Aspects of the present invention disclose compounds that modulate the aggregation of amyloidogenic proteins or peptides. In some aspects, disclosed compounds modulate the aggregation of disease-associated proteins and natural -amyloid peptides. In a preferred embodiment, the compounds can inhibit natural amyloid aggregation. Pharmaceutical compositions comprising the compounds of the embodiments, and diagnostic and treatment methods for diseases (e.g., amyloidogenic diseases) using the compounds, are also disclosed. In addition, there is provided an integrated bacterial platform for the discovery of rescuers of disease-associated protein misfolding.

Methods and compositions for modifying the expression of endogenous genes or modifying the coding sequence of endogenous genes using rare-cutting endonucleases and transposases.

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

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 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.

The present invention provides a novel adsorbent composition for recovering biomolecules from a fluid. The composition comprises positively and negatively charged microparticles in the form of ground particles. The adsorbent is particularly useful for purification of biomolecules from the cell culture.

This invention provides a stably transformed plant, plant part and/or plant cell, comprising a heterologous polynucleotide encoding a superoxide reductase (SOR) from an archaeon species, wherein said stably transformed plant, plant cell, and/or plant part has increased disease resistance. The invention further provides a method of increasing disease resistance in a plant, plant cell, or plant part, comprising: introducing into said plant, plant cell, or plant part a heterologous polynucleotide encoding a superoxide reductase from an archaeon species to produce a stably transformed plant, plant cell, or plant part, thereby producing a plant, plant part, or plant cell having increased disease resistance as compared to a control. Additionally provided are plants, plant parts, and plant cells produced by the methods of the invention, as well as progeny and products produced therefrom.

The invention relates to a transdermal peptide with a nuclear localization ability and having an amino acid sequence as shown in SEQ ID NO: 1. The invention provides a fusion protein with a nuclear localization ability, which comprises a macromolecular protein with one end being linked to the transdermal peptide. The invention also discloses the use of the transdermal peptide in the preparation of a medicament or a transdermal preparation for treating skin diseases. The invention further provides a medicament for treating a skin disease, comprising 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.

An expression vector containing appropriate mitochondrion-targeting sequences (MTS) and appropriate 3UTR sequences provides efficient and stable delivery of a mRNA encoding a protein (CDS) to the mitochondrion of a mammalian cell. The MTS and 3UTR sequences guide the CDS mRNA from the nuclear compartment of the cell to mitochondrion-bound polysomes, where the CDS is translated. This provides an efficient translocation of a mature functional protein into the mitochondria. A method of targeting mRNA expressed in the nuclear compartment of a mammalian cell to the mitochondrion is also provided. The vector and methods can be used to treat defects in mitochondrial function.

Provided herein, inter alia, are chemically defined components and compositions that substitute or partially substitute for albumin in cell culture media. The components and compositions may support cell cultures, protect cells, or enhance viability of cultured cells. Further provided are chemically defined culture media supplements for use in cell culture media containing albumin. The chemically defined culture media supplements may rescue cells from albumin-induced toxicity.