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US20140189896 CRISPR-CAS COMPONENT SYSTEMS, METHODS AND COMPOSITIONS FOR SEQUENCE MANIPULATION  
The invention provides for systems, methods, and compositions for manipulation of sequences and/or activities of target sequences. Provided are vectors and vector systems, some of which encode one...
US20130243759 Transgenic Animals  
The present invention relates inter alia to fertile non-human vertebrates such as mice and rats useful for producing antibodies bearing human variable regions, in which endogenous antibody chain...
US20130117870 GENETICALLY MODIFIED ANIMALS AND METHODS FOR MAKING THE SAME  
Compositions and methods for use of TALENs to make genetically modified livestock or other animals are set forth. Some of the embodiments of the invention provide for making an founder animal that...
US20120222143 GENETICALLY MODIFIED ANIMALS AND METHODS FOR MAKING THE SAME  
Compositions and methods for use of TALENs to make genetically modified livestock are set forth. The methods may include reporters for selecting cells or embryos that have been modified by TALENs...
US20120204278 ANIMAL MODELS AND THERAPEUTIC MOLECULES  
The invention discloses methods for the generation of chimaeric human—non-human antibodies and chimaeric antibody chains, antibodies and antibody chains so produced, and derivatives thereof...
US20070093443 Modified proteases that inhibit complement activation  
Provided are methods for and compounds for modulating the complement system. In particular, compounds are provided that inhibit complement activation and compounds are provided that promote...
US20050239202 Methods and compositions for inhibiting the function of polynucleotide sequences  
A therapeutic composition for inhibiting the function of a target polynucleotide sequence in a mammalian cell includes an agent that provides to a mammalian cell an at least partially...
US20170044569 RNA-Guided Human Genome Engineering  
A method of altering a eukaryotic cell is provided including transfecting the eukaryotic cell with a nucleic acid encoding RNA complementary to genomic DNA of the eukaryotic cell, transfecting the...
US20160046960 METHODS AND COMPOSITIONS FOR TARGETED GENETIC MODIFICATIONS AND METHODS OF USE  
Methods and compositions are provided for generating targeted genetic modifications on the Y chromosome or a challenging target locus. Compositions include an in vitro culture comprising an XY...
US20150050738 COMPOSITIONS AND METHODS FOR MODULATING RNA  
Aspects of the invention relate to methods for increasing gene expression in a targeted manner. In some embodiments, methods and compositions are provided that are useful for posttranscriptionally...
US20140351965 SWINE GENETICALLY MODIFIED WITH SPECIFICITY FOR LDL-R KNOCKOUT  
Compositions and methods for swine LDL-R gene knockouts.
US20140342456 RNA-Guided Human Genome Engineering  
A method of altering a eukaryotic cell is provided including transfecting the eukaryotic cell with a nucleic acid encoding RNA complementary to genomic DNA of the eukaryotic cell, transfecting the...
US20140322812 Mutant Receptors and Their Use in a Nuclear Receptor-Based Inducible Gene Expression System  
This invention relates to the field of biotechnology or genetic engineering. Specifically, this invention relates to the field of gene expression. More specifically, this invention relates to...
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...
US20140242699 DELIVERY, ENGINEERING AND OPTIMIZATION OF SYSTEMS, METHODS AND COMPOSITIONS FOR SEQUENCE MANIPULATION AND THERAPEUTIC APPLICATIONS  
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...
US20140235933 GENETIC MODIFICATION OF RATS  
Compositions and methods are provided for making rat pluripotent and totipotent cells, including rat embryonic stem (ES) cells. Compositions and methods for improving efficiency or frequency of...
US20130315980 CYCLODEXTRIN-BASED MATERIALS, COMPOSITIONS AND USES RELATED THERETO  
This application discloses cyclodextrin-modified materials for carrying drugs and other active agents, such as nucleic acids. Compositions are also disclosed of cyclodextrin-modified materials...
US20130232588 Chimeric Retinoid X Receptors and Their Use in a Novel Ecdysone Receptor-Based Inducible Gene Expression System  
This invention relates to the field of biotechnology or genetic engineering. Specifically, this invention relates to the field of gene expression. More specifically, this invention relates to a...
US20120328584 Biological Pacemaker  
Disclosed are methods and systems for modulating electrical behavior of cardiac cells. Preferred methods include administering a polynucleotide or cell-based composition that can modulate cardiac...
US20120301429 DAC HYP COMPOSITIONS AND METHODS  
The present disclosure relates to compositions of daclizumab suitable for subcutaneous administration and methods of manufacturing thereof.
US20120276579 ORGANIC COMPOUNDS  
The present invention relates to a novel selection system for use in a eukaryotic cell culture process and for expression of a recombinant product of interest. The selection system is based on the...
US20120095080 METHODS FOR PRODUCING INTERFERING RNA MOLECULES IN MAMMALIAN CELLS AND THERAPEUTIC USES FOR SUCH MOLECULES  
Methods for producing interfering RNA molecules in mammalian cells are provided. Therapeutic uses for the expressed molecules, including inhibiting expression of HIV, are also provided.
US20120030783 RATIONALLY DESIGNED MEGANUCLEASES WITH ALTERED SEQUENCE SPECIFICITY AND DNA-BINDING AFFINITY  
Rationally-designed LAGLIDADG meganucleases and methods of making such meganucleases are provided. In addition, methods are provided for using the meganucleases to generate recombinant cells and...
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...
US20100143314 In Vivo Production and Delivery of Erythropoietin or Insulinotropin for Gene Therapy  
The present invention relates to transfected primary and secondary somatic cells of vertebrate origin, particularly mammalian origin, transfected with exogenous genetic material (DNA) which...
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....
