序号 专利名 申请号 申请日 公开(公告)号 公开(公告)日 发明人
241 METHODS AND PRODUCTS FOR EXPRESSING PROTEINS IN CELLS US15156806 2016-05-17 US20160251649A1 2016-09-01 Matthew Angel; Christopher Rohde
The present invention relates in part to nucleic acids encoding proteins, therapeutics comprising nucleic acids encoding proteins, methods for inducing cells to express proteins using nucleic acids, methods, kits and devices for transfecting, gene editing, and reprogramming cells, and cells, organisms, and therapeutics produced using these methods, kits, and devices. Methods and products for altering the DNA sequence of a cell are described, as are methods and products for inducing cells to express proteins using synthetic RNA molecules. Therapeutics comprising nucleic acids encoding gene-editing proteins are also described.
242 Orthogonal Cas9 Proteins for RNA-Guided Gene Regulation and Editing US14903728 2014-07-08 US20160222416A1 2016-08-04 George M. Church; Kevin M. Esvelt; Prashant G. Mali
Methods of modulating expression of a target nucleic acid in a cell are provided including use of multiple orthogonal Cas9 proteins to simultaneously and independently regulate corresponding genes or simultaneously and independently edit corresponding genes.
243 RNA-GUIDED GENE EDITING AND GENE REGULATION US14895316 2014-06-05 US20160201089A1 2016-07-14 Charles A. Gersbach; Isaac B. Hilton; Pablo Perez-Pinera; Ami M. Kabadi; Pratiksha I. Thakore; David G. Ousterout; Joshua B. Black
Disclosed herein are Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR-associated (Cas) 9-based system related compositions and methods of using said CRISPR/Cas9-based system related compositions for altering gene expression and genome engineering. Also disclosed herein are compositions and methods of using said compositions for altering gene expression and genome engineering in muscle, such as skeletal muscle and cardiac muscle.
244 ENGINEERED TRANSCRIPTION ACTIVATOR-LIKE EFFECTOR (TALE) DOMAINS AND USES THEREOF US14913458 2014-08-22 US20160201040A1 2016-07-14 David R. Liu; John Paul Guilinger; Vikram Pattanayak
Engineered transcriptional activator-like effectors (TALEs) are versatile tools for genome manipulation with applications in research and clinical contexts. One current drawback of TALEs is their tendency to bind and cleave off-target sequence, which hampers their clinical application and renders applications requiring high-fidelity binding unfeasible. This disclosure provides engineered TALE domains and TALEs comprising such engineered domains, e.g., TALE nucleases (TALENs), TALE transcriptional activators, TALE transcriptional repressors, and TALE epigenetic modification enzymes, with improved specificity and methods for generating and using such TALEs.
245 DELIVERY SYSTEM FOR FUNCTIONAL NUCLEASES US14916679 2014-09-05 US20160200779A1 2016-07-14 David R. LIU; John Anthony ZURIS; David B. THOMPSON
Compositions, methods, strategies, kits, and systems for the supercharged protein-mediated delivery of functional effector proteins into cells in vivo, ex vivo, or in vitro are provided. Compositions, methods, strategies, kits, and systems for delivery of functional effector proteins using cationic lipids and cationic polymers are also provided. Functional effector proteins include, without limitation, transcriptional modulators (e.g., repressors or activators), recombinases, nucleases (e.g., RNA-programmable nucleases, such as Cas9 proteins; TALE nuclease, and zinc finger nucleases), deaminases, and other gene modifying/editing enzymes. Functional effector proteins include TALE effector proteins, e.g., TALE transcriptional activators and repressors, as well as TALE nucleases. Compositions, methods, strategies, and systems for the delivery of functional effector proteins into cells is useful for therapeutic and research purposes, including, but not limited to, the targeted manipulation of a gene associated with disease, the modulation of the expression level of a gene associated with disease, and the programming of cell fate.
