Shielding enzymes are made by modifying the enzyme surface with silica precursors and then depositing silica to a desired thickness while retaining biological activity of the enzyme.

The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting a SOD1 gene, as well as methods of inhibiting expression of a SOD1 gene and methods of treating subjects having a SOD1-associated neurodegenerative disease or disorder, e.g., Amyotrophic Lateral Sclerosis (ALS), Alzheimer's disease (AD), Parkinson's disease (PD), and Down's syndrome (DS), using such dsRNAi agents and compositions.

The present invention provides biomarkers of oxidative stress in subjects with retinitis pigmentosa, age-related macular degeneration, diabetic retinopathy, Fuchs' dystrophy, diabetic macular edema (DME), geographic atrophy, Stargardt's disease, or retinal vein occlusion (RVO), and their use in identifying subjects in need of treatment and methods for staging the severity of the disease.

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 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 a biocompatible peptide inhibiting the aggregation of a β-amyloid protein, and more particularly, to a biocompatible peptide derived from superoxide dismutase 1 (SOD1) and specifically binding to β-amyloid, a β-amyloid aggregation inhibitor including the peptide, and a pharmaceutical composition for treating a β-amyloid aggregation-associated disease. The peptide of the present invention has a strong binding strength to β-amyloid, inhibits the formation of a β-amyloid protein aggregate, and prevents neural cell death, thereby treating a neurodegenerative disease such as Alzheimer's disease. In addition, since the peptide of the present invention does not have cytotoxicity, it has no side effect of disturbing an immune system.

Provided herein are compositions that include proteins that include a mitochondrial localization amino acid sequence attached to a base editor fusion protein including a nucleotide base-modifying enzyme and an RNA-guided DNA endonuclease enzyme with modified endonuclease activity. Provided herein are nucleic acids encoding the proteins and vectors including the nucleic acids. Provided herein are pharmaceutical compositions including the compositions, proteins, nucleic acid and vectors. Provided herein are related methods for modifying mitochondrial DNA and treating mitochondrial disorders.

Provided herein are systems comprising Class2, Type V CRISPR polypeptides, guide nucleic acids (gNA), and optionally donor template nucleic acids useful in the modification of a PCSK9 gene. The systems are also useful for introduction into cells, for example eukaryotic cells having mutations in the PCSK9 gene. Also provided are methods of using such CasX:gNA systems to modify cells having such mutations.

A transdermal peptide with a nuclear localization ability and having an amino acid sequence as shown in SEQ ID NO: 1 is disclosed. A fusion protein including a macromolecular protein with one end being linked to the transdermal peptide is also disclosed. The transdermal peptide can be used in the preparation of a medicament or a transdermal preparation for treating skin diseases. A medicament for treating a skin disease includes the transdermal peptide and a pharmaceutically acceptable excipient. The transdermal peptide enters the cells autonomously to locate in the nuclei, and can penetrate through the stratum corneum of the skin into cells in the dermis. The peptide is conveniently synthesized artificially and suitable for transdermal administration, and has a therapeutic potential via transdermal administration by carrying a drug for treating skin diseases.

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