National Institutes of Health, a Component of the US Dept. of Health & Human Services

University of Pennsylvania

The Medical College of Wisconsin, Inc.

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

University of Virginia Patent Foundation

Daniel H Fowler

Carl H June

Bruce L Levine

James L Riley

Jeffrey A Medin

Ronald E Gress

Shoba Amarnath

Unsu Jung

Hyun K Kim

Charles L Carter

Brian J Czerniecki

Peter A Cohen

Murtaza S Nagree

Robyn A Oldham

Tania Felizardo

The present disclosure provides methods of treating lysosomal storage disorders, e.g., Fabry disease, Gaucher disease, Farber disease, and Pompe disease. The method comprises producing vector-transduced T-Rapa cells that express a transgene of interest and administering the cells to a patient in need thereof. The T-Rapa cells may be transduced with a dual promoter lentivirus vector.

Use of lentivector-transduced T-Rapa cells for amelioration of lysosomal storage disorders

The present disclosure provides chimeric antigen receptor (CAR)-T-Rapa cells and methods of making and using them. Specifically, methods of producing T-Rapa cells that can express chimeric antigen receptors is provided.

T-Rapa cells as novel effector cell type for chimeric antigen receptor therapy

The present invention provides methods and compositions for converting a T cell into a cell that exhibits at least one regulatory T cell phenotype. The converted T cell is generated by contacting a T cell with a cell that is modified to comprise an agent capable of activating PD1 signaling in a T cell. The converted T cell is useful for preventing, suppressing, blocking or inhibiting an immune response. For example the converted T cell is useful for preventing rejection of a transplanted tissue in a human or other animal host, or protecting against graft versus host disease. The converted T cell can also be used to treat autoimmune diseases.

Use of PDL1 expressing cells to convert T cells into regulatory T cells

Methods for generating highly enriched Th1/Tc1 and Th2/Tc2 functions are described. In particular, the generation of these functions are attained by the addition of an immune suppression drug, rapamycin or a rapamycin derivative compound. In addition to enhanced purity of T cell function, the T cells generated in rapamycin also express molecules that improve immune T cell function such as CD28 and CD62L. Such rapamycin generated functional T cell subsets may have application in the prevention or treatment of GVHD after allogeneic hematopoietic stem cell transplantation, the treatment of autoimmunity, or the therapy of infection or cancer.

Rapamycin-resistant T cells and therapeutic uses thereof

A method of isolating dendritic cells is described. This method involves elutriating peripheral blood samples in at least four flow rates from an elutriation rotor. Calcium ionophore is used to stimulate monocytes isolated during the process into dendritic cells. Treatments for diseases involving re-introduction of activated dentritic cells are also described.

Method for isolating dendritic cells

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Bethesda, MD, United States of America

Daniel H Fowler