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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...
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...
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...
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...
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...
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...
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...
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...
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...
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...
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...
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...
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....
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...
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...
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...
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...
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...
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...
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...
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....
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...
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...
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...
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...
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...
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...
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...
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...
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...
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...
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...
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...
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...
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...
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...
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...
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...
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...
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...
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...
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,...
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,...
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...
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...
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...
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...
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...
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...
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%...