C12Y101/01205

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

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.

Kits and methods to distinguish false labor and true labor

Kits and methods to distinguish between false and true labor are provided. The kits and methods can utilize differences in abundance and/or differences in the rate of change in abundance of B7-H2, SORC2, TF, C1-Esterase Inhibitor, Ran, IMDH1 and/or PGAM1, as markers of true labor.

USE OF LENTIVECTOR-TRANSDUCED T-RAPA CELLS FOR AMELIORATION OF LYSOSOMAL STORAGE DISORDERS

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.

NOVEL IN VITRO AND IN VIVO ENRICHMENT STRATEGY TARGETING LYMPHOCYTES DERIVED FROM VECTOR TRANSDUCED HSCS FOR THERAPY OF DISORDERS
20250188428 · 2025-06-12 ·

The present invention is related to a dual promoter lentiviral vector and methods of use for the treatment of diseases and disorders, specifically lysosomal storage disorders.

KITS AND METHODS TO DISTINGUISH FALSE LABOR AND TRUE LABOR

Kits and methods to distinguish between false and true labor are provided. The kits and methods can utilize differences in abundance and/or differences in the rate of change in abundance of B7-H2, SORC2, TF, C1-Esterase Inhibitor, Ran, IMDH1 and/or PGAM1, as markers of true labor.

TARGETED AUGMENTATION OF NUCLEAR GENE OUTPUT

Provided herein are methods and compositions for increasing production of a target protein or functional RNA by a cell.