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US20160273003 |
USE OF MEGANUCELASES FOR INDUCING HOMOLOGOUS RECOMBINATION EX VIVO AND IN TOTO IN VERTEBRATE SOMATIC TISSUES AND APPLICATION THEREOF
A single chain homing endonuclease, comprising a first variant of I-CreI having the amino acid sequence of accession number pdb 1g9y and a second variant of I-CreI variant having the amino acid... |
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US20170145393 |
NPP1 FUSION PROTEINS
The present invention provides a novel fusion polypeptide containing a catalytic domain of NPP1 fused to a targeting moiety, nucleic acids encoding the fusion polypeptide, a vector containing the... |
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US20170042977 |
METHODS AND COMPOSITIONS FOR CNS DELIVERY OF HEPARAN N-SULFATASE
The present invention provides, among other things, compositions and methods for CNS delivery of lysosomal enzymes for effective treatment of lysosomal storage diseases. In some embodiments, the... |
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US20160367703 |
DIAGNOSIS AND TREATMENT OF MULTIPLE SULFATASE DEFICIENCY AND OTHER SULFATASE DEFICIENCIES
This invention relates to methods and compositions for the diagnosis and treatment of Multiple Sulfatase Deficiency (MSD) as well as other sulfatase deficiencies. More specifically, the invention... |
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US20160319256 |
SIRP-ALPHA VARIANT CONSTRUCTS AND USES THEREOF
The invention relates to compositions and methods of constructs comprising a SIRP-α polypeptide, including SIRP-α variants. The constructs may be engineered in a variety of ways to respond to... |
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US20160052968 |
BONE DELIVERY CONJUGATES AND METHOD OF USING SAME TO TARGET PROTEINS TO BONE
A bone delivery conjugate having a structure selected from the group consisting of: A) X-Dn-Y-protein-Z; and B) Z-protein-Y-Dn-X, wherein X is absent or is an amino acid sequence of at least one... |
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US20160046913 |
Modified Phosphatases
The invention relates to phosphatases and more in specific to (genetically) modified phosphatases, pharmaceutical compositions comprising (genetically) modified phosphatases and the use of... |
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US20150044192 |
METHODS FOR IDENTIFYING A TARGET SITE OF A CAS9 NUCLEASE
Some aspects of this disclosure provide strategies, methods, and reagents for selecting a site-specific endonuclease based on determining its target site preferences and specificity. Methods and... |
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US20150004151 |
USE OF HSP70 AS A REGULATOR OF ENZYMATIC ACTIVITY
The present invention concerns a method for modulating the enzymatic activity of an enzyme, wherein said enzyme interacts with BMP, said method comprising the step of administering or inducing... |
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US20140294773 |
MODIFIED CASCADE RIBONUCLEOPROTEINS AND USES THEREOF
A clustered regularly interspaced short palindromic repeat (CRISPR)-associated complex for adaptive antiviral defence (Cascade); the Cascade protein complex comprising at least CRISPR-associated... |
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US20140227244 |
COUPLING ENDONUCLEASES WITH END-PROCESSING ENZYMES DRIVES HIGH EFFICIENCY GENE DISRUPTION
The present disclosure relates to the co-expression of an endonuclease with an end-processing enzyme for the purpose of enhanced processing of the polynucleotide ends generated by endonuclease... |
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US20140193388 |
Modified Phosphatases
The invention relates to phosphatases and more in specific to (genetically) modified phosphatases, pharmaceutical compositions comprising (genetically) modified phosphatases and the use of... |
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US20140004097 |
METHOD OF PRODUCING RECOMBINANT IDURONATE-2-SULFATASE
The present invention provides, among other things, methods and compositions for large-scale production of recombinant I2S protein using suspension culture of mammalian cells in serum-free medium.... |
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US20130273116 |
ANTI-FOULING COMPOSITION COMPRISING AN AEROGEL
The present invention also relates to an anti-fouling composition comprising one or more aerogels. In one embodiment the aerogel encapsulate one or more bioactive agents. The one or more... |
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US20130177531 |
Inhibitors of Serine Proteases
This invention relates to compounds of formula I: or a pharmaceutically acceptable salt or mixtures thereof wherein C* represents a diastereomeric carbon comprising a mixture of R and S isomers... |
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US20130028881 |
DIAGNOSIS AND TREATMENT OF MULTIPLE SULFATASE DEFICIENCY AND OTHER SULFATASE DEFICIENCIES
This invention relates to methods and compositions for the diagnosis and treatment of Multiple Sulfatase Deficiency (MSD) as well as other sulfatase deficiencies. More specifically, the invention... |
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US20120036599 |
NOVEL FUNGAL ENZYMES
This invention relates to novel enzymes and novel methods for producing the same. More specifically this invention relates to a variety of fungal enzymes. Nucleic acid molecules encoding such... |
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US20120030838 |
