Abstract: The present invention relates to antisense RNAs targeting PMP22 and able to inhibit from 40% to 60% the expression of PMP22 in the cells and a pharmaceutical composition comprising thereof. The antisense RNAs are preferably siRNA and are preferably provided in the form of nanoparticles. The present invention also relates to the use of these antisense RNAs targeting PMP22 for the treatment of Charcot-Marie-Tooth 1A disease.

Abstract: Devices and methods are presented for delivery of various macrostructures into cells, such as depleted cells as well as methods of generating engineered cells using said devices and methods. Cells are placed on a porous membrane. A force is applied by a configurable actuator to a deformable fluid reservoir that generates an applied pressure to macrostructures in a solution, causing the macrostructures to pass through the porous membrane and triggering uptake of at least some of the macrostructures into the cells to form transfected cells. Said devices and methods may be used in a process to replace defective endogenous mtDNA with corrected mtDNA to generate cellular-based therapeutics for administration to a patient. The customizable actuator may be configured with parameters to optimize efficiency for a given cell type and material to be transfected. Machine learning techniques also may be utilized to optimize transfection parameters.

Abstract: The present description relates to a conjugated compound comprising cholic acid (ChAc) or a variant thereof, the ChAc conjugated to a non-cell penetrating peptide comprising a nuclear localization sequence (NLS) conjugated to a compound of interest.

Abstract: A method of modifying the genome of an organism, wherein the modification method includes modifying the genome of the organism by using in a cell of the organism a protein having an optimal temperature for double-stranded DNA breakage activity in an ordinary temperature region.

Abstract: The present invention relates to compositions and methods for the treatment of nucleotide repeat expansion disorders such as myotonic dystrophy.

Abstract: The present invention relates to a method of converting human fibroblasts into neural stem cells, and more particularly, to a method of directly converting human fibroblasts into neural stem cells using only a combination of small-molecule compounds without any introduction of a foreign gene, and to the use of the neural stem cells. The method of directly converting human fibroblasts into neural stem cells using only small-molecule compounds without any introduction of a foreign gene makes it possible to obtain genetically stable neural stem cells in an amount sufficient for use in cell therapy by deriving them from human fibroblasts. The neural stem cells obtained according to the method of the present invention can differentiate into functional neural cells and are not tumorigenic. Thus, these neural stem cells are useful as cellular therapeutic agents for treatment of brain diseases.

Abstract: The present invention concerns new modular base-per-base specific nucleic acid binding domains (MBBBD) derived from newly identified proteins from the bacterial endosymbiont Burkholderia Rhizoxinica and their use for engineering nucleic acid processing enzymes, such as specific endonucleases or transcription activators.

Abstract: Methods and compositions comprising single guide RNAs targeted to an endogenous mammalian ? globin gene (HBB), gamma globin gene (HBG1), B-cell lymphoma/leukemia 11A (BCL11A) gene, Kruppel-like factor 1 (KLF1) gene, CCR5 gene, CXCR4 gene, PPP1R12C (AAVS1) gene, hypoxanthine phosphoribosyltransferase (HPRT) gene, albumin gene, Factor VIII gene, Factor IX gene, Leucine-rich repeat kinase 2 (LRRK2) gene, Hungtingin (Htt) gene, rhodopsin (RHO) gene, Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene, surfactant protein B gene (SFTPB), T-cell receptor alpha (TRAC) gene, T-cell receptor beta (TRBC) gene, programmed cell death 1 (PD1) gene, Cytotoxic T-Lymphocyte Antigen 4 (CTLA-4) gene, human leukocyte antigen (HLA) A gene, a LMP7 gene, a Transporter associated with Antigen Processing (TAP) gene, tapasin gene (TAPBP), a class II major histocompatibility complex transactivator (CIITA) gene, a dystrophin gene (DMD), a glucocorticoid receptor gene (GR), IL2RG gene or RFX5 for genetic alteration of cells a

Abstract: The present invention relates to a bacterial cell with texturizing property, starter cultures comprising the cell, and dairy products fermented with the starter culture.