246 COMPOSITIONS OF AND METHODS FOR IN VITRO VIRAL GENOME ENGINEERING US14970458 2015-12-15 US20160186147A1 2016-06-30 Kyle C. Cady; E. Magda Barbu; Christen G. DiPetrillo
The present disclosure relates to a method of in vitro engineering of nucleic acids. This disclosure further relates to in vitro engineering of viral genomes and to the improvement of viral properties by in vitro genomic engineering of viral genomes. Specifically, the disclosure relates to in vitro viral genomic digestion using RNA-guided Cas9, the assembly of a recombinant genome by the insertion of a DNA or RNA fragment into the digested viral genome and transformation of a host cell with the recombinant genome. This method also related to in vitro engineering for error correction of nucleic acids.
247 METHODS AND FEED SUPPLEMENTS FOR IMPROVING NUTRITION INTAKE OF MEAT-TYPE POULTRIES US14955820 2015-12-01 US20160158326A1 2016-06-09 Marc DE BEER; Chris PROVINCE; Nelson E. WARD
The present invention provides methods of improving growth performance, improving the efficiency of feed utilization, and increasing feed digestibility of poultry receiving animal feed. Composition of the multi-component enzyme mixture designed to achieve the same are also provided.
248 DELIVERY, ENGINEERING AND OPTIMIZATION OF SYSTEMS, METHODS AND COMPOSITIONS FOR TARGETING AND MODELING DISEASES AND DISORDERS OF POST MITOTIC CELLS US14971169 2015-12-16 US20160153004A1 2016-06-02 Feng ZHANG; Matthias HEIDENREICH; Lukasz SWIECH
The invention provides for delivery, engineering and optimization of systems, methods, and compositions for manipulation of sequences and/or activities of target sequences. Provided are delivery systems and tissues or organ which include post mitotic cells which are targeted as sites for delivery. Also provided are vectors and vector systems some of which encode one or more components of a CRISPR complex, as well as methods for the design and use of such vectors. Also provided are methods of directing CRISPR complex formation in eukaryotic cells to ensure enhanced specificity for target recognition and avoidance of toxicity and to edit or modify a target site in a genomic locus of interest to alter or improve the status of a disease or a condition.
249 Methods for Expressing a Polynucleotide of Interest in the Retina of a Subject US14900350 2014-06-27 US20160144057A1 2016-05-26 Deniz Dalkara; Alvaro Rendon Fuentes; Jose Alain Sahel; Ophelie VACCA
The present invention relates to methods for expressing a polynucleotide of interest in the retina of a subject. In particular, the present invention relates to a method for expressing a polynucleotide of interest in the retina of a subject comprising the step consisting of injecting into the vitreous an amount of a vector containing the polynucleotide of interest in combination with an amount of an inhibitor of Dp71 expression.
250 Substituted-6,8-dioxabicyclo[3.2.1]octane-2,3-diol compounds as targeting agents of ASGPR US14714484 2015-05-18 US09340553B2 2016-05-17 Spiros Liras; Vincent Mascitti; Benjamin Thuma
Compounds of Formula (A) are described herein and the uses thereof for the treatment of diseases, conditions and/or disorders mediated by pharmaceutical compositions and the uses thereof as asialoglycoprotein receptor (ASGPR) targeting agents.
251 COMPOUNDS AND METHODS FOR TRANS-MEMBRANE DELIVERY OF MOLECULES US14830799 2015-08-20 US20160106855A1 2016-04-21 Ilan ZIV
A novel delivery system for drugs, and especially macromolecules such as proteins or oligonucleotides through biological membranes is provided, and specifically delivery of siRNA The delivery system comprises conjugation of the macromolecule drug to a moiety that enables effective passage through the membranes. Respectively, novel compounds and pharmaceutical compositions are provided, utilizing said delivery system. In one aspect of the invention, the compounds may be utilized in medical practice, for example, in delivery of siRNA or antisense oligonucleotides across biological membranes for the treatment of medical disorders.
252 METHODS FOR IDENTIFYING A TARGET SITE OF A CAS9 NUCLEASE US14874123 2015-10-02 US20160090622A1 2016-03-31 David R. Liu; Vikram Pattanayak
Some aspects of this disclosure provide strategies, methods, and reagents for determining nuclease target site preferences and specificity of site-specific endonucleases. Some methods provided herein utilize a novel “one-cut” strategy for screening a library of concatemers comprising repeat units of candidate nuclease target sites and constant insert regions to identify library members that can been cut by a nuclease of interest via sequencing of an intact target site adjacent and identical to a cut target site.
