Gene Therapy Patents (Class 977/916)
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Patent number: 9040626Abstract: The invention discloses novel morphology shifting micelles and amphiphilic coated metal nanofibers. Methods of using and making the same are also disclosed.Type: GrantFiled: February 1, 2012Date of Patent: May 26, 2015Assignee: The Regents of the University of CaliforniaInventors: Miao-Ping Chien, Nathan C. Gianneschi
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Patent number: 9029604Abstract: 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: GrantFiled: September 28, 2011Date of Patent: May 12, 2015Assignee: Sirna Therapeutics, Inc.Inventors: John A. Bawiec, III, Zhengwu J. Deng
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Publication number: 20140335356Abstract: Problem. Provided are a method for synthesizing spherical porous titanium oxide nanoparticles, which is easy to operate, does not take a long time for synthesis, and can easily adjust the particle diameter and the pore diameter of the spherical porous titanium oxide nanoparticles in accordance with the application thereof; spherical porous titanium oxide nanoparticles produced by the synthesizing method; and a gene gun carrier consisting of the spherical porous titanium oxide nanoparticles. Solution. A method for synthesizing spherical porous titanium oxide nanoparticles, includes: a step of reacting titanium isopropoxide and carboxylic acid in supercritical fluid, wherein the supercritical fluid is supercritical methanol, and the carboxylic acid is formic acid, acetic acid, benzoic acid, o-phthalic acid, fumaric acid, or maleic acid.Type: ApplicationFiled: January 27, 2012Publication date: November 13, 2014Applicant: Kochi University of TechnologyInventors: Kazuya Kobiro, Pengyu Wang, Takeshi Ohama
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Patent number: 8785177Abstract: Methods for creating a transient nanoscale opening in a cell membrane and methods for transporting a desired species through the nanoscale opening are provided. A nano-sized needle-like tip can be used to mechanically slice the cell membrane to create a transient, localized nanoscale slit. The nanoscale slit may be used for transferring exogenous molecules into a living cell.Type: GrantFiled: November 5, 2012Date of Patent: July 22, 2014Assignee: The Board of Trustees of the University of Illinois, a body Corporate and Politic of the State of IllinoisInventors: Min-Feng Yu, Kyungsuk Yum, Ning Wang
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Patent number: 8466122Abstract: The present invention provides compositions and methods for the delivery of therapeutic agents to cells. In particular, these include novel cationic lipids and nucleic acid-lipid particles that provide efficient encapsulation of nucleic acids and efficient delivery of the encapsulated nucleic acid to cells in vivo. The compositions of the present invention are highly potent, thereby allowing effective knock-down of a specific target protein at relatively low doses. In addition, the compositions and methods of the present invention are less toxic and provide a greater therapeutic index compared to compositions and methods previously known in the art.Type: GrantFiled: September 16, 2011Date of Patent: June 18, 2013Assignee: Protiva Biotherapeutics, Inc.Inventors: James Heyes, Mark Wood, Alan Martin
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Patent number: 8445025Abstract: Disclosed are the nanoparticle and the method for the same, and the preparing method includes steps of mixing polyethylenimine (PEI) with the poly(acrylic acid)-bound iron oxide (PAAIO) to form a PEI-PAAIO polyelectrolyte complex (PEC) and mixing the PEI-PAAIO PEC with genetic material such as plasmid DNA to form the PEI-PAAIO/pDNA magnetic nanoparticle. The PEI-PAAIO/pDNA magnetoplex is highly water dispersible and suitable for long term storage, shows superparamagnetism, low cytotoxicity, high stability and nice transfection efficiency, and thus the PEI-PAAIO PEC can replace PEI as a non-viral gene vector.Type: GrantFiled: March 29, 2011Date of Patent: May 21, 2013Assignee: Kaohsiung Medical UniversityInventors: Li-Fang Wang, Shuo-Li Sun, Yu-Lun Lo
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Patent number: 8318207Abstract: In accordance with certain embodiments of the present disclosure, a method for intracellular delivery of small molecules is provided. The method includes encapsulation of small molecules in a thermally responsive nanocapsule by decreasing the temperature of the nanocapsule to increase the permeability of the nanocapsule and allowing the small molecules to be suck into or diffuse into the nanocapsule. The nanocapsule is delivered into a cell by increasing the temperature of the nanocapsule. The small molecules are released from the nanocapsule into the cell in a controllable manner by cooling and heating treatments.Type: GrantFiled: February 12, 2010Date of Patent: November 27, 2012Assignee: University of South CarolinaInventor: Xiaoming He
