The present disclosure relates generally to the use of sphingolipid-metabolizing proteins to mitigate or minimize tissue damage resulting from injury or from disease, for example, pulmonary arterial hypertension (PAH) when the sphingolipid-metabolizing protein is delivered via expression from an Anc80 vector.

The invention provides compounds, methods, and pharmaceutical compositions for reducing the amount or activity of N-acylsphingosine amidohydrolase (acid ceramidase) 1 (ASAH1) mRNA, and in certain embodiments reducing the amount of ASAH1 protein in a cell or animal, wherein reducing the amount or activity of ASAH1 would be beneficial.

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.

Proteins, compositions, and methods for treating Farber disease are provided.

The present disclosure relates generally to the use of sphingolipid-metabolizing proteins to mitigate or minimize tissue damage resulting from injury or from disease, for example, pulmonary arterial hypertension (PAH) when the sphingolipid-metabolizing protein is delivered via expression from an Anc80 vector.

The present invention is related to a method for diagnosing Farber's disease in a subject, wherein the method comprises detecting C26 ceramide in a sample from the subject.

The present invention relates to a method for treating or preventing pathogenic infections in a subject having Cystic Fibrosis, COPD, and/or an open wound. This method involves selecting a subject having Cystic Fibrosis, COPD, and/or an open wound and administering to the selected subject a ceramidase under conditions effective to reduce ceramide and to treat or prevent the pathogenic infection. The method also involves the use of a ceramidase in combination with other drugs to reduce infection, reduce ceramide, or improve lung function in Cystic Fibrosis, COPD, and/or open wound patients.

Described herein are cell lines, systems, kits, and methods for determining the specificity and potency of inhibitors for human ceramidase inside the cell and in a test tube.

The present invention provides an oncolytic virus comprising nucleotide sequence(s) encoding one or more immune checkpoint modulator(s). It also concerns a pharmaceutical composition comprising effective amount of said oncolytic virus and, eventually, a pharmaceutically acceptable vehicle and its use for treating proliferative diseases such as cancers.

The present disclosure relates to a method of treating an orthopedic condition, an orthopedic injury, or both in a subject in need of such treatment. The method includes administering a therapeutically effective amount of an adeno-associated viral vector to the subject. The adeno-associated viral vector transfects cells with a polynucleotide sequence that encodes and drives expression of an acid ceramidase. Also disclosed is a method of treating orthopedic inflammation in a subject in need of such treatment. The method includes administering a therapeutically effective amount of an adeno-associated viral vector that codes for expression of acid ceramidase to the subject.