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142results about "Naked nucleic acid ingredients" patented technology

Modulation of exon recognition in pre-mRNA by interfering with the secondary RNA structure

The invention provides a method for generating an oligonucleotide with which an exon may be skipped in a pre-mRNA and thus excluded from a produced mRNA thereof. Further provided are methods for altering the secondary structure of an mRNA to interfere with splicing processes and uses of the oligonucleotides and methods in the treatment of disease. Further provided are pharmaceutical compositions and methods and means for inducing skipping of several exons in a pre-mRNA.
Owner:ACADEMISCH ZIEKENHUIS BIJ DE UNIV VAN AMSTERDAM ACADEMISCH MEDISCH CENT

Porous nanoparticle-supported lipid bilayers (protocells) for targeted delivery and methods of using same

ActiveUS20140079774A1Promoting death of cancer cellEfficient packagingBiocideSpecial deliveryLipid formationBinding peptide
The present invention is directed to protocells for specific targeting of hepatocellular and other cancer cells which comprise a nanoporous silica core with a supported lipid bilayer; at least one agent which facilitates cancer cell death (such as a traditional small molecule, a macromolecular cargo (e.g. siRNA or a protein toxin such as ricin toxin A-chain or diphtheria toxin A-chain) and / or a histone-packaged plasmid DNA disposed within the nanoporous silica core (preferably supercoiled in order to more efficiently package the DNA into protocells) which is optionally modified with a nuclear localization sequence to assist in localizing protocells within the nucleus of the cancer cell and the ability to express peptides involved in therapy (apoptosis / cell death) of the cancer cell or as a reporter, a targeting peptide which targets cancer cells in tissue to be treated such that binding of the protocell to the targeted cells is specific and enhanced and a fusogenic peptide that promotes endosomal escape of protocells and encapsulated DNA. Protocells according to the present invention may be used to treat cancer, especially including hepatocellular (liver) cancer using novel binding peptides (c-MET peptides) which selectively bind to hepatocellular tissue or to function in diagnosis of cancer, including cancer treatment and drug discovery.
Owner:NAT TECH & ENG SOLUTIONS OF SANDIA LLC +1

Single-chain circular RNA and method of producing the same

InactiveUS20100137407A1Efficient productionLess susceptible to enzymatic degradationOrganic active ingredientsSugar derivativesGeneticsSense strand
The present invention relates to a single-chain circular RNA having a sustained or slow-releasing RNA interference effect, characterized in that the single-chain circular RNA comprises a sense strand sequence, an antisense strand sequence complementary to the sense strand sequence, identical or different two loop sequences between the sense strand and the antisense strand, connecting both strands, wherein the sense strand and the antisense strand are paired to form a stem.
Owner:RIKEN +1

Modulation of exon recognition in pre-mRNA by interfering with the secondary RNA structure

The invention provides a method for generating an oligonucleotide with which an exon may be skipped in a pre-mRNA and thus excluded from a produced mRNA thereof. Further provided are methods for altering the secondary structure of an mRNA to interfere with splicing processes and uses of the oligonucleotides and methods in the treatment of disease. Further provided are pharmaceutical compositions and methods and means for inducing skipping of several exons in a pre-mRNA.
Owner:ACADEMISCH ZIEKENHUIS BIJ DE UNIV VAN AMSTERDAM ACADEMISCH MEDISCH CENT

Sustained-Release Lipid Pre-Concentrate of Pharmacologically Active Substance And Pharmaceutical Composition Comprising The Same

Disclosed is a sustained release lipid pre-concentrate, comprising: a) a sorbitan unsaturated fatty acid ester having a polar head with at least two or more —OH (hydroxyl) groups; b) a phospholipid; and c) a liquid crystal hardener, free of an ionizable group, having a hydrophobic moiety of 15 to 40 carbon atoms with a triacyl group or a carbon ring structure. The lipid pre-concentrate exists as a liquid phase in the absence of aqueous fluid and forms into a liquid crystal in the presence of aqueous fluid. Also, a pharmaceutical composition further comprising a pharmacologically active ingredient plus the pre-concentrate is provided.
Owner:CHONG KUN DANG PHARMA CORP

Methods and pharmaceutical compositions for the treatment of cardiomyopathy due to friedreich ataxia

A method for preventing or treating cardiomyopathy due to energy failure in a subject in need thereof is provided. The method comprises administering to the subject a therapeutically effective amount of a vector which comprises a nucleic acid sequence encoding a gene that can reverse energy failure. An exemplary cardiomyopathy is that which is associated with Friedreich ataxia and an exemplary nucleic acid sequence comprises a nucleic acid that encodes frataxin (FXN).
Owner:INST NAT DE LA SANTE & DE LA RECHERCHE MEDICALE (INSERM) +5