US20090263900 Linear donor constructs for targeted integration  
Disclosed herein are linear donor molecules comprising homology arms of 50-750 base pairs (e.g., 50-100 base pairs) flanking one or more sequences of interest. The donor molecules and/or...
US20090226460 COMPOSITIONS AND METHODS FOR THE TREATMENT OF DISEASE  
The present invention relates to pharmaceutical compositions for the treatment and/or prophylaxis of disease associated with fibrosis in a vertebrate, said composition comprising at least one...
US20090136465 Therapeutic Gene-Switch Constructs and Bioreactors for the Expression of Biotherapeutic Molecules, and Uses Thereof  
The present invention relates to methods and compositions for treating, ameliorating or preventing a disease or disorder in a subject by introducing into cells of the subject a therapeutic gene...
US20090111106 Vector System  
The present invention provides a vector system comprising a mutated post-transcriptional regulatory element. In particular, the present invention relates to a mutated WPRE sequence that can...
US20090088398 RECOMBINANT ADENOVIRAL VECTORS AND METHODS OF USE  
This invention provides a recombinant adenovirus expression vector characterized by the partial or total deletion of the adenoviral protein IX DNA and having a gene encoding a foreign protein or a...
US20080221054 Inhibiting Gene Expression with dsRNA  
The present invention relates to the specific inhibition of gene expression in mammals by bringing the target gene into contact with double stranded RNA (dsRNA).
US20080209581 Induction of exon skipping in eukaryotic cells  
Described is a method for at least in part decreasing the production of an aberrant protein in a cell, the cell comprising pre-mRNA comprising exons coding for the protein, by inducing so-called...
US20080194514 Methods and Compositions for Inhibiting the Function of Polynucleotide Sequences  
A therapeutic composition for inhibiting the function of a target polynucleotide sequence in a mammalian cell includes an agent that provides to a mammalian cell an at least partially...
US20080182810 ANTISENSE MODULATION OF ACYL COA CHOLESTEROL ACYLTRANSFERASE-2 EXPRESSION  
Antisense compounds, compositions and methods are provided for modulating the expression of acyl CoA cholesterol acyltransferase-2. The compositions comprise antisense compounds, particularly...
US20080166295 Antibodies, polypeptides and uses thereof  
Provided are methods of inhibiting angiogenesis in an individual in need thereof comprising administering an antibody that selectively binds to the extracellular region of human magic roundabout...
US20080159977 Rational evolution of cytokines for higher stability, the cytokines and encoding nucleic acid molecules  
Compositions of modified cytokines and uses thereof generated using processes and systems for the high throughput directed evolution of peptides and proteins, particularly cytokines that act in...
US20080050345 Adeno-associated virus serotype I nucleic acid sequences, vectors and host cells containing same  
The nucleic acid sequences of adeno-associated virus (AAV) serotype 1 are provided, as are vectors and host cells containing these sequences and functional fragments thereof. Also provided are...
US20070238178 Methods and compositions for inhibiting the function of polynucleotide sequences  
A therapeutic composition for inhibiting the function of a target polynucleotide sequence in a mammalian cell includes an agent that provides to a mammalian cell an at least partially...
US20070219151 Methods and compositions for inhibiting the function of polynucleotide sequences  
A therapeutic composition for inhibiting the function of a target polynucleotide sequence in a mammalian cell includes an agent that provides to a mammalian cell an at least partially...
US20070179107 DOUBLE STRAND COMPOSITIONS COMPRISING DIFFERENTIALLY MODIFIED STRANDS FOR USE IN GENE MODULATION  
The present invention provides double stranded compositions wherein each strand is modified to have a motif defined by positioning of β-D-ribonucleosides and sugar modified nucleosides. More...
US20070179106 DOUBLE STRAND COMPOSITIONS COMPRISING DIFFERENTIALLY MODIFIED STRANDS FOR USE IN GENE MODULATION  
The present invention provides double stranded compositions wherein each strand is modified to have a motif defined by positioning of β-D-ribonucleosides and sugar modified nucleosides. More...
US20070172948 DOUBLE STRAND COMPOSITIONS COMPRISING DIFFERENTIALLY MODIFIED STRANDS FOR USE IN GENE MODULATION  
The present invention provides double stranded compositions wherein each strand is modified to have a motif defined by positioning of β-D-ribonucleosides and sugar modified nucleosides. More...
US20070172504 In vivo cell surface engineering  
The present invention provides methods and compositions for the in vivo engineering of cell surfaces, such as tumor cell surfaces, with one or more immune co-stimulatory polypeptides. The methods,...
US20060252104 Methods of selecting compounds for modulation of bladder function  
The present invention involves an assay system and method of selecting compounds useful in the treatment of bladder instability and related bladder conditions through the activation of KCNQ...
US20060246586 Chromosome-based platforms  
Artificial chromosomes, including ACes, that have been engineered to contain available sites for site-specific, recombination-directed integration of DNA of interest are provided. These artificial...
US20060134098 Immunoglobulin variable region cassette exchange  
The invention provides methods for generating human antibodies with the specificity of a reference antibody by replacement of portions of the VH and VL sequences of the reference antibody with...
US20060040882 Compostions and methods for enhancing delivery of nucleic acids into cells and for modifying expression of target genes in cells  
Polynucleotide delivery-enhancing polypeptides are admixed or complexed with, or conjugated to, nucleic acids for enhancing delivery the nucleic acids into cells. The transported nucleic acids are...
US20050277610 Methods and compositions for the specific inhibition of gene expression by double-stranded RNA  
The invention provides compositions and methods for selectively reducing the expression of a gene product from a desired target gene, as well as treating diseases caused by expression of the gene....
US20050215502 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.