Overview of Gene and Cell Therapeutics

The most common strategy is to use engineered nucleic acids that can be delivered to target cells and express functional protein. This strategy is discussed further in Section 15.2. 

More than 5000 monogenic disorders have been linked to DNA alteration at a specific gene locus. Cell and gene therapy, although conceived primarily as a means to correct monogenic disorders, is now recog- 

Biotechnology and Biopharmaceuticals, by Rodney J. Y. Ho and Milo Gibaldi ISBN 0-471-20690-3 Copyright 2003 by John Wiley Sons, Inc. 

ITABLE 15.1. Medical conditions that could benefit from cell and gene therapy 

Phenylketourea Gancher disease Viral hepatitis (i.e., hepatitis B and C) Neurological disorders 

---