The Polynucleotide Is Coated With Or Encapsulated Within A Lipid Containing Material (e.g., Liposome, Etc.) Patents (Class 435/458)
  • Patent number: 10227589
    Abstract: Liposomal compositions comprising of liposomes of guanidinylated cationic amphiphiles as the main lipid and cholesterol/1,2-dioleoyl-sn-glycero-3-phosphocholine (DOPC)/aminopropyl polyethyleneglycol carbamyl-distearoylphosphatidyl-ethanolamine (DSPE-peg-NH2) as co-lipids are described. These liposomal compositions containing encapsulated or electrostatically complexed therapeutic small interfering RNAs (siRNAs) against Cdc20, a key cell cycle regulator, inhibit solid tumor growth and melanoma tumor growth on lung in C57BL/6J mice.
    Type: Grant
    Filed: January 28, 2013
    Date of Patent: March 12, 2019
    Assignee: COUNCIL OF SCIENTIFIC AND INDUSTRIAL RESEARCH
    Inventors: Anubhab Mukherjee, Jayanta Bhattacharyya, Arabinda Chaudhuri
  • Patent number: 10143758
    Abstract: Disclosed herein are compositions and methods of modulating the expression of gene or the production of a protein by transfecting target cells with nucleic acids. The compositions disclosed herein demonstrate a high transfection efficacy and are capable of ameliorating diseases associated with protein or enzyme deficiencies.
    Type: Grant
    Filed: March 13, 2013
    Date of Patent: December 4, 2018
    Assignee: Translate Bio, Inc.
    Inventors: Braydon Charles Guild, Frank DeRosa, Michael Heartlein
  • Patent number: 9999671
    Abstract: Compositions, methods, strategies, kits, and systems for the delivery of negatively charged proteins, protein complexes, and fusion proteins, using cationic polymers or lipids are provided. Delivery of proteins into cells can be effected in vivo, ex vivo, or in vitro. Proteins that can be delivered using the compositions, methods, strategies, kits, and systems provided herein include, without limitation, enzymes, transcription factors, genome editing proteins, Cas9 proteins, TALEs, TALENs, nucleases, binding proteins (e.g., ligands, receptors, antibodies, antibody fragments; nucleic acid binding proteins, etc.), structural proteins, and therapeutic proteins (e.g., tumor suppressor proteins, therapeutic enzymes, growth factors, growth factor receptors, transcription factors, proteases, etc.), as well as variants and fusions of such proteins.
    Type: Grant
    Filed: October 30, 2014
    Date of Patent: June 19, 2018
    Assignee: President and Fellows of Harvard College
    Inventors: David R. Liu, David B. Thompson, John Anthony Zuris
  • Patent number: 9914926
    Abstract: Methods of treating a wound in a subject are provided comprising administering to the subject an amount of an inhibitor of Fidgetin-like 2. Compositions and pharmaceutical compositions comprising an amount of an inhibitor of Fidgetin-like 2 are also provided. Methods are also provided for identifying an inhibitor of Fidgetin-like 2.
    Type: Grant
    Filed: March 1, 2016
    Date of Patent: March 13, 2018
    Assignee: Albert Einstein College of Medicine, Inc.
    Inventors: David J. Sharp, Rabab Charafeddine
  • Patent number: 9849086
    Abstract: A medicament for treating cystitis and a method of treatment are provided. An agent to repair the damaged glycosaminoglycan (“GAG”) layers lining the urothelium, noted in cystitis, such as pentosan polysulfate, hyaluronic acid, chondroitin, etc., is provided in a liposomal carrier. Cystitis is treated by intravesically administering to a patient a therapeutically effective dose of the medicament.
    Type: Grant
    Filed: March 13, 2013
    Date of Patent: December 26, 2017
    Assignee: Nanologix Research, Inc.
    Inventors: Elliot B. Lander, Jackie R. See
  • Patent number: 9783821
    Abstract: A cell of the present invention contains a nucleic acid construct encoding a WT1 gene product or a fragment of the WT1 gene product. The nucleic acid construct contains (i) a region encoding a desired fragment of the WT1 gene product and (ii) only AUG as a functional start codon. The present invention can provide a cell into which the nucleic acid construct is introduced so that an expression level of a WT1 gene product or a fragment of the WT1 gene product is remarkably enhanced.