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Patent number: 8283333Abstract: The present invention provides novel, serum-stable lipid particles comprising one or more active agents or therapeutic agents, methods of making the lipid particles, and methods of delivering and/or administering the lipid particles. More particularly, the present invention provides serum-stable nucleic acid-lipid particles (SNALP) comprising a nucleic acid (e.g., one or more interfering RNA molecules), methods of making the SNALP, and methods of delivering and/or administering the SNALP (e.g., for the treatment of cancer). In particular embodiments, the present invention provides tumor-directed lipid particles that preferentially target solid tumors. The tumor-directed formulations of the present invention are capable of preferentially delivering a payload such as a nucleic acid to cells of solid tumors compared to non-cancerous cells.Type: GrantFiled: June 30, 2010Date of Patent: October 9, 2012Assignee: Protiva Biotherapeutics, Inc.Inventors: Ed Yaworski, Stephen Reid, James Heyes, Adam Judge, Ian MacLachlan
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Publication number: 20120214867Abstract: A T4 nanoparticle is a non-infectious, tail-less variant of a T4 bacteriophage. In one embodiment, eukaryotic cells are labeled with dyed T4 nanoparticles, wherein each dyed T4 nanoparticle comprises at least 350 dye molecules covalently bound thereto. In another embodiment, T4 nanoparticles are used to deliver exogenous DNA to eukaryotic cells for protein expression therein. It is contemplated that T4 nanoparticles may be used to deliver other exogenous material to eukaryotic cells.Type: ApplicationFiled: February 14, 2012Publication date: August 23, 2012Applicant: The Government of the United States of America, as represented by the Secretary of the NavyInventors: Jinny Lin Liu, Kelly L. Robertson, Carissa M. Soto
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Publication number: 20120076735Abstract: Disclosed are drug delivery systems and methods for extravascular administration of drug, vaccine, and/or diagnostic agents, for use in research and medical applications.Type: ApplicationFiled: March 24, 2011Publication date: March 29, 2012Applicant: GENESEGUES, INC.Inventor: Gretchen Unger
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Publication number: 20120003278Abstract: The present invention includes single-walled carbon nanotube compositions for the delivery of bioactive agents and methods of making such single-walled carbon nanotube compositions.Type: ApplicationFiled: July 1, 2011Publication date: January 5, 2012Applicant: ENSYSCE BIOSCIENCES, INC.Inventors: D. Lynn Kirkpatrick, Michelle K. Weiss
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Publication number: 20110305734Abstract: The present invention provides nanoparticle compositions including one or more nucleic acids. The present invention achieves delivery (particularly transdermal delivery) of such nucleic acids without the need for nucleic acid modification, or for use of chemical or mechanical abrasion or disruption of skin.Type: ApplicationFiled: May 30, 2008Publication date: December 15, 2011Applicant: ANTERIOS, INC.Inventors: Jonathan Edelson, Timothy Kotyla, Boke Zhang
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Publication number: 20110268810Abstract: Polymeric microparticles are used to deliver recombinagenic or mutagenic nucleic acid molecules such as donor nucleic acid alone, or in combination with triplex-forming molecules, to induce a site-specific mutation in the target DNA. Target cells endocytose the particles, releasing the nucleic acid molecules inside of the cell, where they induce mutagenesis or recombination at a target site. The examples demonstrate that triplex forming oligonucleotides, preferably PNAs, preferably in combination with a donor nucleotide molecule, can be encapsulated into polymeric microparticles, which are delivered into cells. Results demonstrate significantly greatly levels of uptake and expression, and less cytotoxicity, as compared to direct transfer of the nucleic acid molecules into the cell by nucleofection.Type: ApplicationFiled: November 2, 2010Publication date: November 3, 2011Inventors: William Mark Saltzman, Peter M. Glazer, Joanna Chin, Nicole McNeer