Abstract: The instant invention provides for methods for detecting the internalization of a transmembrane protein of interest expressed at the surface of a cell. More specifically, the methods involve (a) labelling the protein of interest with a fluorescent metal complex, the lifetime of which is greater than 0.1 ms, (b) adding to the reaction medium a composition capable of causing the internalization of the protein of interest, (c) adding to the reaction medium (1) a modulating agent which is a fluorescent FRET acceptor compound compatible with the fluorescent metal complex, the final concentration of which in the reaction medium is greater than 10?7M; or (2) a reducing agent, the redox potential of which is less than +0.1 V and preferably between 0.25 and 0.

Abstract: Compositions and methods are provided for genome modification of a target sequence in the genome of a plant or plant cell. The methods and compositions employ a guide RNA/Cas endonuclease system to provide an effective system for modifying or altering target sites within the genome of a plant, plant cell or seed. Also provided are compositions and methods employing a guide polynucleotide/Cas endonuclease system for genome modification of a nucleotide sequence in the genome of a cell or organism, for gene editing, and/or for inserting or deleting a polynucleotide of interest into or from the genome of a cell or organism. Once a genomic target site is identified, a variety of methods can be employed to further modify the target sites such that they contain a variety of polynucleotides of interest. Breeding methods and methods for selecting plants utilizing a two component RNA guide and Cas endonuclease system are also disclosed.

Abstract: The present invention relates to isolated polypeptides having cellulolytic enhancing activity and isolated polynucleotides encoding the polypeptides. The invention also relates to nucleic acid constructs, vectors, and host cells comprising the polynucleotides as well as methods of producing and using the polypeptides.

Abstract: Methods of modulating expression of a target nucleic acid in a cell are provided including introducing into the cell a first foreign nucleic acid encoding one or more RNAs complementary to DNA, wherein the DNA includes the target nucleic acid, introducing into the cell a second foreign nucleic acid encoding a nuclease-null Cas9 protein that binds to the DNA and is guided by the one or more RNAs, introducing into the cell a third foreign nucleic acid encoding a transcriptional regulator protein or domain, wherein the one or more RNAs, the nuclease-null Cas9 protein, and the transcriptional regulator protein or domain are expressed, wherein the one or more RNAs, the nuclease-null Cas9 protein and the transcriptional regulator protein or domain co-localize to the DNA and wherein the transcriptional regulator protein or domain regulates expression of the target nucleic acid.

Abstract: 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 eukaryotic cell with a nucleic acid encoding an enzyme that interacts with the RNA and cleaves the genomic DNA in a site specific manner, wherein the cell expresses the RNA and the enzyme, the RNA binds to complementary genomic DNA and the enzyme cleaves the genomic DNA in a site specific manner.

Abstract: 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 eukaryotic cell with a nucleic acid encoding an enzyme that interacts with the RNA and cleaves the genomic DNA in a site specific manner, wherein the cell expresses the RNA and the enzyme, the RNA binds to complementary genomic DNA and the enzyme cleaves the genomic DNA in a site specific manner.

Abstract: The present invention relates to the generation of stable haploid cell cultures, uses of said cells in forward and reverse genetics, especially the identification of target genes associated with a modified phenotype and in particular identifying genetic targets associated with toxin resistance, especially ricin toxicity resistance, and therapeutic uses of target compounds.

Abstract: Provided are isolated polypeptides having xylanase activity, catalytic domains and cellulose binding domains, and polynucleotides encoding the polypeptides, catalytic domains or cellulose binding domains. Also provided are nucleic acid constructs, vectors and host cells comprising the polynucleotides as well as methods of producing and using the polypeptides, catalytic domains or cellulose binding domains.

Abstract: Materials and Methods related to gene targeting (e.g., gene targeting with transcription activator-like effector nucleases; “TALENS”) are provided.

Abstract: A method for designing a protein capable of binding in an RNA base selective manner or RNA base sequence specific manner is provided.

Abstract: The present invention provides a newly isolated bacterial strain of Paenibacillus, designated Paenibacillus alvei strain TS-15 for use as a biocontrol agent in the inhibition and/or elimination of a human foodborne pathogen, e.g., Salmonella, on a plant or plant organ, e.g., a tomato or tomato plant. Strain TS-15 or mutants thereof may also be used in the control of plant pathogens.