253 COMPOSITIONS AND METHODS TO TREAT LATENT VIRAL INFECTIONS US14725888 2015-05-29 US20160060655A1 2016-03-03 STEPHEN R. QUAKE; JIANBIN WANG
Viral infection is a persistent cause of human disease. Guided nuclease systems target the genomes of viral infections, rendering the viruses incapacitated.
254 Methods of generating and screening for lytic chimeric polypeptides US13704759 2011-06-17 US09249447B2 2016-02-02 Anna Scherzinger; Sonja Molinaro; Bernd Buchberger
The present invention relates to novel methods of generating and screening for chimeric polypeptides, which can be used in the treatment and prophylaxis of pathogenic bacterial contamination, colonization and infection. The novel methods are based on random recombination of protein domains, and the chimeric polypeptides obtainable by the methods according to the invention are characterized in that they comprise at least one enzymatic active domain (EAD) and at least one cell binding domain (CBD). The present invention also relates to a library of chimeric polypeptides obtainable by the methods of the present invention.
255 Increasing Specificity for RNA-Guided Genome Editing US14776620 2014-03-14 US20160024524A1 2016-01-28 J. Keith Joung; James Angstman; Jeffry D. Sander; Morgan Maeder; Shengdar Tsai
Methods for increasing specificity of RNA-guided genome editing, e.g., editing using CRISPR/Cas9 systems.
256 Modified zinc finger binding proteins US10055711 2002-01-22 US09234187B2 2016-01-12 Edward Rebar; Andrew Jamieson
Disclosed herein are compositions and method comprising non-canonical (e.g., non-C2H2) zinc finger proteins.
257 METHODS AND COMPOSITIONS FOR NUCLEASE DESIGN US14731821 2015-06-05 US20150353917A1 2015-12-10 Jeffrey C. Miller
Methods and compositions for genetic alteration of cells are provided.
258 SUBSTITUTED-6,8-DIOXABICYCLO[3.2.1]OCTANE-2,3-DIOL COMPOUNDS AS TARGETING AGENTS OF ASGPR US14714484 2015-05-18 US20150329555A1 2015-11-19 Spiros Liras; Vincent Mascitti; Benjamin Thuma
Compounds of Formula (A) are described herein and the uses thereof for the treatment of diseases, conditions and/or disorders mediated by pharmaceutical compositions and the uses thereof as asialoglycoprotein receptor (ASGPR) targeting agents.
259 PECTIN EXTRACTION FROM COFFEE PULP US14647569 2013-11-27 US20150307634A1 2015-10-29 Andres Felipe Belalcazar Otalora
The invention provides a coffee pulp treatment process comprising (a) Providing coffee pulp, obtainable from a production process for producing green coffee beans from coffee cherries; (b) extracting from the coffee pulp a pectin comprising extract, wherein extraction is performed under acid conditions or alkaline conditions, to provide the pectin comprising extract; (c) enzymatic treatment of the pectin comprising extract, wherein the enzymatic treatment comprises a treatment with one or more enzymes selected from the group consisting of an esterase and a reductase, to provide a enzymatically treated pectin material; and (d) extraction of polyphenol functionalized coffee pectin extract from the enzymatically treated pectin material.
260 STRAIN EXPRESSING FRSA AND METHOD FOR PRODUCING ETHANOL USING SAME US14439317 2013-10-18 US20150299686A1 2015-10-22 Kyung-Jo LEE; Yu Ra KIM; Jung Kee LEE; Sun-Shin CHA; Kyu-Ho LEE
The present application relates to a strain expressing the FrsA protein, and a method for producing ethanol using the same. The FrsA of the present application has a high PDC enzyme activity for a pyruvate, which is a substrate, and thus can be used in a process for producing ethanol. In addition, an FrsA mutant having improved stability in a host cell can be more effective in producing ethanol due to the increase in stability when the FrsA mutant is overexpressed together with IIAGlc, compared with when using conventional Zymomonas mobilis-derived PDC
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