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US20120213738 Cardiac Repair By Reprogramming of Cardiac Fibroblasts Into Cardiomyocytes  
The present invention involves the use of transcription factors including Tbx5, Mef2C, Hand2, myocardin and Gata4 to reprogram cardiac fibroblasts into cardiomyocytes, both in vitro and in vivo....
US20110165683 CHIMERIC GAMMARETROVIRUS  
The invention is directed to a chimeric gammaretrovirus comprising an gammaretroviral virion which contains a lentiviral Vpx protein and methods of use thereof.
US20120302464 METHODS FOR PRODUCING RECOMBINANT PROTEINS  
Methods for producing recombinant cell populations are disclosed. The disclosed methods may be used to produce therapeutic polyclonal proteins.
US20140170123 Hepatocyte Based Insulin Gene Therapy For Diabetes  
A method and vectors for controlling blood glucose levels in a mammal are disclosed. In one embodiment, the method comprises the steps of: treating the hepatocyte cells of a patient with a first,...
US20120315255 Hepatocyte Based Insulin Gene Therapy For Diabetes  
A method and vectors for controlling blood glucose levels in a mammal are disclosed. In one embodiment, the method comprises the steps of: treating the hepatocyte cells of a patient with a first,...
US20140205570 RECOMBINANT ADENO-ASSOCIATED VIRUS-MEDIATED EXPRESSION OF FRACTALKINE FOR TREATMENT OF NEUROINFLAMMATORY AND NEURODEGENERATIVE DISEASES  
The subject invention pertains to the use of fractalkine (FKN, CX3CL1) and its receptor CX3CR1 for treatment of neuroinflammation and/or neurodegeneration. In one embodiment, the present invention...
US20150159143 GENERATION OF HUMAN iPS CELLS BY A SYNTHETIC SELF-REPLICATIVE RNA  
The disclosure provides methods and compositions useful for obtaining induced stem cells, methods of making and use thereof.
US20150166618 METHODS AND COMPOSITIONS FOR TREATING HEMOPHILIA  
Disclosed herein are methods and compositions for insertion of transgene sequences encoding proteins involved in clotting into the genome of a cell for treating conditions including hemophilias.
US20100325746 Methods and sequences to suppress primate huntington gene expression in vivo  
Disclosed herein are sequences, molecules and methods used to suppress the expression of HD genes encoding for huntingtin protein in primates including Macaca mulatta and Homo sapiens. These...
US20130052267 Methods of Inducing Tissue Regeneration  
Methods are provided for producing cells within a lineage (lineage restricted cells) from post-mitotic differentiated cells of the same lineage ex vivo and in vivo, and for treating a subject in...
US20140056854 VIRAL VECTORS WITH MODIFIED TRANSDUCTION PROFILES AND METHODS OF MAKING AND USING THE SAME  
The present invention provides AAV capsid proteins, virus capsids comprising said capsid proteins and virus vectors comprising said capsid proteins, wherein the AAV capsid proteins have one or...
US20120202291 SIMPLIFIED BASIC MEDIA FOR HUMAN PLURIPOTENT CELL CULTURE  
Fully defined media that support pluripotent cell viability, proliferation, cloning, and derivation, as well as methods and compositions including these media are described. Methods for deriving...
US20120294838 Regulation of endogenous gene expression in cells using zinc finger proteins  
The present invention provides methods for modulating expression of endogenous cellular genes using recombinant zinc finger proteins.
US20140271551 Synthetic Adeno-Associated Virus Inverted Terminal Repeats  
This invention relates to synthetic adeno-associated virus (AAV) inverted terminal repeats (ITRs) that exhibit altered activities compared to a naturally occurring AAV ITR and methods of using the...
US20120046340 Down Regulation of the Gene Expression by Means of Nucleic Acid-Loaded Virus-Like Particles  
The present invention relates to compositions of virus-like particles for the introduction of RNA-interference (RNAi-) inducing molecules into eukaryotic cells and methods for the cell...
US20120151614 VECTOR UTILIZING BORNA DISEASE VIRUS AND USE THEREOF  
Disclosed is a viral vector comprising (a) a cDNA of a recombinant viral RNA having at least the N gene, the X gene, the P gene, and the L gene of a Borna disease virus genome in the same order as...
US20130004581 CONTROLLING UPTAKE BY CELLS  
Methods and devices for causing uptake of materials by cells, temporary using local chemical environment modification. The modification may be caused chemically by reducing pH. The uptake method...
US20140335063 DELIVERY METHODS AND COMPOSITIONS FOR NUCLEASE-MEDIATED GENOME ENGINEERING  
Disclosed herein are methods and compositions for delivery of engineered nucleases and donor molecules into the genome of a cell.