NOVEL FUNGAL ENZYMES
This invention relates to novel enzymes and novel methods for producing the same. More specifically this invention relates to a variety of fungal enzymes. Nucleic acid molecules encoding such... |
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US20100239558 |
Lipid Hydrolysis Therapy for Atherosclerosis and Related Diseases
The present invention comprises a method to diminish and/or eliminate atherosclerotic plaques, in mammals, through direct and indirect treatment of these plaques, in situ, using suitable... |
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US20100111924 |
Chromosomal Modification Involving the Induction of Double-Stranded DNA Cleavage and Homologous Recombination at the Cleavage Site
Methods of modifying, repairing, attenuating and inactivating a gene or other chromosomal DNA in a cell are disclosed. Also disclosed are methods of treating or prophylaxis of a genetic disease in... |
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US20100111907 |
Methods and compositions for gene inactivation
Disclosed herein are methods and compositions for inactivating CCR-5 genes, using zinc finger nucleases (ZFNs) comprising a zinc finger protein and a cleavage domain or cleavage half-domain.... |
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US20080075708 |
Broad specturm anti-viral therapeutics and prophylaxis
Provided are new compositions and methods for preventing and treating pathogen infection. In particular, provided are compounds having an anchoring domain that anchors the compound to the surface... |
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US20070264249 |
Gene-based lipid hydrolysis therapy for atherosclerosis and related diseases
The present invention comprises a method to diminish and/or eliminate atherosclerotic plaques, in mammals, through direct and indirect treatment of these plaques, in situ, using suitable... |
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US20170037434 |
CHROMOSOMAL MODIFICATION INVOLVING THE INDUCTION OF DOUBLE-STRANDED DNA CLEAVAGE AND HOMOLOGOUS RECOMBINATION AT THE CLEAVAGE SITE
Methods of modifying, repairing, attenuating and inactivating a gene or other chromosomal DNA in a cell are disclosed. Also disclosed are methods of treating or prophylaxis of a genetic disease in... |
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US20160158324 |
METHODS AND COMPOSITIONS FOR CNS DELIVERY OF IDURONATE-2-SULFATASE
The present invention provides, among other things, compositions and methods for CNS delivery of lysosomal enzymes for effective treatment of lysosomal storage diseases. In some embodiments, the... |
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US20160058889 |
PREVENTION OF MUSCULAR DYSTROPHY BY CRISPR/CAS9-MEDIATED GENE EDITING
Duchenne muscular dystrophy (DMD) is an inherited X-linked disease caused by mutations in the gene encoding dystrophin, a protein required for muscle fiber integrity. The disclosure reports... |
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US20150166985 |
METHODS FOR CORRECTING VON WILLEBRAND FACTOR POINT MUTATIONS
Some aspects of this disclosure provide strategies, systems, reagents, methods, and kits that are useful for the targeted editing of nucleic acids, including editing a nucleic acid encoding a... |
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US20150166984 |
METHODS FOR CORRECTING ALPHA-ANTITRYPSIN POINT MUTATIONS
Some aspects of this disclosure provide strategies, systems, reagents, methods, and kits that are useful for the targeted editing of nucleic acids, including editing a nucleic acid encoding a... |
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US20150166982 |
METHODS FOR CORRECTING PI3K POINT MUTATIONS
Some aspects of this disclosure provide strategies, systems, reagents, methods, and kits that are useful for the targeted editing of nucleic acids, including editing a nucleic acid encoding a... |
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US20150071879 |
Stabilization of Aqueous Compositions of Proteins with Displacement Buffers
An aqueous composition having increased protein stability is obtained by: a. determining a pH at which the protein has stability at the desired temperature; b. adding to the composition at least... |
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US20150004134 |
TREATMENT OF CANCER WITH HETEROCYCLIC INHIBITORS OF GLUTAMINASE
The invention relates to novel heterocyclic compounds and pharmaceutical preparations thereof. The invention further relates to methods of treating or preventing cancer using the novel... |
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US20140348752 |
METHODS FOR TREATING LYSOSOMAL ACID LIPASE DEFICIENCY IN PATIENTS
The present invention provides methods of treating LAL deficiency comprising administering to a mammal a therapeutically effective amount of lysosomal acid lipase with an effective dosage... |
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US20140314735 |
PROCESS FOR CONCENTRATION OF A POLYPEPTIDE
The present invention comprises a method of concentrating a composition comprising a polypeptide of interest and the use of such a concentrated composition for the treatment of diseases in... |
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US20140056868 |
Supercoiled MiniVectors as a Tool for DNA Repair, Alteration and Replacement
In some embodiments the present disclosure provides a composition for targeted alteration of a DNA sequence and methods of altering the targeted DNA sequence using the composition. In some... |