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Patent number: 7981446Abstract: The present invention relates to methods of delivering nucleic acids into cells using a nucleic acid binding molecule containing a multimeric or spacer-incorporated protein transduction domain (PTD). The invention also relates to novel compositions that contain a nucleic acid complexed or conjugated with a nucleic acid binding molecule. The nucleic acid binding molecule may contain a multimeric or spacer-incorporated PTD, and may further contain a nucleic acid binding region. The nucleic acid complexes or conjugations of the present invention may be employed to inhibit expression of a target gene, and/or determine the function of a target gene.Type: GrantFiled: November 24, 2008Date of Patent: July 19, 2011Assignee: ForHumanTech. Co., Ltd.Inventors: Sang-Kyou Lee, Seung-Kyou Lee, Ki-Doo Choi
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Publication number: 20110171276Abstract: The present invention provides chitosan-based nanoparticles that can protect nucleic acids and deliver the same into gut mucosal cells. Compositions and methods for the expression of therapeutic nucleic acids in cells of the gut mucosa are provided. Compositions and methods for delivering therapeutic proteins systemically from cells of the gut mucosa are also provided.Type: ApplicationFiled: March 24, 2011Publication date: July 14, 2011Applicant: enGene, Inc.Inventors: Anthony T. Cheung, Eric C. Hsu
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Publication number: 20110097389Abstract: The present invention relates to the identification of p53 biomarker profiles that predict response in patients with hyperproliferative disease such as cancer to a therapy, and their use in methods of treating such patients with an anti-hyperproliferative disease gene therapy.Type: ApplicationFiled: January 26, 2009Publication date: April 28, 2011Applicant: P53Inventors: Robert E. Sobol, Kerstin Menander
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Patent number: 7902441Abstract: Tumor suppressor genes play a major role in the pathogenesis of human lung cancer and other cancers. Cytogenetic and allelotyping studies of fresh tumor and tumor-derived cell lines showed that cytogenetic changes and allele loss on the short arm of chromosome 3 (3p) are most frequently involved in about 90% of small cell lung cancers and greater than 50% of non-small cell lung cancers. A group of recessive oncogenes, Fus1, 101F6, Gene 21 (NPRL2), Gene 26 (CACNA2D2), Luca 1 (HYAL1), Luca 2 (HYAL2), PL6, 123F2 (RaSSFI), SEM A3 and Beta* (BLU), as defined by homozygous deletions in lung cancers, have been located and isolated at 3p21.3.Type: GrantFiled: May 27, 2003Date of Patent: March 8, 2011Assignees: Board of Regents, The University of Texas, The United States of America as represented by the Department of Health and Human ServicesInventors: Lin Ji, John Dorrance Minna, Jack Roth, Michael Lerman
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Publication number: 20110038937Abstract: Disclosed herein are formulations of siRNA suitable for delivery by ocular iontophoresis, devices for iontophoretic delivery of siRNA and methods of use thereof.Type: ApplicationFiled: December 5, 2008Publication date: February 17, 2011Inventors: William Schubert, Peyman Moslemy, Mike Patane, Phil Isom
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Publication number: 20100310473Abstract: It is disclosed here that nucleic acid-based agents can be delivered to the brain of a human or non-human animal having a leakage in the blood brain barrier by administering the agents through the eye. Brain tissues and cells can be imaged in vivo (e.g., by magnetic resonance imaging) by linking a contrast agent to a targeting nucleic acid that can hybridize to a target nucleic acid located at the brain site to be imaged and administering the contrast agentltargeting nucleic acid conjugate through the eye. Similarly, a nucleic acid based drug (e.g., as an antisense nucleic acid or a therapeutic agent linked to a targeting nucleic acid that can hybridize to a target nucleic located at a disease site in the brain) can be administered through the eye to treat a brain disease.Type: ApplicationFiled: July 30, 2008Publication date: December 9, 2010Applicant: THE GENERAL HOSPITAL CORPORATIONInventors: Philip K. Liu, Christina H. Liu
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Patent number: 7138098Abstract: A method of manufacturing a nanocrystallite from a M-containing salt forms a nanocrystallite. The nanocrystallite can be a member of a population of nanocrystallites having a narrow size distribution and can include one or more semiconductor materials. Semiconducting nanocrystallites can photoluminesce and can have high emission quantum efficiencies.Type: GrantFiled: October 8, 2004Date of Patent: November 21, 2006Assignee: Massachusetts Institute of TechnologyInventors: Moungi Bawendi, Nathan E. Stott