Baylor College of Medicine

Other

The United States of America As Represented by the Department of Health

Fred D Ledley

Bert W O'Malley

Edna Mozes

Leonard D Kohn

Susan June Henning

Dinah S Singer

Jocelyn Weissman

Motoyasu Saji

Giorgio Napolitano

Jozsef Stankovics

A complex for gene transfer a DNA molecule specifically and non-specifically bound to DNA binding protein. Additionally, it can include a chimeric compound for gene transfer. The chimeric compound has a DNA-binding element and a ligand binding element. The chimeric recombinant DNA can also include a binding protein which has a first element for binding to a receptor, a second element for binding to DNA, a third element for destabilizing endosomes and a fourth element for directing the traffic in a protein containing complex in the nucleus of a cell. The complex will be used for the treatment of a variety of diseases.

Natural or recombinant DNA binding proteins as carriers for gene transfer or gene therapy

The present invention provides methods for treating autoimmune diseases in mammals and for preventing or treating transplantation rejection in a transplant recipient. The methods of treatment involve the use of drugs capable of suppressing expression of MHC Class I molecules. In particular the use of the drug methimazole to suppress expression of MHC Class I molecules in the treatment of autoimmune diseases and the prevention or treatment of rejection in a transplant recipient is disclosed. In addition in vivo and in vitro assays are provided for the assessment and development of drugs capable of suppressing MHC Class I molecules.

Methods of treating autoimmune diseases and transplantation rejection

Method for in vivo introduction of a nucleic acid cassette into stem cells of intestinal epithelium. The nucleic acid cassette is introduced via vector solution. The vector solution can be delivered via the intestinal lumen in a variety of ways, including through an insertion device such as an endoscope, through catheters, through ligating and clamping the intestine after laparotomy or through slow release capsules. The vector solution once introduced into the intestinal epithelium is allowed to contact the stem cells for sufficient time for incorporation, usually between 1 and 48 hours. After sufficient incorporation, the insertion device and/or clamping and ligation procedure blockage are removed. Preferably, the procedure includes sufficient fluid to distend the intestine and provide additional access to the stem cells and the crypts. The procedure is useful in treating a variety of diseases including metabolic disorders, endocrine disorders, circulatory disorders, coagulation disorders, cancer, and gastrointestinal disease.

Gene therapy using the intestine

This invention relates to the transfer and expression of genes in cells associated with fluid spaces, such as follicles of the thyroid, the synovium of the joint, the vitreous of the eye and the inner or middle ear. Formulated DNA expression vectors comprising a gene are introduced with or without formulation elements directly into a fluid space under conditions in which the cells associated with the fluid space can incorporate the formulated DNA expression vector and express the transformed gene.

Tranformation of cells associated with fluid spaces

Gene transfer to the intestine

This invention relates to somatic gene therapy to cells associated with fluid spaces, such as follicles of the thyroid, the synovium of the joint, the vitreous of the eye and the inner or middle ear. Formulated DNA expression vectors are introduced with or without formulation elements into fluid spaces under conditions in which cells associated with the fluid space can incorporate the formulated DNA expression vector. Formulated DNA expression-mediated gene therapy allows treatment of diseases involving cells associated with fluid spaces.

Somatic gene therapy to cells associated with fluid spaces

Methods for assessing the ability of a candidate drug to suppress MHC

Growing community of inventors

Houston, TX, United States of America

Fred D Ledley