The invention relates to inhibitory nucleic acids and rAAV-based compositions, methods and kits useful for treating Amyotrophic Lateral Sclerosis.

Provided are compositions comprising super-oxide dismutase and a soluble fiber. The compositions may additionally comprise other antioxidants, vitamins and nutrients. The compositions can be used as dietary supplements and for improving health and well-being.

The invention concerns Flavine adenine dinucleotide (FAD) for use in preventing and/or treating cancer. The FAD is favorably encapsulated at least partially in a particle with a vector to improve its absorption and distribution while limiting its destruction, in particular by blood hydrolases. The invention relates to the pharmaceutical field and more specifically to oncology or cancerology.

The present disclosure provides materials and methods for treating a patient with one or more conditions associated with SOD1 whether ex vivo or in vivo. In addition, the present disclosure provides materials and methods for editing and/or modulating the expression of SOD1 gene in a cell by genome editing.

The present disclosure is drawn to superoxide dismutase (SOD) compositions and methods thereof. A topical composition can comprise a combination of a therapeutically effective amount of superoxide dismutase (SOD) with a stabilizing carrier that is suitable for topical administration. A method of treating a condition in a subject that is responsive to treatment with superoxide dismutase (SOD) can comprise administering a therapeutically effective amount of the topical composition. A method of stabilizing a superoxide dismutase (SOD) composition can comprise combining an amount of SOD with deoxygenated water to form an SOD solution, and minimizing exposure of the SOD solution to reactive oxygen species (ROS).

The present invention relates to AAVs encoding a SOD1 targeting polynucleotide which may be used to treat amyotrophic lateral sclerosis (ALS) and/or canine degenerative myelopathy (DM).

The invention provides compositions and methods for the treatment of lysosomal storage diseases such as metachromatic leukodystrophy and related disorders. A significant unmet clinical need still exists with the current clinical lentiviral vectors due to the high integration requirement and currently insufficient levels of ARSA expression to correct onset of early symptomatic disease. Methods and compositions for producing a protein such as sulfatase and for treating a disease or disorder such as metachromatic leukodystrophy and related disorders are disclosed.

The present invention relates, in part, to methods of preventing or treating macular degeneration in a subject by co-administering a superoxide dismutase enzyme and probiotic Bacillus sp. spores, especially a Bacillus amyloliquefaciens GF423 or GF424 mutant strain. The present invention also provides pharmaceutical and/or food compositions comprising a superoxide dismutase enzyme and probiotic Bacillus sp. spores.

Disclosed are means of augmenting efficacy of stem cell therapy for articular tissues through introduction of a step to reduce oxidative stress inside the joint before administration of stem cells. In one embodiment reduction of oxidative stress and/or inflammation is accomplished by administration of anti-oxidant agents locally in the tissue in which stem cell administration is anticipated to. In one embodiment administration of hydrogen gas is provided as a “preconditioning” step before administration of stem cell therapy. In one embodiment cell therapy is a same day non manipulated procedure involving administration of bone marrow aspirate.

The present invention relates to small interfering RNA (siRNA) molecules against the SOD1 gene, adeno-associated viral (AAV) vectors encoding siRNA molecules and methods for treating amyotrophic lateral sclerosis (ALS) using the siRNA molecules and AAV vectors.