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US20130224172 |
METHODS OF TREATING BEHAVIORAL SYMPTOMS OF NEUROLOGICAL AND MENTAL DISORDERS
Disclosed herein are methods of using coated digestive enzyme preparations and enzyme delivery systems and pharmaceutical compositions comprising the preparations for treatment of subjects having... |
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US20130209437 |
I-CREI HOMING ENDONUCLEASE VARIANTS HAVING NOVEL CLEAVAGE SPECIFICITY AND USE THEREOF
A method for engineering I-CreI homing endonuclease variants able to cleave mutant I-CreI sites having variation in positions ±8 to ±10. A I-CreI homing endonuclease variant obtainable by said... |
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US20120195857 |
Hepatitis C Virus Inhibitors
The present disclosure relates to compounds, compositions and methods for the treatment of hepatitis C virus (HCV) infection. Also disclosed are pharmaceutical compositions containing such... |
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US20110250251 |
Cosmetic Methods And Compositions For Repairing Human Skin
The present invention relates to methods and compositions for repairing adverse effects of the environment, daily stress, sun exposure, or pre-mature aging on human skin, comprising applying to... |
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US20100322872 |
NOVEL METHODS AND MEDICAMENT FOR TREATING INFECTIOUS DISEASES INVOLVING MICROBIAL BIOFILMS
The present invention provides a method for prophylaxis and therapy of infectious diseases caused by microorganisms present in biofilms adherent to cell surfaces comprising the step of... |
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US20100143384 |
NOVEL 4-CYANO, 4-AMINO, AND 4-AMINOMETHYL DERIVATIVES OF PYRAZOLO[1,5-a]PYRIDINES, PYRAZOLO[1,5-c]PYRIMIDINES AND 2H-INDAZOLE COMPOUNDS AND 5-CYANO, 5-AMINO, AND 5-AMINOMETHYL DERIVATIVES OF IMIDAZO[1,2-a]PYRIDINES, AND IMIDAZO[1,5-a]PYRAZINES AS CYCLIN DEPENDENT KINASE INHIBITORS
In its many embodiments, the present invention provides a novel class of 4-cyano, 4-amino, and 4-aminomethyl derivatives of pyrazolo[1,5-a]pyridine, pyrazolo[1,5-c]pyrimidine, and 2H-Indazole... |
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US20090269303 |
METHOD FOR TREATING OR PREVENTING CONDITIONS BY ELEVATION OF THE ADENOSINE LEVEL IN AN INDIVIDUAL
This invention concerns a method of inducing an elevated level of adenosine in an individual by administering to the individual an effective amount of either i) recombinant protein CD73 or ii) a... |
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US20090202516 |
INHIBITION AND TREATMENT OF GASTROINTESTINAL BIOFILMS
Orally administered physiologically acceptable anti-biofilm compositions comprising enzymes and if desired additional components such as antimicrobials, antibiotics, antifungals, herbals,... |
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US20090087412 |
Methods and compositions relating to HDAC 4 and 5 regulation of cardiac gene expression
The present invention relates to cardiac hypertrophy. More particularly, the present invention defines the molecular events linking calcium stimulation to cardiac hypertrophy. More specifically,... |
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US20080226617 |
Pharmaceutical Preparation and Method of Treatment of Human Malignancies with Arginine Deprivation
The present invention provides an isolated and substantially purified recombinant human arginase having sufficiently high enzymatic activity and stability to maintain Adequate Arginine Depletion... |
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US20070166368 |
Protein-Stabilized Liposomal Formulations of Pharmaceutical Agents
The present invention relates to protein stabilized liposomes. Specifically, the present invention discloses compositions and methods for protein stabilized liposomes, the creation of protein... |
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US20170087225 |
COMPOSITIONS AND METHODS FOR LATENT VIRAL TRANSCRIPTION REGULATION
The invention provides compositions and methods that can be used to regulate viral transcription. Using a catalytically inactive nuclease such as deactivated Cas9, or dCas9, a guide RNA can be... |
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US20160160195 |
ANTI-COCAINE COMPOSITIONS AND TREATMENT
Embodiments of the invention disclosed herein generally relate to anti-cocaine therapeutics. Specifically, some embodiments of the invention relate to highly efficient, thermostable, and... |
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US20160051472 |
PANCREATIC ENZYME COMPOSITIONS AND METHODS FOR TREATING PANCREATITIS AND PANCREATIC INSUFFICIENCY
Compositions of the present invention, comprising the combination of enterically coated and uncoated pancreatic enzyme-containing beads are useful for treating or preventing pancreatitis pain, and... |
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US20150374656 |
Compositions and methods of use of phorbol esters
Methods and compositions containing a phorbol ester or a derivative of a phorbol ester are provided for the treatment of cytopathic diseases. Cytopathic diseases may be caused by a variety means... |
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US20130115297 |
STABLE, DURABLE GRANULES WITH ACTIVE AGENTS
A stable, durable granule for feed compositions has a core, at least one active agent; and at least one coating. The active agent of the granule retains at least 50% activity, at least 60%... |