The present invention relates to adeno-associated viral (AAV) particles encoding siRNA molecules and methods for treating amyotrophic lateral sclerosis (ALS).

The present disclosure includes the use of a miRNA inhibitor for treating amyotrophic lateral sclerosis (ALS) associated with a decreased level of SIRT1 protein or SIRT1 gene expression, PGC-1α protein and/or PGC-1α gene expression, CD36 protein and/or CD36 gene expression, NRG1 protein and/or NRG1 gene expression, STMN2 protein and/or STMN2 gene expression, and/or NRXN1 protein and/or NRXN1 gene expression.

The invention relates to a method for enriching a biomass of a microalga of the species Tetraselmis chuii in superoxide dismutase (SOD) by placing said microalga under abiotic stress conditions. The invention also relates to a biomass enriched in SOD as well as to an extract of the microalga and to the uses thereof as a pharmaceutical composition, as a cosmetic or in foodstuff.

Provided herein is an in-series synthetic receptor circuit for dual-antigen AND-gate control over expression of a therapeutic payload by engineered cells. In some embodiments, the circuit may be composed of a first binding-triggered transcriptional switch, a second binding-triggered transcriptional switch and a therapeutic payload (e.g., a chimeric antigen receptor), where binding of the first binding-triggered transcriptional switch to a first antigen activates expression of the second binding-triggered transcriptional switch, and binding of the second binding-triggered transcriptional switch to a second antigen activates expression of the therapeutic payload. If the cell is an immune cell and the therapeutic payload is a chimeric antigen receptor, then the immune cell may be activated by binding of the chimeric antigen receptor to a third antigen. Methods of treatment using the cell also provided.

The present disclosure generally relates to methods and formulations for treating central nervous system (CNS) disorders. The present disclosure involves intranasal delivery of at least one antioxidant compound, allowing for effective treatment of a central nervous system disorder such as traumatic brain injury or stroke.

The present invention refers to steroidal nitrone (E)-N-((8S,9S,10R,13R,14S,17R)-10,13-di methyl-17-(R)-6-methyl hepta n-2-yl)-7,8,9,11,12,13,14,15,16,17-deca hydro-1H-cyclopenta[a]phenanthren-3(2H,6H,10H)-ylidene)methanamine oxide (F2), as well as any pharmaceutically acceptable salt thereof, for use in the prevention and/or treatment of permanent cerebral ischemia.

Disclosed is a composition for preventing, treating or ameliorating a viral infection disease, which is safe for human application since the safety of a reactive oxygen species production inhibitor and a reactive oxygen species scavenger has already been verified. The disclosure utilizes inversely the mechanism of viruses which induces oxidative damage of a host cell and then activates viral replication and infection. Although not having direct specificity to a specific virus like existing vaccines, the composition can effectively inhibit proliferation of all viruses including oxidative stress, regardless of virus type or mutations. It can be applied as a sub-universal infection inhibitor and therapeutic agent as a limited alternative to vaccines that must be newly developed each year due to emergence of new viruses or viral mutations, and is expected to be used as a preventive agent or auxiliary agent to prevent epidemics.

The invention provides topical compositions and methods for using the compositions. The compositions can be used for the treatment of fibrotic or connective tissue disorders involving scarring, sub-dermal plaque accumulations, or fibrosis of muscle tissue. The disorders can be painlessly treated by the topical application of a composition described herein. One or more calcium channel blocker agents can serve as an active ingredient of the compositions, optionally in combination with, for example, one or more of emu oil and superoxide dismutase. The composition can further include pharmaceutically acceptable carriers that can facilitate the non-invasive transdermal delivery of the active(s) to subdermal sites.

The present invention relates to antisense oligonucleotides that are complimentary to SOD1, leading to decreased expression of SOD1. Reduced expression of SOD1 is beneficial in medical disorders such as Amyotrophic Lateral Sclerosis.

A method of treating a cancer in a mammalian subject with a tumor signature characterized by any one or more of (i) a level of sirtuin (SIRT3) protein that is below a first predetermined threshold level, (ii) a level of manganese superoxide dismutase acetylated at the lysine 68 residue (AcK68) that exceeds a second predetermined threshold level, and (iii) expression levels of hypoxia-inducible factor 2.sub.α (HIF2.sub.α) that exceed a third predetermined threshold level indicative of lineage plasticity for stemness, the method comprising administering to the mammalian subject a therapeutically effective amount of a pentaaza macrocyclic ring complex corresponding to the Formula (I) below, optionally with administration of a further anti-cancer therapeutic agent.

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