A composition for the delivery of a nucleic acid compound is provided which comprises a cationic peptide or polymer and a lipidoid compound. The nucleic acid compound may be any chemically modified or unmodified DNA or RNA. The amount of the lipidoid in the composition is preferably low, relative to the cationic peptide or polymer.

Improved compositions and methods for treating a disease or disorder through target exon skipping, and preferably muscular dystrophy by administering antisense thiomorpholino molecules capable of binding to a selected target site in the human dystrophin gene to induce exon skipping to produce a functional Dystrophin protein.

Synthetic peptides mimicking the nsp10 sequence in the region interacting with nsp16 capable of penetrating cell membranes and inhibiting SARS-CoV-2 replication for the treatment of moderate to severe COVID-19. The invention relates to peptides inhibiting SARS-CoV-2 replication, likely through inhibition of Methyltransferase complexes (NSP10/NSP16 and NSP10/NSP14). The peptide of the present invention, P3, contains sequences corresponding to amino acids 89-96 of the non-structural protein 10 (NSP10) of SARS-CoV-2, with the only Cysteine modified to a Methionine. This peptide was made based on two previous designs P1 and P2, which constituted the amino acids 68-96 of the NSP10 protein of SARS-CoV-2.

The invention concerns a vesicle, such as an extracellular vesicle or lipid vesicle, that has been loaded with a cargo molecule comprising a CD24 molecule, covalently or non-covalently coupled to a cell penetrating polypeptide (resulting in a “binding complex”), and the cargo molecule or binding complex has been internalized by the vesicle, associated with the vesicle, or a combination thereof. Advantageously, the CD24 molecule may have extracellular amino acids that become displayed on the outer surface of the vesicle upon loading. Other aspects of the invention concern a vesicle loaded with cargo molecule comprising a CD24 molecule, a method for loading a vesicle with a cargo molecule comprising a CD24 molecule, and a method for delivering a CD24 molecule into a cell in vitro or in vivo, to control inflammation such as for treatment, prevention, or delay of onset of cytokine storm.

A method for treating a cancer comprises administering to a subject in need of such treatment an effective amount of a pharmaceutical composition comprising an anti-cancer agent having at least one secretion modifying region (SMR) peptide from HIV-1 Nef fused to at least one cell-penetrating peptide (CPP) or at least one Clusterin (Clu)-binding peptide (Clu-BP).

Disclosed in the present invention is a lysosome-targeting antibody-drug conjugate and use thereof. The structure of the antibody-drug conjugate is Dr.sub.n1AbO.sub.n2; wherein, Dr is a drug, Ab is an antibody, and O is a lysosome-targeting small molecule or a functional peptide for increasing the lysosomal targeting ability of the antibody-drug conjugate; n1 and n2 are integers greater than or equal to 1, and n1 and n2 are identical or different.

Described herein is a composition and method of treating COVID-19 with lipid-peptide fusion antiviral therapy. Also described is a composition and method of treating Ebola with lipid-peptide fusion antiviral therapy.

According to the present disclosure, a technology for efficiently introducing a desired foreign substance from the outside of eukaryotic cells into at least the cytoplasm (and the nucleolus) of the cells is provided. A method disclosed here includes a step of preparing a construct for introducing a foreign substance including a carrier peptide fragment composed of any of amino acid sequences: KKRTLRKKKRKKR (SEQ ID NO: 1), KKRTLRKRRRKKR (SEQ ID NO: 2), KKRTLRKRKRKKR (SEQ ID NO: 3) and KKRTLRKKRRKKR (SEQ ID NO: 4), and a foreign substance that is bound to an N-terminal side and/or C-terminal side of the carrier peptide fragment, a step of supplying the construct for introducing a foreign substance to a sample containing desired eukaryotic cells, and a step of incubating the sample to which the construct for introducing a foreign substance is supplied and introducing the construct into eukaryotic cells in the sample.

The disclosure relates generally to polyglutamated antifolates, formulations containing liposomes filled with alpha or D-gamma polyglutamated antifolates, methods of making the polyglutamated antifolates and liposome containing formulations, and methods of using polyglutamated antifolates and liposome containing formulations to treat hyerproliferative disorders (e.g., cancer) and disorders of the immune system (e.g., an autoimmune disease such as rheumatoid arthritis).

The present disclosure relates to compounds useful for the detection or modulation of target nucleic acids, including DNA and RNA. The present disclosure further relates to methods for treatment of trinucleotide repeat disorders, which can include administration of oligonucleotide analogues that can bind pathogenic nucleotide repeats in DNA or RNA.