Abstract: The present invention relates to isolated polypeptides having beta-glucosidase activity, beta-xylosidase activity, or beta-glucosidase and beta-xylosidase activity and isolated polynucleotides encoding the polypeptides. The invention also relates to nucleic acid constructs, vectors, and host cells comprising the polynucleotides as well as methods of producing and using the polypeptides.

Abstract: The present invention relates to isolated polypeptides having cellulolytic enhancing activity and polynucleotides encoding the polypeptides. The invention also relates to nucleic acid constructs, vectors, and host cells comprising the polynucleotides as well as methods of producing and using the polypeptides.

Abstract: Disclosed herein are methods and compositions for modifying TCR genes, using nucleases (zinc finger nucleases or TAL nucleases) to modify TCR genes.

Abstract: Methods and compositions for reducing expression of genes on Chromosome 21 (“Chr 21”) by targeting an XIST transgene to the Dual specificity tyrosine-phosphorylation-regulated kinase 1A (DYRK1A) gene or a Regulator of calcineurin 1 (RCAN1) gene, and cells and transgenic animals comprising an XIST transgene inserted into a DYRK1A or RCAN1 allele, e.g., cells and animals trisomic for human Chr 21 and mouse Chr 16.

Abstract: Plant metabolism and alkaloid levels can be regulated by transcription factors that regulate the nicotinic alkaloid biosynthetic pathway. In one embodiment, the disclosure provides a transcription factor that negatively regulates alkaloid biosynthesis, such as nicotine biosynthesis.

Abstract: The present invention concerns new modular base-per-base specific nucleic acid binding domains (MBBBD) derived from newly identified proteins from the bacterial endosymbiont Burkholderia Rhizoxinica and their use for engineering nucleic acid processing enzymes, such as specific endonucleases or transcription activators.

Abstract: The present invention provides compositions and methods for DNA targeting using TAL effectors and TAL effector based proteins, including but not limited to targeted gene regulation and targeted cleavage of cellular chromatin in a region of interest and/or homologous recombination at a predetermined site in cells. Compositions include fusion polypeptides comprising a TAL effector or a TAL effector binding domain in combination with other domains, including but not limited to a cleavage domain. The TAL effector binding domain includes modifications that increase activity of the same and also remove the constraints that the DNA target sequence be preceded by a thymine.

Abstract: The present invention provides the use of acid pretreated biomass such as jatropha seed cake, pongamia seed cake and banana stems as renewable feedstock for butanol production by the mutant Clostridium acetobutylicum MTCC 587. Chemical mutagenesis was carried out for improvement of the strain for better butanol tolerance and production. This present invention for the first time has reported an efficient process for high yield of butanol obtained in single batch fermentation using acid pretreated jatropha seed cake.

Abstract: Isolated peptides comprising nuclear targeting activity or being capable of preventing endogenous nuclear targeting activity are disclosed. Polynucleotides encoding same, pharmaceutical compositions comprising same, as well as uses thereof are also disclosed.

Abstract: A series of independent non-transgenic mutations found at the waxy loci of wheat; wheat plants having these mutations in their waxy loci; and a method of creating and finding similar and/or additional mutations of the waxy by screening pooled and/or individual wheat plants. The wheat plants of the present invention exhibiting altered waxy activity in the wheat without having the inclusion of foreign nucleic acids in their genomes. The invention also includes food and non-food products as well as non-food products that incorporate seeds from the wheat plants having non-transgenic mutations in one or more waxy genes.

Abstract: A soybean cultivar designated 1906039 is disclosed. The invention relates to the seeds of soybean cultivar 1906039, to the plants of soybean cultivar 1906039, to the plant parts of soybean cultivar 1906039, and to methods for producing progeny of soybean cultivar 1906039. The invention also relates to methods for producing a soybean plant containing in its genetic material one or more transgenes and to the transgenic soybean plants and plant parts produced by those methods. The invention also relates to soybean cultivars or breeding cultivars, and plant parts derived from soybean cultivar 1906039. The invention also relates to methods for producing other soybean cultivars, lines, or plant parts derived from soybean cultivar 1906039, and to the soybean plants, varieties, and their parts derived from use of those methods. The invention further relates to hybrid soybean seeds, plants, and plant parts produced by crossing cultivar 1906039 with another soybean cultivar.

