Gene-based medicines and cellular therapy for disease

Abstract

Herein disclosed are compositions comprising synthetic chromosomes and methods of their use to treat diseases and disorders (e.g., cancers, genetic and autoimmune diseases). Specifically described are methods of constructing synthetic chromosome compositions bearing one or multiple genes as well as regulatory sequences that control expression of the gene(s) such that, when these are expressed from the synthetic chromosome in an animal cell, at least one medicinal gene product is reliably, faithfully and indefinitely produced by the animal cells. As an example, the presently disclosed compositions and methods are used to bioengineer cells to enable them to express the entire dystrophin gene and additional regulatory nucleic acid sequences under tightly controlled conditions, allowing the present methods and compositions to be used as cellular medicines for treatment of diseases such as muscular dystrophies.

Dependencies are inferred from the claim text.