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1
US10214723B2
Publication/Patent Number: US10214723B2
Publication date: 2019-02-26
Application number: 15/209,516
Filing date: 2016-07-13
Abstract: The present invention relates to a method of producing a non-human, mammalian oocyte carrying a modified target sequence in its genome, the method comprising the steps of introducing into a non-human, mammalian oocyte: (a) a clustered, regularly interspaced, short palindromic repeats (CRISPR)-associated protein 9 (Cas9 protein) or a nucleic acid molecule encoding said Cas9 protein; and (b-i) a target sequence specific CRISPR RNA (crRNA) and a trans-activating crRNA (tracr RNA) or a nucleic acid molecule encoding said RNAs; or (b-ii) a chimaeric RNA sequence comprising a target sequence specific crRNA and tracrRNA or a nucleic acid molecule encoding said RNA; wherein the Cas9 protein introduced in (a) and the RNA sequence(s) introduced in (b-i) or (b-ii) form a protein/RNA complex that specifically binds to the target sequence and introduces a single or double strand break within the target sequence. The present invention further relates to the method of the invention, wherein the target sequence is modified by homologous recombination with a donor nucleic acid sequence further comprising the step: (c) introducing a nucleic acid molecule into the cell, wherein the nucleic acid molecule comprises the donor nucleic acid sequence and regions homologous to the target sequence. The present invention also relates to a method of producing a non-human mammal carrying a modified target sequence in its genome. The present invention relates to a method of producing a non-human, mammalian oocyte carrying a modified target sequence in its genome, the method comprising the steps of introducing into a non-human, mammalian oocyte: (a) a clustered, regularly interspaced, short palindromic ...more ...less
2
US10214731B2
Publication/Patent Number: US10214731B2
Publication date: 2019-02-26
Application number: 15/167,729
Filing date: 2016-05-27
Abstract: This invention is directed to a vector which comprises a promoter operably linked to a nucleic acid sequence encoding the human C1 esterase inhibitor or Factor XII. The invention is also directed to a composition comprising the vector and a method of using the vector to treat or prevent hereditary angioedema. This invention is directed to a vector which comprises a promoter operably linked to a nucleic acid sequence encoding the human C1 esterase inhibitor or Factor XII. The invention is also directed to a composition comprising the vector and a method of using the vector to treat or ...more ...less
3
US10219493B2
Publication/Patent Number: US10219493B2
Publication date: 2019-03-05
Application number: 15/413,785
Filing date: 2017-01-24
Abstract: The invention provides genetically modified non-human animals that express a humanized MHC II protein (humanized MHC II α and β polypeptides), as well as embryos, cells, and tissues comprising the humanized MHC II protein. Also provided are constructs for and methods of making said genetically modified non-human animals. Methods of using the genetically modified non-human animals to study various aspects of the human immune system are provided. The invention provides genetically modified non-human animals that express a humanized MHC II protein (humanized MHC II α and β polypeptides), as well as embryos, cells, and tissues comprising the humanized MHC II protein. Also provided are constructs for and methods ...more ...less
4
US10285387B2
Publication/Patent Number: US10285387B2
Publication date: 2019-05-14
Application number: 15/072,286
Filing date: 2016-03-16
Abstract: An animal model for motor neuron dysfunction in disease, e.g., amyotrophic lateral sclerosis (ALS), comprising a genetically modified non-human animal that comprises a genetically modified DR6 allele and exhibits normal phenotypes at birth and for a few weeks or months after birth. However, as the non-human animal ages, it develops motor neuron dysfunction that presents as one or more ALS-like symptoms, which may progress rapidly after onset. Methods of identifying candidate agents that may be used to prevent, delay or treat ALS are also provided. An animal model for motor neuron dysfunction in disease, e.g., amyotrophic lateral sclerosis (ALS), comprising a genetically modified non-human animal that comprises a genetically modified DR6 allele and exhibits normal phenotypes at birth and for a few weeks or months after ...more ...less
5
US10238093B2
Publication/Patent Number: US10238093B2
Publication date: 2019-03-26
Application number: 13/788,997
Filing date: 2013-03-07