Adeno-associated virus variant capsids and methods of use thereof

Provided herein are variant adeno-associated virus (AAV) capsid proteins having one or more modifications in amino acid sequence relative to a parental AAV capsid protein, which, when present in an AAV virion, confer increased infectivity of one or more types of retinal cells as compared to the infectivity of the retinal cells by an AA V virion comprising the unmodified parental capsid protein. Also provided are recombinant AAV virions and pharmaceutical compositions thereof comprising a variant AAV capsid protein as described herein, methods of making these rAAV capsid proteins and virions, and methods for using these rAAV capsid proteins and virions in research and in clinical practice, for example in, e.g., the delivery of nucleic acid sequences to one or more cells of the retina for the treatment of retinal disorders and diseases.
Owner:4D MOLECULAR THERAPEUTICS INC

Methods and compositions for targeted delivery of gene therapeutic vectors

Embodiments of the present invention relate to methods and compositions for tissue- specific delivery of a gene therapeutic, transgenic nucleic acid in mammals. Methods and compositions of the invention include the steps of providing a nucleic acid comprising a transgene flanked by two terminal repeats and, within the same or on a separate nucleic acid, a nucleotide sequence encoding a transposase, wherein the transgene comprises a biotherapeutic gene, contacting the nucleic acid with perfluorocarbon gas-filled microbubbles to form a mixture, introducing the mixture into the bloodstream of a mammal, and focusing ultrasound pulses on a specific tissue of said mammal, wherein said pulses disrupt said microbubbles of said mixture and release said nucleic acid into the bloodstream within the target tissue, thereby enabling uptake of the transgenic nucleic acid into the cells of said target tissue.
Owner:UNIV OF HAWAII

Methods and pharmaceutical compositions for the treatment of cardiomyopathy due to friedreich ataxia

A method for preventing or treating cardiomyopathy due to energy failure in a subject in need thereof is provided. The method comprises administering to the subject a therapeutically effective amount of a vector which comprises a nucleic acid sequence encoding a gene that can reverse energy failure. An exemplary cardiomyopathy is that which is associated with Friedreich ataxia and an exemplary nucleic acid sequence comprises a nucleic acid that encodes frataxin (FXN).
Owner:INST NAT DE LA SANTE & DE LA RECHERCHE MEDICALE (INSERM) +5

Methods and compositions for nucleic acid and protein payload delivery

Provided are methods and compositions for delivering a nucleic acid, protein, and / or ribonucleoprotein payload to a cell. Also provided are delivery molecules that include a peptide targeting ligand conjugated to a protein or nucleic acid payload (e.g., an siRNA molecule), or conjugated to a charged polymer polypeptide domain (e.g., poly-arginine such as 9R or a poly-histidine such as 6H, and the like). The targeting ligand provides for (i) targeted binding to a cell surface protein, and (ii) engagement of a long endosomal recycling pathway. As such, when the targeting ligand engages the intended cell surface protein, the delivery molecule enters the cell (e.g., via endocytosis) but is preferentially directed away from the lysosomal degradation pathway.
Owner:LIGANDAL INC

Porous nanoparticle-supported lipid bilayers (protocells) for targeted delivery and methods of using same

The present invention is directed to protocells for specific targeting of hepatocellular and other cancer cells which comprise a nanoporous silica core with a supported lipid bilayer; at least one agent which facilitates cancer cell death (such as a traditional small molecule, a macromolecular cargo (e.g. siRNA or a protein toxin such as ricin toxin A-chain or diphtheria toxin A-chain) and / or a histone-packaged plasmid DNA disposed within the nanoporous silica core (preferably supercoiled in order to more efficiently package the DNA into protocells) which is optionally modified with a nuclear localization sequence to assist in localizing protocells within the nucleus of the cancer cell and the ability to express peptides involved in therapy (apoptosis / cell death) of the cancer cell or as a reporter, a targeting peptide which targets cancer cells in tissue to be treated such that binding of the protocell to the targeted cells is specific and enhanced and a fusogenic peptide that promotes endosomal escape of protocells and encapsulated DNA. Protocells according to the present invention may be used to treat cancer, especially including hepatocellular (liver) cancer using novel binding peptides (c-MET peptides) which selectively bind to hepatocellular tissue or to function in diagnosis of cancer, including cancer treatment and drug discovery.
Owner:NAT TECH & ENG SOLUTIONS OF SANDIA LLC +1

Implantable liposome embedded matrix composition, uses thereof, and polycaprolactone particles as scaffolds for tissue regeneration