Abstract: The present invention relates to methods and compositions for engineering Clostridia species. In particular, embodiments of the present invention relate to the expression of recombinant resolvase proteins in Clostridia species.

Abstract: A soybean cultivar designated S110128 is disclosed. The invention relates to the seeds of soybean cultivar S110128, to the plants of soybean cultivar S110128, to the plant parts of soybean cultivar S110128, and to methods for producing progeny of soybean cultivar S110128. The invention also relates to methods for producing a soybean plant containing in its genetic material one or more transgenes and to the transgenic soybean plants and plant parts produced by those methods. The invention also relates to soybean cultivars or breeding cultivars, and plant parts derived from soybean cultivar S110128. The invention also relates to methods for producing other soybean cultivars, lines, or plant parts derived from soybean cultivar S110128, and to the soybean plants, varieties, and their parts derived from use of those methods. The invention further relates to hybrid soybean seeds, plants, and plant parts produced by crossing cultivar S110128 with another soybean cultivar.

Abstract: A soybean cultivar designated S100108 is disclosed. The invention relates to the seeds of soybean cultivar S100108, to the plants of soybean cultivar S100108, to the plant parts of soybean cultivar S100108, and to methods for producing progeny of soybean cultivar S100108. The invention also relates to methods for producing a soybean plant containing in its genetic material one or more transgenes and to the transgenic soybean plants and plant parts produced by those methods. The invention also relates to soybean cultivars or breeding cultivars, and plant parts derived from soybean cultivar S100108. The invention also relates to methods for producing other soybean cultivars, lines, or plant parts derived from soybean cultivar S100108, and to the soybean plants, varieties, and their parts derived from use of those methods. The invention further relates to hybrid soybean seeds, plants, and plant parts produced by crossing cultivar S100108 with another soybean cultivar.

Abstract: A soybean cultivar designated S100242 is disclosed. The invention relates to the seeds of soybean cultivar S100242, to the plants of soybean cultivar S100242, to the plant parts of soybean cultivar S100242, and to methods for producing progeny of soybean cultivar S100242. The invention also relates to methods for producing a soybean plant containing in its genetic material one or more transgenes and to the transgenic soybean plants and plant parts produced by those methods. The invention also relates to soybean cultivars or breeding cultivars, and plant parts derived from soybean cultivar S100242. The invention also relates to methods for producing other soybean cultivars, lines, or plant parts derived from soybean cultivar S100242, and to the soybean plants, varieties, and their parts derived from use of those methods. The invention further relates to hybrid soybean seeds, plants, and plant parts produced by crossing cultivar S100242 with another soybean cultivar.

Abstract: A soybean cultivar designated S100239 is disclosed. The invention relates to the seeds of soybean cultivar S100239, to the plants of soybean cultivar S100239, to the plant parts of soybean cultivar S100239, and to methods for producing progeny of soybean cultivar S100239. The invention also relates to methods for producing a soybean plant containing in its genetic material one or more transgenes and to the transgenic soybean plants and plant parts produced by those methods. The invention also relates to soybean cultivars or breeding cultivars, and plant parts derived from soybean cultivar S100239. The invention also relates to methods for producing other soybean cultivars, lines, or plant parts derived from soybean cultivar S100239, and to the soybean plants, varieties, and their parts derived from use of those methods. The invention further relates to hybrid soybean seeds, plants, and plant parts produced by crossing cultivar S100239 with another soybean cultivar.

Abstract: A soybean cultivar designated 1347284 is disclosed. The invention relates to the seeds of soybean cultivar 1347284, to the plants of soybean cultivar 1347284, to the plant parts of soybean cultivar 1347284, and to methods for producing progeny of soybean cultivar 1347284. The invention also relates to methods for producing a soybean plant containing in its genetic material one or more transgenes and to the transgenic soybean plants and plant parts produced by those methods. The invention also relates to soybean cultivars or breeding cultivars, and plant parts derived from soybean cultivar 1347284. The invention also relates to methods for producing other soybean cultivars, lines, or plant parts derived from soybean cultivar 1347284, and to the soybean plants, varieties, and their parts derived from use of those methods. The invention further relates to hybrid soybean seeds, plants, and plant parts produced by crossing cultivar 1347284 with another soybean cultivar.