    Type: Grant
    Filed: July 30, 2012
    Date of Patent: October 10, 2017
    Assignee: RIKEN
    Inventors: Shin-ichiro Fujii, Kanako Shimizu, Jun Shinga
  • Patent number: 9696315
    Abstract: A protein transduction method for efficiently delivery of exogenous proteins into mammalian cells is invented, which has the capability of targeting different cellular compartments and protection from degradation of the delivered proteins from cellular proteases. A composition for treat proteins has cation reagents, lipids and enhancers in a carrier. The method can be used in a number of ways including: production of large quantities of properly folded, post-translationally modified proteins using mammalian cell machinery, a in-cell fluorescence spectroscopy and imaging using small molecule fluorophores and a in-cell NMR spectroscopy using living mammalian cells. The method permits cell biology at atomic resolution that is physiologically and pathological relevant and permits protein therapy to treat human diseases. The method can also be used to deliver exogenous protein inside mammalian cells, wherein the exogenous proteins follow a similar secretion pathway as that of the endogenous protein.
    Type: Grant
    Filed: April 25, 2014
    Date of Patent: July 4, 2017
    Assignee: Wayne State University
    Inventors: Jianjun Wang, Qianqian Li
  • Patent number: 9670489
    Abstract: The present disclosure provides a method for treating and/or preventing myopia, including: administering an RNA interference (RNAi) to a subject, wherein the RNA interference is capable of counteracting another RNA interference, and the other RNA interference is an RNA interference capable of inhibiting an expression of PAX-6 gene, and the RNA interference capable of inhibiting an expression of PAX-6 gene comprises microRNA-328.
    Type: Grant
    Filed: March 31, 2015
    Date of Patent: June 6, 2017
    Assignee: Kaohsiung Medical University
    Inventors: Suh-Hang Juo, Ku-Chung Chen, Edward Hsi, Chung-Ling Liang
  • Patent number: 9655848
    Abstract: The present invention provides a liposome based composition wherein the liposome includes a peptide conjugated thereto via a peptide bond, wherein the peptide includes a spacer amino acid and a short Apolipoprotein E recognition sequence or a short Amyloid beta recognition sequence. This invention further provides a process for making the liposome and methods of utilizing the liposome based composition for therapeutic and diagnostic purposes.
    Type: Grant
    Filed: November 19, 2013
    Date of Patent: May 23, 2017
    Assignee: Technion Research & Development Foundation Limited
    Inventors: Nahum Allon, Moshe Gavish
  • Patent number: 9566295
    Abstract: The invention relates to a double-stranded ribonucleic acid (dsRNA) targeting a G-alpha q subunit (GNAQ) of a heterotrimeric G gene, and methods of using the dsRNA to inhibit expression of GNAQ.
    Type: Grant
    Filed: October 6, 2014
    Date of Patent: February 14, 2017
    Assignee: Alnylam Pharmaceuticals, Inc.
    Inventors: Jared Gollob, Gregory Hinkle, Ivanka Toudjarska, David Bumcrot
  • Patent number: 9481888
    Abstract: The present invention provides a recombinant bacterium and methods of using the recombinant bacterium to induce an immune response.
    Type: Grant
    Filed: November 22, 2011
    Date of Patent: November 1, 2016
    Assignee: The Arizona Board of Regents for and on Behalf of Arizona State University
    Inventors: Roy Curtiss, III, Wei Kong
  • Patent number: 9439856
    Abstract: Disclosed is an effective method of preparing a composition for delivering an anionic drug, the composition comprising an anionic drug as an active ingredient, a cationic lipid, and an amphiphilic block copolymer, wherein the anionic drug forms a complex with the cationic lipid, and the anionic drug/cationic lipid complex is entrapped in a micelle structure formed by the amphiphilic block copolymer.
    Type: Grant
    Filed: April 3, 2013
    Date of Patent: September 13, 2016
    Assignee: Samyang Biopharmaceuticals Corporation
    Inventors: Muhn-Ho La, Ji-Yeon Son, Sang-Hoon Kim
  • Patent number: 9394542
    Abstract: The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of Discs large homolog (DLG), in particular, by targeting natural antisense polynucleotides of Discs large homolog (DLG). The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorder associated with the expression of DLG.
    Type: Grant
    Filed: January 22, 2015
    Date of Patent: July 19, 2016
    Assignee: CuRNA, Inc.
