11 results about "Homeobox" patented technology

The disclosure provides a liposome nanoparticle, a pharmaceutical composition comprising liposome nanoparticles, and a method of treating brain cancer. In some aspects, the liposome nanoparticle comprising a folate bearing lipid and nucleic acid molecules (e.g., siRNA or shRNA) that reduce the expression of H2.0 Like Homeobox (HLX). The liposome nanoparticle optionally comprises a diameter of about 50 nm to about 400 nm. In some aspects, the pharmaceutical composition comprises the liposome nanoparticles and a pharmaceutically acceptable carrier, diluent, or excipient. In some aspects, the method of treating brain cancer comprises administering to a subject in need thereof the liposome nanoparticle.

Provided herein are oligonucleotides for inhibiting the expression of Double Homeobox 4 (DUX4) and methods of using the oligonucleotides for reducing DUX4 expression in a subject.

The present invention relates to novel use of a paired related homeobox 1 (PRRX1) gene and / or an ATP binding cassette subfamily C member 9 (ABCC9) gene as lung cancer-specific methylation marker(s) and, in particular, to a composition for diagnosing lung cancer by detecting methylation by using the PRRX1 or ABCC9 gene as a biomarker, a kit comprising same, and a method for providing information for lung cancer diagnosis.

In particular, compositions comprising iPSC-derived parietal cell precursors (iMPCs) generated using NK3 homeobox 1 (NKX3.1; parietal cell fate-determining transcription factor) and methods for their preparation and use are described herein. In addition, in particular, methods for maturing iMPCs into functional parietal cell subtypes, including smooth muscle cells, perivascular cells, and fibroblasts, as well as methods for increasing angiogenesis, angiogenesis, and cell junctions, are described herein. iMPCs mediate the formation of functional blood vessels when transplanted together with endothelial cells (ECs); thus, methods for modeling vascular diseases (e.g., 3D vascular organoids (VOs)) and therapeutic vascularization, comprising a method of administering iMPCs and ECs and a step of administering iMPCs and ECs, are also described herein.

An RNA interference-based product and method for inhibiting the expression of the double homeobox 4 (DUX4) gene on human chromosome 4q35 are disclosed. The invention comprises the silencing of the Cas13 protein of RNA, wherein Cas13 is specifically targeted to a DUX4 region of interest using a sequence-specific guide RNA (gRNA). The recombinant adeno-associated virus of the invention is constructed with a Cas13 direct repeat sequence and delivers DNA encoding an inhibitory gRNA that knocks down DUX4 expression. The method is applicable to the treatment of muscular dystrophy, including but not limited to facial-scapulohumeral muscle dystrophy (FSHD), and other disorders, including cancer, associated with elevated DUX4 expression.

RNA interference-based nucleic acids encoding double homeobox 4 (DUX4)-encoding guide RNAs (gRNA), or gRNAs that specifically hybridize to a target nucleic acid encoding DUX4 and methods for inhibiting the expression of DUX4 using the gRNAs and Cas13 are provided. The methods have application in the treatment of muscular dystrophies including, but not limited to, facioscapulohumeral muscular dystrophy (FSHD), and cancer, and other disorders associated with elevated DUX4 expression.

Use of a homeobox containing 1 (HMBOX1) inhibitor in preparation of a drug for prevention and / or treatment of muscle atrophy. Functional experiments at the cellular level show that inhibiting the expression of HMBOX1 may effectively suppress the occurrence of muscle atrophy. Furthermore, functional experiments at the animal level demonstrate that inhibiting HMBOX1 expression has preventive and therapeutic effects against muscle atrophy. Therefore, the HMBOX1 inhibitor is used for the prevention and / or treatment of muscle atrophy, offering a new approach for the development of drugs that inhibit muscle atrophy.

In one aspect, the invention provides a method of screening a human subject to determine if said subject has a genetic predisposition to develop, or is suffering from Facioscapulohumeral Dystrophy (FSHD), said method comprising: (a) providing a biological sample comprising genomic DNA from the subject; and (b) analyzing the portion of the genomic DNA in the sample corresponding to the distal D4Z4-pLAM region on chromosome 4 and determining the presence or absence of a polymorphism resulting in a functional polyadenylation sequence operationally linked to exon 3 of the DUX4 gene.

Nucleic acids, vectors, compositions, systems, and methods for expressing a structural maintenance of chromosomes hinge domain containing 1 (SMCHD1) polypeptide to epigenetically silence double homeobox 4 (DUX4) for the treatment of a disease or disorder associated with DUX4 are provided. DUX4 regulates gene expression and plays a role in development, muscular dystrophy (including, but not limited to, facioscapulohumeral dystrophy (FSHD)), a cancer, or Bosma arhinia microphthalmia syndrome (BAMS). The disclosure describes a split intein-mediated protein trans-splicing approach that was utilized to express SMCHD1 to downregulate or inhibit DUX4 expression.

The invention relates to a new application of a homologous box gene, in particular to an application of a BARX homologous frame 1 (BARX1) and an NK1 homologous frame 1 (NKX1-1) in resisting hepatitis B virus (HBV). The invention discovers that BARX1 and NKX1-1 are novel host limiting factors of HBV for the first time. BARX1 and NKX1-1 synergistically inhibit HBV through dual mechanisms: on one hand, the degradation of HBx protein is horizontally promoted after transcription; on the other hand, the activity of an HBV enhancer II / core promoter is inhibited, so that the hepatitis B virus is inhibited. In-vivo and in-vitro experiments prove that HBV gene expression and genome replication can be remarkably reduced by overexpressing BARX1 or NKX1-1. The invention provides an effective molecular target for developing a novel anti-HBV drug and a gene therapy strategy.