28 results about "Homeobox" patented technology

The purpose of the present invention is to provide a compound, a method, and a pharmaceutical composition for normalizing dual homologous frame 4 (DUX4) in an individual in which the DUX4 gene is abnormally expressed, and to provide a modified oligonucleotide comprising 12-30 residues, comprising at least 8 contiguous nucleic acid base sequences, the modified oligonucleotide has a nucleic acid base sequence which is complementary to the isometric portion of the nucleic acid base of the mature mRNA of DUX4 of SEQ ID NO: 1 from the 5'terminal thereof at positions 126th to 147th, positions 232th to 248, positions 1306th to 1325th, or positions 1472nd to 1495th, and the nucleic acid base sequence of the modified oligonucleotide has at least 90% complementarity with the isometric portion of the nucleic acid base sequence of the mature mRNA of DUX4 of SEQ ID NO: 1.

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.

Disclosed are compositions including a population of human enteric neural stem cells (ENSCs), wherein the population comprises at least one of a cell expressing nerve growth factor receptor (NGFR), a cell expressing integrin alpha 6 (ITGA6), a cell expressing L1 cell adhesion molecule (L1CAM), a cell that expresses proteolipid protein 1 (PLP1), and / or a cell that expresses paired-like homeobox 2B (PHOX2B), methods of producing the compositions, and their uses.

A method of treating a cancer, the method comprising: providing a modified macrophage or monocyte that contains an exogenous nucleic acid sequence encoding a Hom- 1 polypeptide or a fragment thereof that contains the Hom-1 homeobox domain, wherein the modified macrophage or monocyte expresses the Hom-1 polypeptide or the fragment thereof; and administering the modified macrophage or monocyte to a subject with a cancer.

The present invention relates to compositions and methods for modifying WUSCHEL (WUS) related homologous box (WOX) functional genes in plants of the Rosaceae family, optionally to improve the configuration of thorns and / or thorns, to reduce the number and / or size of thorns and skin thorns, or to eliminate thorns and / or skin thorns. The present invention also relates to plants of the Rosaceae family having thorns and / or skin thorns of improved configuration, a reduced number and / or size, or no thorns and / or skin thorns produced using the methods and compositions of the present invention. The invention further relates to a method for identifying and / or cultivating a plant having thorns and / or barks of improved configuration, a reduced number and / or size, or no thorns and / or barks.

The disclosure pertains to methods and compositions for the rapid and efficient transformation of plants. The disclosure further provides methods for producing a transgenic plant, comprising (a) transforming a cell of an explant with an expression construct comprising (i) a nucleotide sequence encoding a WUS / WOX homeobox polypeptide; (ii) a nucleotide sequence encoding a polypeptide comprising two AP2-DNA binding domains; or (iii) a combination of (i) and (ii); and (b) allowing expression of the polypeptide of (a) in each transformed cell to form a regenerable plant structure in the absence of exogenous cytokinin, wherein no callus is formed; and (c) germinating the regenerable plant structure to form the transgenic plant. This abstract is intended as a scanning tool for purposes of searching in the particular art and is not intended to be limiting of the present disclosure.

The purpose of the present invention is to provide a compound, a method, and a pharmaceutical composition for normalizing dual homologous frame 4 (DUX4) in an individual in which the DUX4 gene is abnormally expressed, and to provide a modified oligonucleotide comprising 12-30 residues, comprising at least 8 contiguous nucleic acid base sequences, the modified oligonucleotide has a nucleic acid base sequence which is complementary to the isometric portion of the nucleic acid base of the mature mRNA of DUX4 of SEQ ID NO: 1 from the 5'terminal thereof at positions 126th to 147th, positions 232th to 248, positions 1306th to 1325th, or positions 1472nd to 1495th, and the nucleic acid base sequence of the modified oligonucleotide has at least 90% complementarity with the isometric portion of the nucleic acid base sequence of the mature mRNA of DUX4 of SEQ ID NO: 1.

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.

WUSCHEL and WUSCHEL-related homeobox polypeptides that can be used to improve the capacity of the plant cells to regenerate embryogenic plant tissues, plant organs, and whole plants are disclosed. Also disclosed are plant cells comprising the polypeptides and related methods for improving the capacity of the plant cells to regenerate embryogenic plant tissues, plant organs, and whole plants.

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.

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

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.

A Ginkgo biloba wuschel-related homeobox 3A (GbWOX3A) gene and an application thereof in plant tissue culture are provided. The GbWOX3A gene is derived from a Ginkgo biloba embryo, the gene sequence of the GbWOX3A gene is SEQ ID NO: 1, and the amino acid sequence encoded by the GbWOX3A gene is SEQ ID NO: 2. An expression of the GbWOX3A gene in developing embryo of Ginkgo biloba and regenerating a callus has specificity, and transgenic function verification experiments prove that overexpression of the GbWOX3A gene can promote regeneration of adventitious buds during the plant tissue culture. Therefore, the GbWOX3A gene has important theoretical significance and application value in the plant tissue culture, and especially plays an important role in improving the regeneration efficiency of gymnosperms.