    Inventors: Joseph Collard, Olga Khorkova Sherman, Carlos Coito
  • Patent number: 9330295
    Abstract: Methods, articles and systems that provide imagewise mapping or display of gene expression of a biosample, by contacting the biosample, such as a tissue slice or metacommunity, to a detector which captures material from the biosample and processes the captured material. In one embodiment the detector has an array of one or more capture sites at defined positions on the detector, each site carrying an immobilized capture oligonucleotide and a site-indexing oligonucleotide. The array captures mRNA from the biosample contacted thereto, and the captured mRNA is processed to form a sequenceable amount of amplified captured material which includes the site-indexing oligonucleotide, so that when sequenced, detection of the site-indexing oligonucleotide indicates the original capture location on the array, thereby mapping the sequenced material to its capture location and imaging display of gene expression distribution in the original biosample.
    Type: Grant
    Filed: March 17, 2014
    Date of Patent: May 3, 2016
    Assignee: BROWN UNIVERSITY
    Inventor: Casey Dunn
  • Patent number: 9220779
    Abstract: Disclosed are a carrier for delivering a negatively charged drug, comprising a cationic lipid represented by formula 1, and a preparation method thereof. Also disclosed is a pharmaceutical composition comprising a negatively charged drug and a cationic lipid represented by formula 1, wherein the negatively charged drug forms a complex with the cationic lipid. The composition can increase the in vivo stability of the negatively charged drug after local or systemic administration and allows the intracellular delivery of the negatively charged drug. Thus, the composition will be useful for improving the therapeutic effect of the negatively charged drug.
    Type: Grant
    Filed: December 30, 2011
    Date of Patent: December 29, 2015
    Assignee: SAMYANG BIOPHARMACEUTICALS CORPORATION
    Inventors: Sung-Won Choi, Muhn-Ho La, Ji-Yeon Son, Min-Hyo Seo
  • Patent number: 9206420
    Abstract: This invention relates to compounds, compositions, and methods useful for reducing HIF-1? target RNA and protein levels via use of dsRNAs, e.g., Dicer substrate siRNA (DsiRNA) agents.
    Type: Grant
    Filed: July 15, 2013
    Date of Patent: December 8, 2015
    Assignee: Dicerna Pharmaceuticals, Inc.
    Inventors: Bob Dale Brown, Henryk T. Dudek
  • Patent number: 9173840
    Abstract: Libraries of nanoparticles comprising therapeutic agents and/or imaging agents are disclosed, as well as methods of making, customizing, and using such libraries of nanoparticles.
    Type: Grant
    Filed: October 9, 2009
    Date of Patent: November 3, 2015
    Assignee: Northeastern University
    Inventors: Mansoor M. Amiji, Arun K. Iyer
  • Patent number: 9057067
    Abstract: A method for transfecting nucleic acid to cell comprising a step for forming a nucleic acid complex by bringing a double-stranded nucleic acid molecule into contact with a nucleic acid carrier having an amino acid sequence of alternating a basic amino acid and a hydrophobic amino acid, which has a peptide chain that forms a ?-sheet structure in which a side chain of a positively charged basic amino acid is disposed on one surface side and a side chain of a hydrophobic amino acid is disposed on the opposite surface side in the presence of the double-stranded nucleic acid molecule having a double helix structure, and by binding the double-stranded nucleic acid molecule and the peptide chain through either one or both of the electrostatic interaction between the side chains of the basic amino acid and phosphate groups and hydrogen bonds between the double stranded-nucleic acid molecule and the peptide chain and a nucleic acid complex used for the same are disclosed.
    Type: Grant
    Filed: July 7, 2011
    Date of Patent: June 16, 2015
    Assignee: Kinki University
    Inventor: Masayuki Fujii
  • Patent number: 9029604
    Abstract: The instant invention provides for novel cationic lipids that can be used in combination with other lipid components such as cholesterol and PEG-lipids to form lipid nanoparticles with oligonucleotides. It is an object of the instant invention to provide a cationic lipid scaffold that demonstrates enhanced efficacy along with lower liver toxicity as a result of lower lipid levels in the liver. The present invention employs low molecular weight cationic lipids with one short lipid chain to enhance the efficiency and tolerability of in vivo delivery of siRNA.
    Type: Grant
    Filed: September 28, 2011
    Date of Patent: May 12, 2015
    Assignee: Sirna Therapeutics, Inc.
    Inventors: John A. Bawiec, III, Zhengwu J. Deng
  • Patent number: 9029338
    Abstract: The invention relates to lipid formulated double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of a gene from the Ebola virus.
