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.

The invention relates to a method for the treatment of amyotrophic lateral sclerosis (ALS). Specifically, the invention implements the use of an antisense sequence adapted to affect alternative splicing in a human SOD1 pre-mRNA, thereby leading to the destruction of the skipped m RNA by the cell machinery.

The present disclosure belongs to the technical field of genetic modification of an enzyme preparation and particularly relates to a preparation method of a high-stability superoxide dismutase with a transmembrane capability. The method includes the following steps: extracting mRNA from Geobacillus stearothermophilus, synthesizing cDNA by a reverse transcription method, amplifying a large number of coding regions of the cDNA by designing a specific primer, ligating the coding regions to an E. coli expression vector, and transforming the coding regions into engineering bacteria BL21 (DE3). A point mutation technology is used to enhance stability of the superoxide dismutase and a flexible polypeptide linker GGGSGGGS (SEQ ID NO: 11) is designed, such that a soluble fusion expression of a transmembrane peptide YGRKKRRQRRR (SEQ ID NO: 10) and the superoxide dismutase is successfully realized.

The present invention provides compositions and methods for producing magnetic bionanocatalysts (BNCs) comprising metabolically self-sufficient systems of enzymes that include P450 monooxygenases or other metabolic enzymes and cofactor regeneration enzymes.

The invention relates to compositions and methods for the preparation, manufacture and therapeutic use of modulatory polynucleotides.

The present invention provides a composition and method for treating, delaying the onset, delaying progression of, reducing the incidence of or reducing the severity of amyotrophic lateral sclerosis in a subject. The composition a peptide derived from SOD1 which can be used to inhibit formation of SOD1 amyloid-like aggregates or for production of anti-SOD1 antibodies.

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 method of decreasing eye oxidation is described. The method includes administering a therapeutically effective amount of an antioxidant to a subject undergoing an ophthalmic surgical procedure.

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

The present invention provides methods of enhancing the efficacy and specificity of RNAi using single or double blunt-ended siRNA. The invention also provides single and double-blunt ended siRNA compositions, vectors, and transgenes containing the same for mediating silencing of a target gene. Therapeutic methods are also featured.