The invention belongs to the field of gene engineering, and particularly relates to application to plant molecular breeding and crop nutrition improvement. According to the invention, a rice homeobox transcription factor OSH45 (Oryza sativa homeobox 45) gene is cloned through a reverse genetics approach, and the function of the gene is identified through an overexpression technology and a gene editing technology. The invention also provides application of the gene in improvement of phosphorus absorption of crops and in phosphorus-tolerant nutrient stress breeding.

Disclosed herein are products, methods, and uses for treating, ameliorating, delaying the progression of, and / or preventing a muscular dystrophy or a cancer including, but not limited to, facioscapulohumeral muscular dystrophy (FSHD) or a cancer associated with DUX4 expression or overexpression. More particularly, disclosed herein are RNA interference-based products, methods, and uses for inhibiting or downregulating the expression of double homeobox 4 (DUX4). Even more particularly, the disclosure provides microRNA (miRNA) (and vectors and compositions comprising the miRNA encoding polynucleotides) for inhibiting or downregulating the expression of DUX4 and methods of using said miRNA to inhibit or downregulate DUX4 expression in cells and / or in cells of a subject having a muscular dystrophy or a cancer associated with DUX4 expression or overexpression including, but not limited to, FSHD or a cancer associated with DUX4 expression or overexpression.

Chronic wounds are characterized by a persistent hyperinflammatory environment that inhibits progression toward regenerative wound closure. Such chronic wounds are particularly prevalent in diabetic patients, often necessitating distal limb amputation, but they also occur in non-diabetic and elderly patients. Inducible expression of HoxA3, a homeobox family member and master regulatory transcription factor for body patterning, has been shown to promote wound closure in diabetic mice when administered topically as a plasmid encapsulated in a hydrogel. Here, we provide independent reproducibility of basal in vivo studies of diabetic wound closure and further expand on these studies while minimizing the estimated minimum dose threshold. Furthermore, we observed similarities in spontaneous wound healing rates between non-diabetic aged mice and diabetic young mice, which motivated us to test the topical application of HoxA3 plasmids in non-diabetic aged mice, where accelerated wound healing was also observed. In these short studies, no significant adverse effects were observed macroscopically or by local histology. Local administration of HoxA3, either as a plasmid or as a future alternative therapy, is a noteworthy translational option for chronic wounds.

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.

The disclosure pertains to methods and compositions for the rapid and efficient transformation of plants. The disclosure further provides methods for producing a transgenic plant, comprising (a) transforming a cell of an explant with an expression construct comprising (i) a nucleotide sequence encoding a WUS / WOX homeobox polypeptide; (ii) a nucleotide sequence encoding a polypeptide comprising two AP2-DNA binding domains; or (iii) a combination of (i) and (ii); and (b) allowing expression of the polypeptide of (a) in each transformed cell to form a regenerable plant structure in the absence of exogenous cytokinin, wherein no callus is formed; and (c) germinating the regenerable plant structure to form the transgenic plant. This abstract is intended as a scanning tool for purposes of searching in the particular art and is not intended to be limiting of the present disclosure.

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 discloses an application of an SIX family homologous box gene 4 inhibitor in preparation of drugs for promoting growth of neurons of a peripheral nervous system, and the inhibitor inhibits expression of the SIX family homologous box gene 4 through nucleic acid molecules loaded by a carrier so as to realize regulation and control of the neurons of the peripheral nervous system. The invention provides and verifies for the first time that inhibition of expression of the SIX family homologous box gene 4 can promote growth of neurons of the peripheral nervous system, effective regeneration of dorsal root ganglions is realized, recovery of related functions is accelerated, and a new direction is provided for research and development of drugs for treating traumatic or disease injuries of neurons of the peripheral nervous system.

The present application provides a method for producing megakaryocytes, which comprises a step of forcibly expressing an oncogene, a homologous frame gene, and an apoptosis-inhibiting gene in a hematopoietic progenitor cell and culturing the cell. The present application also provides a kit for manufacturing megakaryocytes, mature megakaryocytes, or platelets, comprising a vector comprising an oncogene, a homologous frame gene, and an apoptosis suppressor gene.

A method of treating a cancer, the method comprising: providing a genetically modified macrophage or monocyte that contains a nucleic acid sequence encoding a Hom-1 polypeptide or a fragment thereof that contains the Hom-1 homeobox domain, wherein the nucleic acid sequence is operably linked to a heterologous promoter and the modified macrophage or monocyte expresses the Hom-1 polypeptide or the fragment thereof; and administering the modified macrophage or monocyte to a subject with a cancer.

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.

Disclosed are compositions including a population of human enteric neural stem cells (ENSCs), wherein the population comprises at least one of a cell expressing nerve growth factor receptor (NGFR), a cell expressing integrin alpha 6 (ITGA6), a cell expressing L1 cell adhesion molecule (L1CAM), a cell that expresses proteolipid protein 1 (PLP1), and / or a cell that expresses paired-like homeobox 2B (PHOX2B), methods of producing the compositions, and their uses.