    Type: Grant
    Filed: August 13, 2010
    Date of Patent: May 12, 2015
    Assignee: Alnylam Pharmaceuticals, Inc.
    Inventors: Antonin de Fougerolles, Anna Borodovsky, Tatiana Novobrantseva
  • Patent number: 9012225
    Abstract: Disclosed are lipophilic polynucleotide conjugates including polynucleotide-cholesterol conjugates and methods of delivering therapeutic polynucleotides to a mammalian cell or patient in need of treatment using said conjugates. The disclosure further provides methods of synthesizing the lipophilic polynucleotide conjugates. The conjugates are designed to mimic or target cellular miRNAs. The lipophilic moiety, such as cholesterol or cholesterol derivative, is spaced from the polynucleotide by a substantially linear hydrocarbon group. Due to an absence of significantly polar groups and/or exchangeable protons in the vicinity of the lipophilic moiety, the interaction between the lipophilic moiety and cell membranes is enhanced to provide for efficient entry into cells.
    Type: Grant
    Filed: May 5, 2010
    Date of Patent: April 21, 2015
    Assignee: miRagen Therapeutics
    Inventors: Kurt Vagle, William S. Marshall
  • Patent number: 9006191
    Abstract: The present invention provides compositions comprising interfering RNA (e.g., siRNA, aiRNA, miRNA) that target polo-like kinase 1 (PLK-1) expression and methods of using such compositions to silence PLK-1 expression. More particularly, the present invention provides unmodified and chemically modified interfering RNA molecules which silence PLK-1 expression and methods of use thereof. The present invention also provides serum-stable nucleic acid-lipid particles (e.g., SNALP) comprising an interfering RNA molecule described herein, a cationic lipid, and a non-cationic lipid, which can further comprise a conjugated lipid that inhibits aggregation of particles. The present invention further provides methods of silencing PLK-1 gene expression by administering an interfering RNA molecule described herein to a mammalian subject. The present invention additionally provides methods of identifying and/or modifying PLK-1 interfering RNA having immunostimulatory properties.
    Type: Grant
    Filed: December 23, 2008
    Date of Patent: April 14, 2015
    Assignee: Protiva Biotherapeutics, Inc.
    Inventors: Ian MacLachlan, Adam Judge
  • Patent number: 9006406
    Abstract: An epidermal growth factor receptor (EGFR)-homing vector comprising a double-stranded RNA (dsRNA) molecule with an EGFR-binding peptide or polypeptide, is disclosed for use in combination with immune cells for treatment of cancer overexpressing EGFR.
    Type: Grant
    Filed: December 22, 2009
    Date of Patent: April 14, 2015
    Inventors: Alexander Levitzki, Alexei Shir
  • Patent number: 8993327
    Abstract: Systems and methods are described for parallel macromolecular delivery and biochemical/electrochemical interface to whole cells employing carbon nanostructures including nanofibers and nanotubes. A method includes providing a first material on at least a first portion of a first surface of a first tip of a first elongated carbon nanostructure; providing a second material on at least a second portion of a second surface of a second tip of a second elongated carbon nanostructure, the second elongated carbon nanostructure coupled to, and substantially parallel to, the first elongated carbon nanostructure; and penetrating a boundary of a biological sample with at least one member selected from the group consisting of the first tip and the second tip.
    Type: Grant
    Filed: April 7, 2003
    Date of Patent: March 31, 2015
    Assignee: UT-Battelle, LLC
    Inventors: Timothy E. McKnight, Anatoli V. Melechko, Guy D. Griffin, Michael A. Guillorn, Vladimir L. Merkulov, Michael L. Simpson
  • Publication number: 20150089681
    Abstract: This disclosure provides for compositions and methods for the use of designed nucleic acid-targeting nucleic acids, Argonautes, and complexes thereof.
    Type: Application
    Filed: April 10, 2014
    Publication date: March 26, 2015
    Applicant: Caribou Biosciences, Inc.
    Inventors: John VAN DER OOST, Daniël Christianus SWARTS, Andrew Paul MAY, Rachel E. HAURWITZ
  • Publication number: 20150086617
    Abstract: The present application relates to compositions comprising and methods of using a liposome comprising a pHLIP polypeptide, wherein a lipid bilayer of the liposome is substantially free of the pHLIP polypeptide.