In various embodiments, the present invention describes materials and methods for the local reprogramming of cells in a location where the treatment is applied. The invention can be used to replace lost cells or to restore function to tissue damaged due to disease, injury or genetic defect. In various embodiments, the treatment includes a semisolid hydrogel embedded with liposomes. The liposomes can contain an effector molecule or molecules. When phagocytic cells such as monocytes infiltrate the hydrogel, they encounter the liposomes and incorporate the liposomes carrying the effector molecules into the cells. In some embodiments, the effector molecules can be genetic material encoding the expression of specific proteins such as transcription factors, the expression of which can initiate the reprogramming of the cells. In other embodiments, the effector molecules can induce angiogenesis. In other embodiments, the effector molecules are tumor antigens. The matrix can contain other effector molecules designed to attract specific cells to the matrix. The cells can be released from the matrix as the matrix degrades or by active migration from the matrix. The cells can also remain in the matrix and secret molecules such as proteins and hormones that will diffuse through the matrix material to the surrounding tissue.
Owner:SCHUBERT HLDG

Dna-binding domain of crispr system, non-fucosylated and partially fucosylated proteins, and methods thereof

The present disclosure relates to a method of obtaining a cell where fucosylation pathways are modified, leading to production of partially fucosylated and non-fucosylated protein products, specifically antibodies from the cell. The present disclosure employs the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) technology. The method of the present disclosure targets the Fut8 gene and GMD gene in a cell. Such products are used in developing therapeutics and biomarkers, and in diagnosis and prognosis of diseases.
Owner:ZUMUTOR BIOLOGICS

Implantable liposome embedded matrix composition, uses thereof, and polycaprolactone particles as scaffolds for tissue regeneration

ActiveUS20150050332A1Organic active ingredientsPeptide/protein ingredientsDiseaseMononuclear cell infiltration
In various embodiments, the present invention describes materials and methods for the local reprogramming of cells in a location where the treatment is applied. The invention can be used to replace lost cells or to restore function to tissue damaged due to disease, injury or genetic defect. In various embodiments, the treatment includes a semisolid hydrogel embedded with liposomes. The liposomes can contain an effector molecule or molecules. When phagocytic cells such as monocytes infiltrate the hydrogel, they encounter the liposomes and incorporate the liposomes carrying the effector molecules into the cells. In some embodiments, the effector molecules can be genetic material encoding the expression of specific proteins such as transcription factors, the expression of which can initiate the reprogramming of the cells. In other embodiments, the effector molecules can induce angiogenesis. In other embodiments, the effector molecules are tumor antigens. The matrix can contain other effector molecules designed to attract specific cells to the matrix. The cells can be released from the matrix as the matrix degrades or by active migration from the matrix. The cells can also remain in the matrix and secret molecules such as proteins and hormones that will diffuse through the matrix material to the surrounding tissue.
Owner:BONUS CELLORA LTD

Systems and Methods for Nucleic Acid Expression In Vivo

The present invention provides compositions, systems, kits, and methods for generating expression of one or more proteins and / or biologically active nucleic acid molecules in a subject (e.g., at therapeutic levels for extended periods required to produce therapeutic effects). In certain embodiments, systems and kits are provided that comprise a first composition comprising a first amount of polycationic structures, and a second composition comprising a therapeutically effective amount of expression vectors (e.g., non-viral expression vectors not associated with liposomes) that are CpG-free or CpG-reduced, where the expression vectors comprise a first nucleic acid sequence encoding: i) a first therapeutic protein or proteins, and / or ii) a first biologically active nucleic acid molecule or molecules.
Owner:DNARX

Cell proliferation inhibitory proteins and polynucleotides, antisense polynucleotides to the polynucleotides, cell proliferation inhibitors using the foregoing, cancer diagnostic agents, cancer therapeutic agents and compositions for gene therapy

The genes the expression of which is reduced or disappeared in immortal cells including cancer cells are isolated, their DNA sequences are determined, the genes are expressed to produce cell proliferation inhibitory proteins, and the genes and the proteins are utilized as agents for diagnosis or treatment, including the genetic diagnosis of or the gene therapy of diseases such as cancer.
Owner:HIROMI KUMON

Targeted non-viral DNA insertions

Provided herein are methods and compositions for editing the genome of a cell. In some embodiments, a nucleotide sequence of at least 200 nucleotides in length is inserted into a target region in the genome of a cell.
Owner:RGT UNIV OF CALIFORNIA

Short Non-Coding Protein Regulatory RNAs (sprRNAs) and Methods of Use

The present invention provides isolated nucleic acid molecules comprising short non-coding protein regulatory RNAs (sprRNA), variants, fragments and inhibitors thereof and compositions and methods of using the same.
Owner:UNIV OF MARYLAND BALTIMORE