Abstract: A soybean cultivar designated S090059 is disclosed. The invention relates to the seeds of soybean cultivar S090059, to the plants of soybean cultivar S090059, to the plant parts of soybean cultivar S090059, and to methods for producing progeny of soybean cultivar S090059. The invention also relates to methods for producing a soybean plant containing in its genetic material one or more transgenes and to the transgenic soybean plants and plant parts produced by those methods. The invention also relates to soybean cultivars or breeding cultivars, and plant parts derived from soybean cultivar S090059. The invention also relates to methods for producing other soybean cultivars, lines, or plant parts derived from soybean cultivar S090059, and to the soybean plants, varieties, and their parts derived from use of those methods. The invention further relates to hybrid soybean seeds, plants, and plant parts produced by crossing cultivar S090059 with another soybean cultivar.

Abstract: A soybean cultivar designated S100106 is disclosed. The invention relates to the seeds of soybean cultivar S100106, to the plants of soybean cultivar S100106, to the plant parts of soybean cultivar S100106, and to methods for producing progeny of soybean cultivar S100106. The invention also relates to methods for producing a soybean plant containing in its genetic material one or more transgenes and to the transgenic soybean plants and plant parts produced by those methods. The invention also relates to soybean cultivars or breeding cultivars, and plant parts derived from soybean cultivar S100106. The invention also relates to methods for producing other soybean cultivars, lines, or plant parts derived from soybean cultivar S100106, and to the soybean plants, varieties, and their parts derived from use of those methods. The invention further relates to hybrid soybean seeds, plants, and plant parts produced by crossing cultivar S100106 with another soybean cultivar.

Abstract: A soybean cultivar designated S100324 is disclosed. The invention relates to the seeds of soybean cultivar S100324, to the plants of soybean cultivar S100324, to the plant parts of soybean cultivar S100324, and to methods for producing progeny of soybean cultivar S100324. The invention also relates to methods for producing a soybean plant containing in its genetic material one or more transgenes and to the transgenic soybean plants and plant parts produced by those methods. The invention also relates to soybean cultivars or breeding cultivars, and plant parts derived from soybean cultivar S100324. The invention also relates to methods for producing other soybean cultivars, lines, or plant parts derived from soybean cultivar S100324, and to the soybean plants, varieties, and their parts derived from use of those methods. The invention further relates to hybrid soybean seeds, plants, and plant parts produced by crossing cultivar S100324 with another soybean cultivar.

Abstract: A soybean cultivar designated S110121 is disclosed. The invention relates to the seeds of soybean cultivar S110121, to the plants of soybean cultivar S110121, to the plant parts of soybean cultivar S110121, and to methods for producing progeny of soybean cultivar S110121. The invention also relates to methods for producing a soybean plant containing in its genetic material one or more transgenes and to the transgenic soybean plants and plant parts produced by those methods. The invention also relates to soybean cultivars or breeding cultivars, and plant parts derived from soybean cultivar S110121. The invention also relates to methods for producing other soybean cultivars, lines, or plant parts derived from soybean cultivar S110121, and to the soybean plants, varieties, and their parts derived from use of those methods. The invention further relates to hybrid soybean seeds, plants, and plant parts produced by crossing cultivar S110121 with another soybean cultivar.

Abstract: A soybean cultivar designated 11430023 is disclosed. The invention relates to the seeds of soybean cultivar 11430023, to the plants of soybean cultivar 11430023, to the plant parts of soybean cultivar 11430023, and to methods for producing progeny of soybean cultivar 11430023. The invention also relates to methods for producing a soybean plant containing in its genetic material one or more transgenes and to the transgenic soybean plants and plant parts produced by those methods. The invention also relates to soybean cultivars or breeding cultivars, and plant parts derived from soybean cultivar 11430023. The invention also relates to methods for producing other soybean cultivars, lines, or plant parts derived from soybean cultivar 11430023, and to the soybean plants, varieties, and their parts derived from use of those methods. The invention further relates to hybrid soybean seeds, plants, and plant parts produced by crossing cultivar 11430023 with another soybean cultivar.