US20110257080 Hepatitis B Virus Compositions and Methods of Use  
A polypeptide comprising a preS1 region of hepatitis B virus (HBV), or a fragment thereof, and/or the preS2 region of HBV or a fragment thereof, and methods of use to inhibit virus infection are...
US20120041050 ANTISENSE OLIGONUCLEOTIDES FOR INDUCING EXON SKIPPING AND METHODS OF USE THEREOF  
An antisense molecule capable of binding to a selected target site to induce exon skipping in the dystrophin gene, as set forth in SEQ ID NO: 1 to 202.
US20120022144 Antisense Oligonucleotides for Inducing Exon Skipping and Methods of Use Thereof  
An antisense molecule capable of binding to a selected target site to induce exon skipping in the dystrophin gene, as set forth in SEQ ID NO: 1 to 202.
US20130259833 AAV-Mediated Subcellular Targeting of Heterologous Rhodopsins in Retinal Ganglion Cells  
Microbial type rhodopsins, such as the light-gated cation-selective membrane channel, channelrhodopsin-2 (Chop2/ChR2) or the ion pump halorhodopsin (HaloR) are expressed in retinal ganglion cells...
US20140193912 METHOD FOR INCREASING THE EFFICIENCY OF INDUCING PLURIPOTENT STEM CELLS  
The present invention relates to a method for increasing the efficiency of inducing pluripotent stem cells by utilizing genes Jhdm1a that modify histone. By utilizing Jhdm1a, and a stem cell...
US20150038561 MITOCHONDRIAL TARGETED RNA EXPRESSION SYSTEM AND USE THEREOF  
Described herein is a mitochondrial-targeted RNA expression system (mtTRES) for delivery of RNA molecules to mitochondria. mtTRES vectors generate RNAs in vivo that are un-capped,...
US20150037300 NUCLEIC ACID FOR TREATMENT OR PREVENTION OF IMMUNODEFICIENCY VIRUS INFECTION  
A nucleic acid comprising a transcription regulation sequence whose transcription is induced by a trans-acting factor of a human immunodeficiency virus and a gene encoding a polypeptide having an...
US20130065814 INDUCTIVE PRODUCTION OF PLURIPOTENT STEM CELLS USING SYNTHETIC TRANSCRIPTION FACTORS  
The present invention relates to use of synthetic factors in reprogramming somatic cells to become induced pluripotent stem cells and other cell lineages. Specifically, the present application...
US20110262447 HUMAN SYNTHETIC SINGLE-CHAIN ANTIBODIES DIRECTED AGAINST THE COMMON EPITOPE OF MUTANT P53 AND THEIR USES  
Isolated polypeptides, isolated polynucleotides or expression vectors encoding same, viral display vehicles which can be specifically bind an exposed epitope shared by mutant, but not wild type,...
US20110275105 Methods of inducing pluripotency  
Methods are provided for inducing non-pluripotent cells to become pluripotent. Methods also include identifying and isolating induced pluripotent (iPS) cells and uses thereof. Compositions and...
US20130137104 METHODS AND COMPOSITIONS FOR MODIFICATION OF THE HPRT LOCUS  
Nucleases and methods of using these nucleases for modification of an HPRT locus and for increasing the frequency of gene modification at a targeted locus and clones and for generating animals.
US20130122591 METHODS AND COMPOSITIONS FOR MODIFICATION OF THE HPRT LOCUS  
Nucleases and methods of using these nucleases for modification of an HPRT locus and for increasing the frequency of gene modification at a targeted locus and clones and for generating animals.
US20130198876 INDUCED MALIGNANT STEM CELLS OR PRE-INDUCTION CANCER STEM CELLS CAPABLE OF SELFREPLICATION OUTSIDE OF AN ORGANISM, PRODUCTION METHOD FOR SAME, AND PRACTICAL APPLICATION FOR SAME  
The present invention provides an induced cancer cell capable of self-replication in vitro which is useful in cancer therapy research and the research for cancer-related drug discovery, processes...
US20110268705 HDAC4, HDAC5, HDAC6, HDAC7, AND HIF1 ALPHA MODULATION OF RETINAL CELL SURVIVAL  
Methods for inhibiting retinal cell death by altering expression of one or more of HDAC4, HDAC5, HDAC6, HDAC7, and HIF1α in a retinal cell are provided.
US20150191744 CAS9 EFFECTOR-MEDIATED REGULATION OF TRANSCRIPTION, DIFFERENTIATION AND GENE EDITING/LABELING  
The present disclosure relates to methods of and systems for modifying the transcriptional regulation of stem or progenitor cells to promote their differentiation or reprogramming of somatic...
US20120258540 METHODS FOR MODIFYING VIRUS SURFACES  
Nucleic acid delivery vehicles and methods of their use are provided. One embodiment provides a virus having a lipid-polymer conjugate intercalated into the virus's membrane. The lipid-polymer...