Nucleic acid molecules and uses thereof for non-viral gene therapy

The present disclosure provides nucleic acid molecules comprising a first inverted terminal repeat (ITR), a second ITR, and a genetic cassette encoding a target sequence. In some embodiments, the target sequence encodes a miRNA and / or a therapeutic protein. In certain embodiments, the therapeutic protein comprises a clotting factor, a growth factor, a hormone, a cytokine, an antibody, a fragment thereof, and a combination thereof. In some embodiments, the first ITR and / or the second ITR is an ITR of a non-adeno-associated virus (AAV). The present disclosure also provides methods of treating a metabolic disorder of the liver in a subject comprising administering to the subject the nucleic acid molecule or a polypeptide encoded thereby.
Owner:BIOVERATIV THERAPEUTICS INC

Compositions and methods for modulating fmr1 expression

PendingUS20180256749A1Decrease disease symptomatologyMore chromatin stateOrganic active ingredientsNervous disorderFmr1 geneFMRP Protein
The disclosure relates to methods and compositions for reactivating a silenced FMR1 gene. In some aspects, methods described by the disclosure are useful for treating a FMR1-inactivation-associated disorder (e.g., fragile X syndrome).
Owner:UNIV OF MASSACHUSETTS +1

Nucleic acid therapy to enhance cartilage repair

InactiveUS20050197304A1Promote repairFacilitating regeneration of cartilagePowder deliveryCarbohydrate active ingredientsDNA constructActive agent
The present invention relates to the application of nucleic acid therapy for the repair and regeneration of cartilage. The invention encompasses the introduction of naked DNA encoding bioactive agent(s), whose expression stimulates and otherwise facilitates the repair and regeneration of cartilage. The present invention provides DNA constructs for introduction to the site of cartilage damage. Pharmaceutical compositions comprising nucleic acid encoding one or more bioactive factor, optionally with a matrix or polymer, are provided. Methods for expression of bioactive agent(s) are provided. Methods for enhancing cartilage repair and / or regeneration comprising introduction of bioactive factors as naked DNA are further provided. Methods for the treatment or prevention of cartilage damage in various orthopaedic and rheumatologic conditions are provided.
Owner:NEW YORK SCHOOL OF MEDICINE

Applications of human PTP4A3 gene and related drugs thereof

The invention discloses applications of human PTP4A3 gene and related drugs thereof. Applications of human PTP4A3 gene in tumor treatment, tumor diagnosis, and drug preparation are disclosed. The invention further constructs human PTP4A3 gene micromolecule interference RNA, human PTP4A3 gene interference nucleic acid construct, and human PTP4A3 gene interference slow virus, and discloses the applications thereof. The provided siRNA or nucleic acid construct containing the siRNA and slow virus can inhibit the expression of human PTP4A3 gene specifically, especially the slow virus can infect target cells efficiently and inhibit the expression of PTP4A3 gene in target cells efficiently, thus the tumor cell growth is inhibited, apoptosis of tumor cells is promoted, and thus the provided siRNA, nucleic acid construct, and slow virus have an important meaning in tumor treatment.
Owner:SHANGHAI GENECHEM

Adenoassociated virus vectors for the treatment of mucopolysaccharidoses

The present invention provides new Adeno-associated virus-derived vectors and pharmaceutical compositions containing the same for the treatment of lysosomal storage disorders and specially, for the treatment of mucopolysaccharidoses Type II.
Owner:ESTEVE PHARMA SA +1

Intravascular delivery of non-viral nucleic acid

Disclosed is a process for providing for expression of an exogenous nucleic acid in an extravascular parenchymal cell of a mammal. The nucleic acid is inserted into a vessel of a mammal and the permeability of the vessel is increased. Increasing permeability of the vessel allows delivery of the nucleic acid to an extravascular parenchymal cell.
Owner:WOLFF JON A +7

An mRNA cancer vaccine encoding human GM-CSF fused to multiple tandem epitopes

The present invention provides an mRNA cancer vaccine encoding human GM-CSF fused to multiple tandem epitopes. pVec-GM-CSF-hTes encoding human GM-CSF fused to three tandem hTERT epitopes, pVec-GMKE encoding human GM-CSF fused to three tandem epitopes respectively from MUC1, Kras and EGFR, pVec-hIL-12 encoding human interleukin-12 are respectively constructed, and used as templates for generating the corresponding in vitro transcribed mRNAs, which are mixed together as an mRNA cancer vaccine. This mRNA cancer vaccine contains human GM-CSF used as an immune adjuvant, multiple tandem epitopes constituting as multi-epitope cancer antigens and hIL-12 used to enhance the immunotherapeutic effects.
Owner:DING ENYU
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