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.

Radioprotection, Radiomitigation and Radiorecovery: Timed use of more widely available antiradiation agents in a kit for subjects affected by ionizing radiation, radiomimetic exposure and radio contamination. The method in the form of strategically timed preemptive and post-irradiation compositions; radioprotection, radiomitigation and radiorecovery formulations are comprised of various available anticorporation, antioxidant, decorporation, multi-mechanistic, pro-survival, pro-hematopoietic, anti-fibrotic and other novel ingredients in synergistic mixtures to be used in critically-timed manners. Radioprotection, radiomitigation and radiorecovery of a mammal prior to, during, just after or well-after exposure to ionizing radiation energy—alpha, beta, neutron, gamma, X-ray and damaging radiofrequency radiation—for long or short periods of time, and exposure to or contamination by radioactive elements or compounds such as radioiodine, radiostrontium, radiophosphorus, radiocobalt, radiocadmium, radiopollonium, radioradium, radiocesium, radiouranium, radioamericium, radiopollonium, radiocerium, radioindium, and the like. Radiomimetic protective method for mucosal exposure is also described.

Genetically engineered bacteria, pharmaceutical compositions thereof, and methods of treating or preventing autoimmune disorders, inhibiting inflammatory mechanisms in the gut, and/or tightening gut mucosal barrier function are disclosed.

The present invention relates to RNA-based methods for inhibiting the expression of the superoxide dismutase 1 (SOD-1) gene. Recombinant adeno-associated viruses of the invention deliver DNAs encoding RNAs that knock down the expression of SOD-1. The methods have application in the treatment of amyotrophic lateral sclerosis.

Genetically engineered bacteria, pharmaceutical compositions thereof, and methods of treating or preventing autoimmune disorders, inhibiting inflammatory mechanisms in the gut, and/or tightening gut mucosal barrier function are disclosed.

The disclosure in some aspects, relates to nucleic acids, compositions and kits useful for gene therapy with reduced immune response to transgene products.

Disclosed herein are antisense compounds and methods for decreasing SOD-1 mRNA and protein expression. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate SOD-1 associated diseases, disorders, and conditions. Such SOD-1 associated diseases include amyotrophic sclerosis (ALS).

A composition comprising an endothelial progenitor cell genetically modified to have transiently reduced p53 expression for treating diabetic kidney disease and uses thereof

The present invention is directed to methods for the treatment or prevention of oxidative stress in a cell, e.g., photoreceptor cell, and methods for the treatment and prevention of disorders associated therewith by the administration of an agent, e.g., a nucleic acid molecule, which increases the expression and/or activity of an antioxidant defense protein.

The present disclosure relates to methods of feeding animals by providing feed additives that modulate the gut microbiome to improve the health, nutrition, and growth performance. The present disclosure further relates to methods of modulating metabolites present in the gastrointestinal tract of an animal. Such modulation includes, for example, modulating the level said metabolites.