A polypeptide consisting of sequence SEQ ID NO: 1, or a variant thereof consisting of a sequence at least 85% identical to SEQ ID NO: 1, for use in the prevention and/or treatment of a bone disorder. The nucleic acid sequence that encodes the polypeptide, a gene construct comprising the nucleic acid sequence, or an expression vector comprising the gene construct for said use. The polypeptide, nucleic acid sequence, gene construct, or expression vector may be administered in the form of a pharmaceutical composition together with at least one pharmaceutically acceptable excipient, diluent or carrier.

The present invention relates to the specific inhibition of gene expression in mammals by bringing the target gene into contact with double stranded RNA (dsRNA).

An engineered enzyme, comprising an amino acid sequence that is at least 80% identical to the amino acid sequence of a human beta-glucuronidase, wherein the engineered enzyme exhibits a higher level of alpha-iduronidase enzymatic activity as compared to the human beta-glucuronidase.

Reagents and methods useful for the synthesis of conjugates comprising guanidinylated cyclic acetals are provided. Also provided are methods for increasing the cellular uptake of various therapeutic compounds and treatment modalities using these conjugates.

The invention provides recombinant DNA molecules and constructs, as well as their nucleotide sequences, useful for modulating gene expression in plants. The invention also provides transgenic plants, plant cells, plant parts, and seeds comprising the recombinant DNA molecules operably linked to heterologous transcribable DNA molecules, as are methods of their use.

Provided herein are klotho polypeptide compositions and methods for improving cognitive function in an individual comprising treatment of with klotho polypeptides.

The invention provides recombinant DNA molecules and constructs, as well as their nucleotide sequences, useful for modulating gene expression in plants. The invention also provides transgenic plants, plant cells, plant parts, and seeds comprising the recombinant DNA molecules operably linked to heterologous transcribable DNA molecules, as are methods of their use.

Nanoparticles coated with extracellular matrix (ECM) are provided, in some aspects, for the delivery of a therapeutic protein, nucleic acid, or drug. In some embodiments, the nanoparticles are delivered to a subject via inhalation or aerosol delivery. Also provided, in some aspects, are methods for treating acute lung injury comprising administering -Klotho (Klotho) protein or DNA to a subject.

To provide a base sequence for protein expression that can increase the yield of protein such as diastatic enzyme by further activating a promoter of a particular gene. A base sequence 1 for protein expression includes: a gene 3 encoding protein 2; a promoter 4 of the gene 3, the promoter being linked upstream of the gene 3; and a cis element 5 whose activity is improved by an artificial transcription factor 6, the cis element being linked further upstream of the promoter 4. The cis element 5 is represented by SEQ ID NO: 1.

A delivery (transport) of therapeutic enzymes, which is applicable in medicine, is disclosed. A compound containing a therapeutic enzyme and a transport element that are coupled to one another directly or by a linker is described, the transport element being a Fab fragment of immunoglobulin IgG specific to an insulin receptor epitope, and to the use of said compound to produce a pharmaceutical composition for treating diseases, as well as to the use of said compound for the treatment and prophylaxis of diseases, in particular lysosomal storage diseases, inter alia, for the treatment and prophylaxis of the enzyme deficiency characteristic of the respective lysosomal storage disease, such as mucopolysaccharidosis, in particular mucopolysaccharidosis types I and II.