Abstract: Mice, embryos, cells, and tissues having a restricted immunoglobulin heavy chain locus and an ectopic sequence encoding one or more ADAM6 proteins are provided. In various embodiments, mice are described that have humanized endogenous immunoglobulin heavy chain loci and are capable of expressing an ADAM6 protein or ortholog or homolog or functional fragment thereof that is functional in a male mouse. Mice, embryos, cells, and tissues having an immunoglobulin heavy chain locus characterized by a single human VH gene segment, a plurality of human DH gene segments and a plurality of human JH gene segments and capable expressing an ADAM6 protein or ortholog or homolog or functional fragment thereof are also provided. Mice, embryos, cells, and tissues having a restricted immunoglobulin heavy chain locus and an ectopic sequence encoding one or more ADAM6 proteins are provided. In various embodiments, mice are described that have humanized endogenous immunoglobulin heavy chain loci and are ...more ...less
6
US10246679B2
Publication/Patent Number: US10246679B2
Publication date: 2019-04-02
Application number: 14/635,331
Filing date: 2015-03-02
Abstract: The present invention provides a method for putting a primed pluripotent stem cell in a less differentiated state, a method for producing a pluripotent stem cell having an improved chimera forming ability from a primed pluripotent stem cell, and a method for producing a chimeric animal by using the pluripotent stem cell obtained by any of these methods. The present invention also provides a composition containing a Wnt/β catenin signal inhibitor. The present invention provides a method for putting a primed pluripotent stem cell in a less differentiated state, a method for producing a pluripotent stem cell having an improved chimera forming ability from a primed pluripotent stem cell, and a method for producing a chimeric ...more ...less
7
US10244739B2
Publication/Patent Number: US10244739B2
Publication date: 2019-04-02
Application number: 14/427,776
Filing date: 2013-03-01
Abstract: The present invention relates to a genetically edited animal, especially to a genetically edited pig in which expression or activity of the RELA protein has been modified. Such pigs have at least partial protection against the African Swine Fever Virus. The invention also provides, a cell nucleus, germ cell, stem cell, gamete, blastocyst, embryo, foetus and/or donor cell of a non-human animal comprising a genetic modification which alters the expression or function of RELA protein, methods for editing the genome of animals and methods for screening the efficacy of a pharmaceutical agent in such an animal. The present invention relates to a genetically edited animal, especially to a genetically edited pig in which expression or activity of the RELA protein has been modified. Such pigs have at least partial protection against the African Swine Fever Virus. The invention also ...more ...less
8
US10266848B2
Publication/Patent Number: US10266848B2
Publication date: 2019-04-23
Application number: 16/017,157
Filing date: 2018-06-25
Abstract: The present invention provides methods and compostions to improve the efficiency of somatic cell nuclear transfer (SCNT) and the consequent production of nuclear transfer ESC (ntESC) and transgenic cells and/or non-human animals. More specifically, the present invention relates to the discovery that trimethylation of Histone H3-Lysine 9 (H3K9me3) in reprogramming resistant regions (RRRs) in the nuclear genetic material of donor somatic cells prevents efficient somatic cell nuclear reprogramming or SCNT. The present invention provide methods and compositions to decrease H3K9me3 in methods to improve efficacy of SCNT by exogenous or overexpression of the demethylase Kdm4 family and/or inhibiting methylation of H3K9me3 by inhibiting the histone methyltransferases Suv39h1 and/or Suv39h2. The present invention provides methods and compostions to improve the efficiency of somatic cell nuclear transfer (SCNT) and the consequent production of nuclear transfer ESC (ntESC) and transgenic cells and/or non-human animals. More specifically, the present invention relates ...more ...less
9
US10207012B2
Publication/Patent Number: US10207012B2
Publication date: 2019-02-19
Application number: 15/265,212
Filing date: 2016-09-14
Assignee: Biocrine AB
Abstract: The present invention provides novel drug discovery platforms and methods for treating type I diabetes.
10
US10196431B2
Publication/Patent Number: US10196431B2
Publication date: 2019-02-05
Application number: 15/803,472
Filing date: 2017-11-03
Abstract: Provided herein are compositions comprising light-activated chimeric proteins expressed on plasma membranes and methods of using the same to selectively depolarize excitatory or inhibitory neurons.