    Type: Application
    Filed: September 29, 2014
    Publication date: March 26, 2015
    Applicant: YALE UNIVERSITY
    Inventors: Yana K. Reshetnyak, Oleg A. Andreev, Donald M. Engelman
  • Patent number: 8975079
    Abstract: Provided herein are biodegradable copolymers and nanoplex delivery systems comprising the same and a cargo molecule, such as a nucleic acid, a polynucleotide or other biomolecule. The biodegradable copolymers may comprise a reducible polymer linearly modified with lysine, such as a linear lysine-modified N,N?-cystamine bisacrylamide. The biodegradable copolymers also may be conjugated to a sequestering moiety, such as dietheylamine. The biodegradable copolymers also may comprise one or both of a targeting moiety and one or more moieties to facilitate endosomal escape. Also provided are methods for treating a pathophysiological condition and for increasing biocompatibility of a polymeric delivery system upon delivery to a subject using the biodegradable copolymers and nanoplexes.
    Type: Grant
    Filed: February 11, 2010
    Date of Patent: March 10, 2015
    Assignee: University of Houston System
    Inventor: Malavosklish Bikram
  • Publication number: 20150056226
    Abstract: The present invention is directed to adoptive immunotherapy using a lymphocyte in which an antigen-specific receptor and a bioactive material gene such as an IL-2 gene or a water-soluble TGF-beta receptor gene are transferred. The bioactive material is intensively secreted to, for example, a local site of a tumor, thereby reducing systemic side effects as much as possible, and the survival time of the lymphocyte is increased, thereby further improving the effect of the adoptive immunotherapy.
    Type: Application
    Filed: September 8, 2014
    Publication date: February 26, 2015
    Inventor: Tai-Gyu KIM
  • Patent number: 8956873
    Abstract: The present invention is directed generally to eukaryotic host cells comprising artificial endosymbionts and methods of introducing artificial endosymbionts into eukaryotic host cells. The invention provides artificial endosymbionts that introduce a phenotype to host cells that is maintained in daughter cells. The invention additionally provides eukaryotic host cells containing magnetotactic bacteria.
    Type: Grant
    Filed: January 13, 2012
    Date of Patent: February 17, 2015
    Assignee: Bell Biosystems, Inc.
    Inventors: Caleb B. Bell, III, Alexey V. Bazarov
  • Patent number: 8951511
    Abstract: The present invention relates to a composition for improving the migration potential of a stem cell, a method for evaluating the migration potential of a stem cell and a method for screening an adjuvant of cell therapy improving the migration potential of a stem cell. The present invention may be effectively used for enhancing the efficacy of neurological disease-treatment by inducing therapeutic stem cells to migrate efficiently to the lesion site.
    Type: Grant
    Filed: October 19, 2010
    Date of Patent: February 10, 2015
    Assignee: Corestem Co., Ltd.
    Inventors: Seong Ho Koh, Seung Hyun Kim, Goang Won Cho, Min Young Noh, Kyung Suk Kim
  • Publication number: 20150037435
    Abstract: Provided are a culture medium of an adipose-derived stem cell, a method for preparing the same, and a composition for promoting hair growth, in which the composition includes the culture medium. The adipose-derived stem cell (ADSC-T) according to the present invention exhibits long lifespan, improved cell proliferation rate, and extended proliferation period, as compared with a primary adipose-derived stem cell (ADSC), and thus, the adipose-derived stem cell (ADSC-T) can be usefully used for the study about the adipose-derived stem cell and the mass production of the culture medium of the adipose-derived stem cell. In addition, according to the present invention, the culture medium of the adipose-derived stem cell (ADSC-T) that expresses a T antigen of SV40 exhibits excellent hair growth effectiveness and can be usefully used as a raw material for the hair loss prevention and hair growing agents.
    Type: Application
    Filed: August 29, 2012
    Publication date: February 5, 2015
    Inventors: Dong Wan Kim, Mi Jung Seo, Gwang Lee, Woo Hong Joo, Sun Hee Kim
  • Patent number: 8945927
    Abstract: The present invention relates to a new class of cationic polymers that self-assemble with a pH-sensitive dissolution switch, and their uses to deliver molecules of interest to a cell. The present invention also relates to compositions comprising said cationic polymers non-covalently associated with a molecule of interest, in particular with a siRNA.
