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

The United States of America, as Represented by the Secretary, Department of Health and Human Services

The United States of America as Represented by the Secretary of

Children's Hospital Los Angeles

R Michael Blaese

Steven A Rosenberg

W French Anderson

Richard A Hess

Craig A Mullen

Donald B Kohn

Kenneth W Culver

Robert C Moen

Christopher D Stone

William J Ramsey

Kleanthis G Xanthopoulos

Primary human cells which are genetically engineered with DNA (RNA) encoding a marker or therapeutic which is expressed to be expressed in vivo. Such engineered cells may be used in gene therapy.

Gene therapy

A method of providing a therapeutic effect in a human patient which comprises administering to the patient CD34+ cells obtained from cord blood. The CD34+ cells have been engineered with at least one nucleic acid sequence encoding a therapeutic agent. Such CD34+ cells may be engineered by transducing the cells with a retroviral vector including the nucleic acid sequence encoding the therapeutic agent. This method has been applied in treating newborn infants suffering from ADA deficiency.

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The present invention provides chimeric primary viruses, which are capable of producing secondary viruses in a producer host cell, and methods of making the same.

Transgenomic viruses

A method for treating an tumor involves the initial identification of the tumor as one displaying a 'bystander effect,' whereby in vivo transfer of a gene conferring sensitivity to chemotherapeutic agent affects both transformed and non-transformed tumor cells. Into a tumor thus characterized is introduced in situ a retroviral vector containing the sensitizing gene. The retroviral vector, which may replication-defective or replication-competent, can be introduced directly (if it is replication-competent) or can be provided by means of a packaging cell line. Treatment of the patient with the chemotherapeutic agent thereafter effects tumor regression when as few as 10% of tumor cells are transformed, while normal tissue is not damaged. The anti-tumor impact of the treatment can be increased when the transducing vector also encodes an immune response-enhance substance such as IL-2 or another cytokine.

Bystander effect tumoricidal therapy by expressing an HSV-tk gene

The present invention relates to a system comprising a modified bacterial gene for cytosine deaminase that has been engineered into a eukaryotic expression vector and the expression of the gene by mammalian cells. The present invention further relates to methods, gene therapies and vaccines that employ the negative selectable marker, cytosine deaminase, which has the ability to produce a toxic antimetabolic 5-fluorouracil from 5-fluorocytosine.

Cytosine deaminase negative selection system for gene transfer

The present invention relates to a system comprising a modified bacterial gene for cytosine deaminase that has been engineered into a eukaryotic expression vector and the expression of the gene by mammalian cells. The present invention further relates to methods, gene therapies and vaccines that employ the negative selectable marker, cytosine deaminase, which has the ability to produce a toxic antimetabolite 5-fluorouracil from 5-fluorocytosine.

The invention is a method of modulating or controlling autoimmune disease in a host. The method includes the step of administering to the diseased host a pharmaceutical effective amount of succinylacetone. The pharmaceutically effective amount of succinylacetone is desirably infused by an osmotic minipump in order to modulate the effects of autoimmune disease in the host.

Method for treating autoimmune disease using succinylacetone

A method is disclosed for preventing graft rejection of transplanted solid organs, in mammal recipients thereof, by administering an effective graft rejection preventative amount of succinylacetone to said mammals.

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Rockville, MD, United States of America

R Michael Blaese