University of California

Other

Leland Stanford Junior University

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

Ucl Business Plc

Children's Hospital Los Angeles

Donald B Kohn

Roger Paul Hollis

R Michael Blaese

Adrian Thrasher

Maria Grazia Roncarolo

Bobby Gaspar

Rosa Bacchetta

Robert C Moen

Katelyn E Masiuk

Richard A Morgan

Craig A Mullen

Pia M Challita

Carmen Flores Bjurstroem

In various embodiments, lentiviral vectors expressing FoxP3 using the endogenous gene elements to regulate physiologic gene expression of FoxP3 are provided herein as well as uses of such vectors.

Self-inactivating lentiviral vector comprising a FOXP3 expression cassette

In certain embodiments an optimized derivative of the CCLc-βAS3-FB lentiviral vector termed (CCLc-mGata/ANK-CoreLCR-βAS3-FB), is provided which is capable of driving lineage-restricted expression of a beta-globin gene (e.g., an anti-sickling β-globin like gene (βA83)). In certain embodiments the vectors described herein comprise novel defined LCR HS core sequences (HS2(˜420 bp), HS3β40 bp), HS4(˜410 bp)) which can be used to replace the putative LCR HS sequences present within the 'mini-LCR' (˜3.6 kb reduced to ˜1.2 kb) to produce an “optimized mini-LCR”.

Optimized lentiviral vector for stem cell gene therapy of hemoglobinopathies

The present invention relates to the prevention and/or treatment of ADA-SCID, in a patient.

Method for treating adenosine deaminase severe combined imunodeficiency

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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Retroviral plasmid vectors which include an enhancer region obtained from a virus selected from the group consisting of Myeloproliferative Sarcoma Virus and polyoma virus, and a primer binding site obtained from murine retrovirus. The retroviral plasmid vector does not include a negative control region. The vector also may include a nucleic acid sequence which encodes demethylation of cytosine residues in the proviral LTR. Such plasmid vectors are especially useful in the generation of retroviral vector particles which may be expressed in embryonic cells, such as embryonic stem cells.

Retroviral vectors for expression in embryonic cells

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Tarzana, CA, United States of America

Donald B Kohn