    Type: Grant
    Filed: March 29, 2011
    Date of Patent: February 3, 2015
    Assignees: Universite de Strasbourg, Centre National de la Recherche Scientifique
    Inventors: Guy Zuber, Benoit Frisch, Gaelle Creusat, Jean-Sebastien Thomann
  • Patent number: 8936942
    Abstract: The present invention provides compositions comprising polytheylyene-dialkyloxypropyl conjugates (PEG-DAA), liposomes, SNALP, and SPLP comprising such compositions, and methods of using such compositions, liposomes, SNALP, and SPLP.
    Type: Grant
    Filed: August 6, 2010
    Date of Patent: January 20, 2015
    Assignee: Protiva Biotherapeutics, Inc.
    Inventors: James Heyes, Ian MacLachlan, Ellen Grace Ambegia
  • Patent number: 8920832
    Abstract: The invention provides a nucleic-acid-transfecting composition which exhibits low cytotoxicity, which facilitates an effective nucleic acid transfection into a cell, and which improves expression of the nucleic acid in the cell. The composition for transfecting a nucleic acid into a cell, contains a di(C12-16 alkyl)dimethylammonium halide and a phospholipid.
    Type: Grant
    Filed: September 29, 2011
    Date of Patent: December 30, 2014
    Assignee: Hokkaido System Science Co., Ltd.
    Inventors: Hiroshi Kikuchi, Hideo Kobayashi, Kouichi Hashimoto, Ayako Iijima, Daigo Asano, Junko Yasuda
  • Patent number: 8916530
    Abstract: In certain embodiments, the invention provides methods for treating cancer, comprising: (a) obtaining a specimen of cancer tissue and normal tissue from a patient; (b) extracting total protein and RNA from the cancer tissue and normal tissue; (c) obtaining a protein expression profile of the cancer tissue and normal tissue; (d) identifying over-expressed proteins in the cancer tissue; (e) comparing the protein expression profile to a gene expression profile; (f) identifying at least one prioritized protein target by assessing connectivity of each said over-expressed protein to other cancer-related or stimulatory proteins; (g) designing a first RNA interference expression cassette to modulate the expression of at least one gene encoding the prioritized target protein; (h): designing a first RNA interference expression cassette to modulate the expression of at least one gene encoding a protein of higher priority in the signaling pathway in which the first protein is a component; (i) incorporating the first cass
    Type: Grant
    Filed: October 30, 2009
    Date of Patent: December 23, 2014
    Assignee: Gradalis, Inc.
    Inventors: David Shanahan, John Nemunaitis, Neil Senzer, Phillip Maples, Donald Rao
  • Publication number: 20140370081
    Abstract: The invention relates to enantiomerically pure DOTAP chloride and stable crystal modifications of (2R,S)-, (25)- and (2R)-DOTAP chloride, to a process for the preparation of these modifications, and to the use thereof as constituent for the preparation of medicaments.
    Type: Application
    Filed: September 2, 2014
    Publication date: December 18, 2014
    Applicant: Merck Patent GmbH
    Inventors: Michael Platscher, Alfred Hedinger
  • Publication number: 20140370500
    Abstract: The embodiments herein provide a nanoparticle system for targeted gene delivery or a drug delivery and a method of synthesising the same. The nanoparticle composition for targeted gene transfer and drug delivery comprises a protein, a chitosan and a lipid. A method of synthesizing the nanoparticles involves preparing a gelatine and chitosan gel. A milky colloid solution is prepared with the gelatine, chitosan solution and a phosphatidylcholine. The milky colloid is homogenized for the self assembly of the nanoparticles. The milky colloid is subjected to high speed and high pressure homogenizer. The CHO cells are transfected with nanoparticles and lipofectamine 2000 for comparing the transfection efficiency. The nanoparticles deliver DNA, RNA, ribozyme and nucleotide sequences. The nanoparticles deliver lipophylic and hydrophilic drugs. The transfection efficiency of gene and drug is higher when the target cells are transferred with nanoparticles, compared to the cells transferred with lipofectamine 2000.
    Type: Application
    Filed: September 3, 2014
    Publication date: December 18, 2014
    Inventors: Jalaledin Ghanavi, Poopak Farnia
  • Patent number: 8906390
    Abstract: Compositions and methods for delivering cargo to cells are provided. One aspect provides a synthetic vesicle containing caveolin 1 or a fragment thereof in an amount effective to form lipid rafts in the vesicle. The synthetic vesicles can be used to deliver polynucleotides, proteins, therapeutic agents, or a combination thereof to specific membrane-bound compartments of a cell. In certain aspects, the synthetic vesicles can deliver cargo to cellular organelles such as mitochondria.
