Thomas Jefferson University

University of Pittsburgh

Roger Williams Medical Center

Mark A Zern

Leaf Huang

Roger J Pomerantz

Lingxun Duan

Tony Yoa

Methods of gene therapy, particular ribozyme-mediated gene replacement methods, are disclosed. Method of treating patients suffering from a disease associated with expression of an abnormal form of a gene, such as alpha-1 antitrypsin mutations, are disclosed. The methods comprise the steps of administering to such a patient a nucleic acid construct encoding a ribozyme and a nucleic acid construct comprising a ribozyme resistant gene encoding a wild type form of the gene product. Recombinant vectors and pharmaceutical compositions for practicing the methods are disclosed.

Ribozyme-mediated gene replacement

Liposome carrier systems, methods and pharmaceutical compositions that target an organ preferentially with high concentrations of at least one therapeutic agent utilize a liposome carrier having a lipid membrane and an aqueous space to deliver pharmacologically active agents, such as free radical scavengers and antioxidants, to a target organ such as the liver. At least one free radical scavenger and/or antioxidant and a liposome carrier may be provided to a liver donor prior to harvesting to preserve the liver for transplantation.

Treatment of disease states induced by oxidative stress

Growing community of inventors

Newtown, PA, United States of America

Mark A Zern