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

Ke Liu

Wei Ping Zheng

Juan Jose Marugan

Noel Terrence Southall

Christopher P Austin

Steven A Rosenberg

Wei Zheng

This disclosure relates generally to the treatment of lysosomal storage disorders. Specifically, the disclosure relates to a novel use of &#x3b4; tocopherol in the treatment of diseases and conditions related to lysosomal storage disorders. Included in the present disclosure is a method for the modulation of cholesterol recycling. Further, the disclosure relates to conditions such as Niemann-Pick type C disease, Farber disease, Niemann-Pick type A disease, Wolman disease and Tay Sachs disease. Further included in the present disclosure is a method for treating lysosomal storage disorders comprising the administration of &#x3b4; tocopherol. Further included in the present disclosure is a method for treating lysosomal storage disorders comprising the administration of &#x3b4; tocopherol in combination with cyclodextrin to a patient in need thereof.

Use of delta tocopherol for the treatment of lysosomal storage disorders

This disclosure relates generally to the treatment of lysosomal storage disorders. Specifically, the disclosure relates to a novel use of delta tocopherol in the treatment of diseases and conditions related to lysosomal storage disorders. Included in the present disclosure is a method for the modulation of cholesterol recycling. Further, the disclosure relates to conditions such as Niemann-Pick type C disease, Farber disease, Niemann-Pick type A disease, Wolman disease and Tay Sachs disease. Further included in the present disclosure is a method for treating lysosomal storage disorders comprising the administration of delta tocopherol. Further included in the present disclosure is a method for treating lysosomal storage disorders comprising the administration of delta tocopherol in combination with cyclodextrin to a patient in need thereof.

The invention provides methods of preparing autologous T-lymphocytes for re-introduction into a patient having cancer, which method comprises obtaining peripheral blood mononuclear cells (PBMCs) from a patient immunized with an antigen of the cancer, stimulating the PBMCs with the antigen of the cancer in vitro, transducing the PBMCs with a retroviral vector, which (a) comprises and expresses a human interleukin-2 (IL-2) coding sequence operably linked to a retroviral promoter, (b) does not comprise an exogenously introduced gene that enables phenotypic selection, and (c) comprises a viral envelope that efficiently transduces CD8+ T-lymphocytes; compositions comprising cells obtained in accordance with such methods; and methods of treating a patient having cancer by administering to the patient cells obtained in accordance with such methods or compositions comprising same.

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Cambridge, MD, United States of America

Ke Liu