Abstract: A soybean cultivar designated S110127 is disclosed. The invention relates to the seeds of soybean cultivar S110127, to the plants of soybean cultivar S110127, to the plant parts of soybean cultivar S110127, and to methods for producing progeny of soybean cultivar S110127. The invention also relates to methods for producing a soybean plant containing in its genetic material one or more transgenes and to the transgenic soybean plants and plant parts produced by those methods. The invention also relates to soybean cultivars or breeding cultivars, and plant parts derived from soybean cultivar S110127. The invention also relates to methods for producing other soybean cultivars, lines, or plant parts derived from soybean cultivar S110127, and to the soybean plants, varieties, and their parts derived from use of those methods. The invention further relates to hybrid soybean seeds, plants, and plant parts produced by crossing cultivar S110127 with another soybean cultivar.

Abstract: A method includes providing a eukaryotic cell including mutant mtDNA in which there is at least one mutation in the mtDNA, allowing the cell to come into contact with an active oxygen species or a chemical species that generates such an active oxygen species in the cell (e.g., hydrogen peroxide) and thereby changing the percentage of mutant mtDNA (mitochondrial genomic DNA) in the cell as a result of the contact. Also featured are cells obtained by the above-described method.

Abstract: This invention relates to polypeptides having aldolase activity, including pyruvate activity such as, without limitation, HMG and/or KHG aldolase activity, polynucleotides encoding these polypeptides, and methods of making and using these polynucleotides and polypeptides. In some embodiments, the invention is directed to polypeptides having aldolase activity, including pyruvate activity such as, without limitation, HMG and/or KHG aldolase activity, including thermostable and thermotolerant activity, and polynucleotides encoding these enzymes, and making and using these polynucleotides and polypeptides. The polypeptides in accordance with the invention can be used in a variety of pharmaceutical, agricultural and industrial contexts.

Abstract: Described herein are reprogrammed cells, and methods for cell dedifferentiation, transformation and eukaryotic cell reprogramming. Also descried are cells, cell lines, and tissues that can be transplanted in a patient after steps of in vitro dedifferentiation and in vitro reprogramming. In particular embodiments the cells are Stem-Like Cells (SLCs), including Neural Stem-Like Cells (NSLCs), Cardiac Stem-Like Cells (CSLC), Hematopoietic Stem-Like Cells (HSLC), Pancreatic Progenitor-Like Cells, and Mesendoderm-like Cells. Also described are methods for generating these cells from human somatic cells and other types of cells. Also provided are compositions and methods of using of the cells so generated in human therapy and in other areas.

Abstract: Methods to generate doubled haploid plants and plant components using low mammalian toxicity chromosome doubling agents are disclosed. Chromosome doubling agents provide low mortality rates and higher chromosome doubling rate in plants.

Abstract: A method of selecting cells having zero fucose level useful as host cells for expressing recombinant proteins is disclosed. The method comprises: (d) introducing genetic mutations into a population of CHO cells by contacting the cells with a methotrexate (MTX), (e) contacting the population of CHO cells comprising mutated cells with a non-toxic fucose binding agent for an amount of time that allows binding of the fucose binding agent to a fucose moiety on a cell membrane of the population of cells, wherein the amount of time does not allow killing of the cells; and (f) depleting from the population of cells comprising mutated cells, a subpopulation of cells which bind the fucose binding agent, thereby selecting cells useful as host cells for expressing recombinant proteins, the selected cells having zero fucose content. There are also disclosed cells and cell lines useful as host cells for expressing recombinant proteins.

Abstract: The current invention relates to a method for targeted alteration of acceptor DNA, for example duplex acceptor DNA. The method comprises the use of an oligonucleotide having at least one mismatch relative to the targeted (duplex) acceptor DNA. The mismatch is located at specific positions within said oligonucleotide. Also provided is a kit that comprises instructions for performing the method according to the inventions, and in a preferred embodiment, comprises an oligonucleotide suitable for use in the method.

Abstract: The present invention relates to a novel anaerobic, extreme thermophilic, ethanol high-yielding bacterium. The invention is based on the isolation of the bacterial strain referred to herein as “DTU01”, which produces ethanol as the main fermentation product, followed by acetate and lactate. The isolated organism is an extremely interesting and very promising organism for the establishment of a sustainable bioethanol production process. The invention further relates to a method for producing a fermentation product such as ethanol.