body. When the gene therapy is no longer needed, the implanted fibroblasts can be conveniently removed.

Inventors

• 👤 Inder Mohan Verma
• 👤 Daniel Claude St. Louis

Bibliographic Details