    Type: Grant
    Filed: March 26, 2007
    Date of Patent: December 9, 2014
    Assignee: Gencia Corporation
    Inventor: Shaharyar Khan
  • Patent number: 8889181
    Abstract: The present invention relates to a composition for enhancing an immune response, an epitope having immunogenicity, screening and preparing method thereof, a antibody to peptide antigen and screening and preparing method thereof. The composition of the present invention may be effectively used for preventing or treating diverse immune-deficiency diseases such as cancer, influenza virus, hepatitis C virus and RSV (respiratory syncytial virus) by enhancing immune responses.
    Type: Grant
    Filed: June 16, 2010
    Date of Patent: November 18, 2014
    Assignee: Industry Academic Cooperation Foundation, Hallym University
    Inventor: Hyung-Joo Kwon
  • Patent number: 8889644
    Abstract: The invention relates to a double-stranded ribonucleic acid (dsRNA) targeting a G-alpha q subunit (GNAQ) of a heterotrimeric G gene, and methods of using the dsRNA to inhibit expression of GNAQ.
    Type: Grant
    Filed: September 13, 2012
    Date of Patent: November 18, 2014
    Assignee: Alnylam Pharmaceuticals, Inc.
    Inventors: Jared Gollob, Greg Hinkle, Ivanka Toudjarska, David Bumcrot
  • Publication number: 20140335157
    Abstract: The present invention relates to a compound represented by the formula (1) wherein Xa and Xb are each independently X1 or X2; s is 1 or 2, R4 is an alkyl group having 1-6 carbon atoms, na and nb are each independently 0 or 1, R1a and R1b are each independently an alkylene group having 1-6 carbon atoms, R2a and R2b are each independently an alkylene group having 1-6 carbon atoms, Ya and Yb are each independently an ester bond, an amide bond, a carbamate bond, an ether bond or a urea bond, and R3a and R3b are each independently a sterol residue, a liposoluble vitamin residue or an aliphatic hydrocarbon group having 12-22 carbon atoms, and use thereof.
    Type: Application
    Filed: May 16, 2014
    Publication date: November 13, 2014
    Inventors: Kota TANGE, Masaya ARAI, Kazuhiro KUBO, Hidetaka AKITA, Hideyoshi HARASHIMA, Hiroto HATAKEYAMA, Ryohei ISHIBA, Masami UKAWA, Hiroki TANAKA
  • Patent number: 8853181
    Abstract: Methods of treating a wound in a subject are provided comprising administering to the subject an amount of an inhibitor of Fidgetin-like 2. Compositions and pharmaceutical compositions comprising an amount of an inhibitor of Fidgetin-like 2 are also provided. Methods are also provided for identifying an inhibitor of Fidgetin-like 2.
    Type: Grant
    Filed: July 19, 2012
    Date of Patent: October 7, 2014
    Assignee: Albert Einstein College of Medicine of Yeshiva University
    Inventors: David J. Sharp, Rabab Charafeddine
  • Patent number: 8852472
    Abstract: The present invention is related to a lipid composition comprising at least a first lipid component, at least a first helper lipid, and a shielding compound which is removable from the lipid composition under in vivo conditions.
    Type: Grant
    Filed: December 27, 2005
    Date of Patent: October 7, 2014
    Assignee: Silence Therapeutics GmbH
    Inventors: Oliver Keil, Jörg Kaufmann
  • Publication number: 20140249212
    Abstract: Compositions for transient but prolonged exogenous mRNA expression through the use of the transcription system of negative strand RNA viruses, and methods of use thereof are disclosed. In some embodiments, the system contains only RNAs and does not include any DNA molecules. The compositions typically include an RNA template unit (rTeUn) that includes a virus regulatory sequences operably linked to a coding sequence of interest. The rTeUn is typically transfected to a host cell's cytoplasm in the presence of virus expression system proteins that mediate replication of the rTeUn and transcription of the transgene. The rTeUn RNA bonded to viral proteins exhibits high resistance to degradation, prolonged duration of expression, and is free of viral genes. The compositions can be used to reprogram cell. For example, the compositions and methods can be used to redirected lymphocytes to target cancer cells, or to dedifferentiate somatic cells into induce pluripotent stem cells.
