Abstract: This disclosure features chemical entities (e.g., a compound or a pharmaceutically acceptable salt, and/or hydrate, and/or cocrystal, and/or drug combination of the compound) that comprise one or more ligand moieties for an asialoglycoprotein receptor (ASGPR). Exemplary chemical entities can further comprise an oligonucleotide. Said chemical entities are useful, e.g., in the targeted delivery of oligonucleotides to liver cells (e.g., liver parenchymal cells). The chemical entities are useful e.g., in the treatment of conditions or diseases caused by the expression (e.g., abnormal expression) of one or more genes in liver cells This disclosure also features compositions containing the same as well as methods of using and making the same.

Abstract: The invention relates to a lipid compound of formula (I), including lipid nanoparticles thereof, and the manufacturing method and the use of pharmaceutical delivery. The lipid compounds have formula (I): or a salt or an isomer thereof, wherein R1, R2, R3 n and m are defined herein.

Abstract: Provided are a small interfering RNA (siRNA) molecule which inhibits the expression of the PCSK9 gene and a pharmaceutical composition thereof, as well as a method for reducing the expression level of the PCSK9 gene by using the siRNA molecule or the pharmaceutical composition thereof. The siRNA molecules may be used to treat and/or prevent PCSK9 gene-mediated diseases.

Abstract: This disclosure relates to coronavirus vaccines and uses thereof. In one aspect, the disclosure provides a nucleic acid vaccine, comprising a sequence encoding a spike protein or fragment thereof derived from a coronavirus.

Abstract: The present disclosure falls within the field of biomedical technology, and in particular relates to modified oligonucleotides and a compound that can be used for synthesizing same and a method for modifying oligonucleotides. The present disclosure also relates to the use of the modified oligonucleotides for preventing and/or treating diseases associated with the liver in a subject.

Abstract: Disclosed are a new-type nucleic acid unit for construction of a polymeric nucleic acid and the polymeric nucleic acid for interfering with target gene expression. In the present application, by design and construction of the new-type nucleic acid unit and the self-assembled polymeric nucleic acid thereof, multiple target interference may be realized, wherein the same may be used for inhibiting multiple gene expressions in a signaling pathway of disease occurrence or development, or simultaneously inhibiting multiple disease target genes expression, possessing broad application prospects in multiple subject fields such as biology and chemistry. The polymeric nucleic acid may target multiple sequences simultaneously, wherein the sequences may be located in one or more genes.

Abstract: Provided is a system for DNA editing, comprising crRNA and tracrRNA. The crRNA is an RNA shown in formula I: Nx-ncrRNA (formula I), wherein the Nx is a spacer, and the ncrRNA is an RNA shown in SEQ ID NOs. 1-4 of the Sequence Listing. The tracrRNA is an RNA shown in SEQ ID NOs. 5-8 of the Sequence Listing.

Abstract: The present disclosure falls within the field of biomedical technology, and in particular relates to modified oligonucleatides and a compound that can be used for synthesizing same and a method for modifying oligonucleotides. The present disclosure also relates to the use of the modified oligonucleotides for preventing and/or treating diseases associated with the liver in a subject.

Abstract: Provided are siRNAs or chemically-modified siRNAs targeted against ADAMTS-5 or ADAM17 for inhibiting the expression of ADAMTS-5 or ADAM17. Use of the siRNAs for treating an ADAMTS-5 or ADAM17 associated disease, such as arthritis and other inflammation-related diseases, by injecting the siRNAs or preparations comprising the siRNAs into an articular cavity of the patient, are also provided.

Abstract: Provided are siRNAs or chemically-modified siRNAs targeted against ADAMTS-5 or ADAM17 for inhibiting the expression of ADAMTS-5 or ADAM17. Use of the siRNAs for treating an ADAMTS-5 or ADAM17 associated disease, such as arthritis and other inflammation-related diseases, by injecting the siRNAs or preparations comprising the siRNAs into an articular cavity of the patient, are also provided.

Abstract: A chemical tag can include a core and a plurality of substituents attached directly to the core. The substituents of each chemical tag form a subset of a closed set of possible substituents. The tag can be used to track an object.

Abstract: The present invention relates to the synthesis of novel compounds for the inhibition of peptide synthesis in the ribosome. It also relates to improved methods for directly monitoring peptide bond formation in the ribosome. These compounds can be used for high throughput screening of proposed therapeutically active drug species (e.g., antibiotics) targeted to ribosomes.

Abstract: Methods for synthesizing RNA molecules whose 5′-terminus comprises a thiol group are disclosed. The present invention discloses the formation of 5′-HS-PEG-GMP-RNA and 5′-GMPS-RNA which upon alkaline phosphatase treatment, independently lead to a 5′-HS-RNA molecule.