University of Minnesota

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

Intima Bioscience, Inc.

Healthpartners Research & Education

Regenxbio Inc.

Other

Immusoft Corporation

Sangamo Therapeutics, Inc.

Mcivor, R. Scott

Kozarsky, Karen

Belur, Lalitha R.

R Scott McIvor

Steven A Rosenberg

Nicholas P Restifo

Beau R Webber

Branden Moriarity

Modassir Choudhry

William Howard Frey, Ii

Russell DeKelver

Douglas C Palmer

Walter C Low

Karen Kozarsky

David Largaespada

David W Hunter

Thomas Wechsler

Carolyn Ann Fairbanks

Daniel A Wolf

Leah R Hanson

Lalitha R Belur

Perry B Hackett

Chad May

Jason Bell

Chester B Whitley

Myra Christine Urness-Rusten

Rohaizah I James

Elena Aronovich

Kanut Laoharawee

Li Ou

A method to prevent, inhibit or treat one or more symptoms associated with a disease of the central nervous system by intrathecally, intracerebroventricularly or endovascularly administering a rAAV encoding a gene product associated with the disease, e.g., a mammal in which the gene product is absent or present at a reduced level relative to a mammal without the disease.

Methods to treat mucopolysaccharidosis type II or deficiency in iduronate-2-sulfatase using a recombinant adeno-associated virus (AAV) vector encoding iduronate-2-sulfatase

Genetically modified compositions, such as non-viral vectors and T cells, for treating cancer are disclosed. Also disclosed are the methods of making and using the genetically modified compositions in treating cancer.

Modified cells and methods of therapy

Materials and methods for treating a patient to express a therapeutic agent comprising administering a Kupffer cell-suppressing substance in combination with a vehicle for introducing, into the patient, an exogenous nucleic acid comprising a sequence for expression of the agent.

Method to achieve extended expression of DNA infused into liver

Intracellular genomic transplant and methods of therapy

Methods to treat mucopolysaccharide type I or deficiency in alpha-L-iduronidase using a recombinant adeno-associated virus encoding alpha-L-iduronidase

The invention provides a method to prevent, inhibit or treat one or more neurological symptoms associated with a lysosomal storage disease in a mammal in need thereof, which includes intranasally administering to the mammal a composition comprising an effective amount of a lysosomal storage enzyme or a recombinant adeno-associated virus vector comprising an open reading frame encoding a lysosomal storage enzyme. Also provided are compositions and devices useful in the methods.

Intranasal delivery of AAV encoding therapeutic enzymes to the central nervous system for the treatment of lysosomal storage diseases

Intranasal delivery of therapeutic enzymes to the central nervous system for the treatment of lysosomal storage diseases

Isolated nucleic acid-based vectors and lentivirus vectors, and methods of using those vectors to inhibit or prevent metabolic disorders in a mammal, are provided.

Methods of treating lysosomal storage related diseases by gene therapy

A method to increase the efficiency of hematopoietic stem cell engraftment in vivo is provided.

Method for selective engraftment of drug-resistant hematopoietic stem cells

Growing community of inventors

St. Louis Park, MN, United States of America

R Scott McIvor