US20110052585 COMPOSITIONS AND METHODS FOR INHIBITING INTERLEUKIN PATHWAYS  
Fusion proteins including an IL-17 receptor with a multimerization domain, or an IL-23 receptor and a multimerization domain, and recombinant viral vectors encoding such fusions, are described....
US20140335620 ENGINEERING AND OPTIMIZATION OF IMPROVED SYSTEMS, METHODS AND ENZYME COMPOSITIONS FOR SEQUENCE MANIPULATION  
The invention provides for engineering and optimization of systems, methods, and compositions for manipulation of sequences and/or activities of target sequences. Provided are compositions and...
US20110189730 VIRAL NUCLEOCAPSID PROTEIN AS A MULTIFUNCTIONAL TRANSLATION INITIATION FACTOR AND INCREASED PROTEIN AND POLYPEPTIDE PRODUCTION USING SAME  
The present invention is directed to a system to significantly increase the expression of genes of interest, and in particular proteins and polypeptide products. Expression of hantavirus...
US20070110716 Relationship of a specific metabolite to insulin resistance  
Provided are methods and reagents for reducing ketone levels (for example, β-hydroxybutyrate) in skeletal muscle. Also provided are methods and reagents for treating insulin resistant states, such...
US20110182933 MODIFIED VACCINIA ANKARA VIRUS VARIANT  
The present invention provides an attenuated virus, which is derived from Modified Vaccinia Ankara virus, wherein the MVA-BN virus, or a derivative thereof, induces at least substantially the same...
US20100323383 Methods for in vitro differentiation of Th-17+cells  
The present invention is directed to an in vitro method for promoting differentiation and proliferation of human T helper lymphocytes that express IL17 (Th-IL17+ cells). The instant method may be...
US20150050242 NIPAH VIRUS ENVELOPE PSEUDOTYPED LENTIVIRUSES AND METHODS OF THEIR USE  
The present invention relates to lentiviral particles which have been pseudotyped with Nipah virus (NiV) fusion (F) and attachment (G) glycoproteins (NiVpp-F/G). Additionally, the present...
US20140315984 Methods and Compositions for Treatment of Diabetes and Related Disorders  
The present invention provides a method of stimulating growth of a pancreatic islet beta cell and/or enhancing glucose stimulated insulin secretion of a pancreatic islet beta cell, comprising...
US20110183908 YOPM AS DELIVERY VEHICLE FOR CARGO MOLECULES AND AS BIOLOGICAL THERAPEUTIC FOR IMMUNOMODULATION OF INFLAMMATORY REACTIONS  
The present invention relates to the use of Yersinia outer protein M (YopM), a YopM fragment, or a YopM variant, which is capable of autopenetrating the cell membrane and of integrating into the...
US20140322813 METHODS AND COMPOSITIONS FOR ENHANCING THE EFFICACY AND SPECIFICITY OF RNA SILENCING  
The present invention provides methods of enhancing the efficacy and specificity of RNA silencing. The invention also provides compositions for mediating RNA silencing. In particular, the...
US20150203869 Adenoviral Vectors for Transduction of Vascular Tissue  
The invention relates to the field of gene transfer, and in particular to the use of adenoviral vectors of serotype Ad49 for gene delivery to vascular tissue.
US20110165187 TUMOR CELLS FROM IMMUNE PRIVILEGED SITES AS BASE CELLS FOR CELL-BASED CANCER VACCINES  
The present invention relates to tumor cell-based vaccines and methods of using same, wherein the vaccines are based on naturally immune privileged tumor cells that have been genetically modified...
US20120251498 Gene Delivery to Organs  
Application of a virus with poloxamer alone onto atria results in diffuse epicardial gene transfer with negligible penetration into the myocardium. Progressive increases in protease concentration,...
US20130267029 MANIPULATION OF STEM CELL FUNCTION BY P53 ISOFORMS  
This invention provides methods and compositions for increasing the efficiency of obtaining pluripotent stem cells, the method comprising expressing a 133p53 in cells that are being re-programmed...
US20110243986 POLYNUCLEOTIDES ALLOWING THE EXPRESSION AND SECRETION OF RECOMBINANT PSEUDO-VIRUS CONTAINING FOREIGN EPITOPES, THEIR PRODUCTION, AND USE  
This invention provides a new approach to the design of a virus with a defective replication cycle, which can be rescued by wild type virus co-infection, and which expresses foreign antigenic...
US20150140663 IN VITRO RECONSTITUTED PLANT VIRUS CAPSIDS FOR DELIVERING RNA GENES TO MAMMALIAN CELLS  
The invention provides compositions of matter comprising a cowpea chlorotic mottle virus capsid protein (CCMV CP) and a ribonucleic acid, as well as methods for using such compositions. In such...