    Type: Application
    Filed: September 27, 2012
    Publication date: September 4, 2014
    Applicant: Yale University
    Inventors: Peter M. Rabinovich, Sherman M. Weissman
  • Patent number: 8821922
    Abstract: The present invention provides liposomal compositions and methods of using such compositions in vitro and in vivo. In particular, the present invention provides stable, polymer-caged liposomes comprising a pH responsive delivery mechanism for delivery of nucleic acids, peptides, small molecules, drugs, etc. in vitro and in vivo.
    Type: Grant
    Filed: June 9, 2008
    Date of Patent: September 2, 2014
    Assignee: Northwestern University
    Inventors: Sang-Min Lee, SonBinh Nguyen, Thomas V. O'Halloran
  • Patent number: 8815599
    Abstract: The present invention relates to methods and compositions for the inhibition of gene expression. In particular, the present invention provides oligonucleotide-based therapeutics for the inhibition of oncogenes involved in cancers.
    Type: Grant
    Filed: June 1, 2005
    Date of Patent: August 26, 2014
    Assignee: ProNAi Therapeutics, Inc.
    Inventors: Gholamreza Sheikhnehjad, Mina Patel Sooch, Neal Clifford Goodwin, David Olson
  • Patent number: 8808983
    Abstract: The present invention relates to certain novel shRNA molecules and methods of use thereof. According to certain embodiments of the present invention, methods for reducing the expression level of a target gene are provided. Such methods generally comprise providing a cell with one or more precursor nucleic acid sequences that encode two or more RNA molecules. A first RNA molecule comprises a double stranded sequence, which includes a guide strand sequence that is complementary to a portion of an mRNA transcript encoded by the target gene. In addition, a second RNA molecule comprises a second double stranded sequence, which includes a second guide strand sequence that is partially complementary to a portion of the mRNA transcript encoded by the target gene. Preferably, the second guide strand sequence comprises one or more bases that are mismatched with a nucleic acid sequence of the mRNA transcript encoded by the target gene.
    Type: Grant
    Filed: June 29, 2012
    Date of Patent: August 19, 2014
    Assignee: Gradalis, Inc.
    Inventor: Donald Rao
  • Patent number: 8785200
    Abstract: Disclosed are cationic lipid compounds and compositions of lipid aggregates for delivery of macromolecules and other compounds into cells. The compounds can be used alone or in combination with other compounds to prepare liposomes and other lipid aggregates suitable for transfection or delivery of compounds to target cells, either in vitro or in vivo. The compounds are preferably polycationic and preferably form highly stable complexes with various anionic macromolecules, particularly polyanions such as nucleic acids. These compounds have the property, when dispersed in water, of forming lipid aggregates which associate strongly, via their cationic portion, with polyanions. Also disclosed are intermediates for preparing the compound and compositions of the invention and methods of using the compounds to introduce other compounds into cells.
    Type: Grant
    Filed: March 14, 2012
    Date of Patent: July 22, 2014
    Assignee: Life Technologies Corporation
    Inventors: Yongliang Chu, Malek Masoud, Gulilat Gebeyehu
  • Patent number: 8779113
    Abstract: The invention provides a group of nucleic acid fragments, shown in the sequence listing, for prevention of HIV infection or AIDS and the usage thereof. In the invention, a series of RNA fragments, which are highly homogenous to all the published HIV gene sequences, were obtained by homology compare. The double-stranded RNA (dsRNA) derived from these fragments can effectively inhibit the expression of the HIV genes. The RNA transcribed by plasmid, also can suppress the expression of the HIV in the cell. After the adenovirus or associated virus which carry DNA corresponding above RNA infect the cell, the transcription dsRNA can inhibit the expression of the HIV genes.
    Type: Grant
    Filed: January 10, 2011
    Date of Patent: July 15, 2014
    Assignee: Beijing Solobio Genetechnology Company Ltd.
    Inventors: Zhiwen Zhou, Yuxia Feng, Conglin Zuo, Yuejuan Li
  • Publication number: 20140179004
    Abstract: A cell of the present invention contains a nucleic acid construct encoding a WT1 gene product or a fragment of the WT1 gene product. The nucleic acid construct contains (i) a region encoding a desired fragment of the WT1 gene product and (ii) only AUG as a functional start codon. The present invention can provide a cell into which the nucleic acid construct is introduced so that an expression level of a WT1 gene product or a fragment of the WT1 gene product is remarkably enhanced.
    Type: Application
    Filed: July 30, 2012
    Publication date: June 26, 2014
    Applicant: RIKEN
    Inventors: Shin-ichiro Fujii, Kanako Shimizu, Jun Shinga