序号 专利名 申请号 申请日 公开(公告)号 公开(公告)日 发明人
201 TARGETED TREATMENT OF LEBER CONGENITAL AMOUROSIS US15642165 2017-07-05 US20180010117A1 2018-01-11 David Paschon
Disclosed herein are methods and compositions for inactivating mutant genes associated with LCA, using engineered nucleases comprising a DNA binding domain and a cleavage domain or cleavage half-domain in conditions promoting the cleavage of the mutant genes. Polynucleotides encoding nucleases, vectors comprising polynucleotides encoding nucleases, and cells comprising polynucleotides encoding nucleases and/or cells comprising nucleases are also provided.
202 Engineered CRISPR-Cas9 nucleases with Altered PAM Specificity US15060448 2016-03-03 US20180002681A9 2018-01-04 J. Keith Joung; Benjamin Kleinstiver
Engineered CRISPR-Cas9 nucleases with altered and improved PAM specificities and their use in genomic engineering, epigenomic engineering, and genome targeting.
203 Chimeric proteins and methods of regulating gene expression US15403058 2017-01-10 US09856497B2 2018-01-02 Lei S. Qi; P. C. Dave P. Dingal
The present disclosure provides systems, compositions and methods for regulating expression of a target polynucleotide in a cell. The systems, compositions and methods comprise a chimeric receptor polypeptide comprising a G-protein coupled receptor (GPCR) or a fragment thereof, a chimeric adaptor polypeptide, at least one actuator moiety and a cleavage moiety.
204 Methods for engineering T cells for immunotherapy by using RNA-guided CAS nuclease system US15045368 2016-02-17 US09855297B2 2018-01-02 Philippe Duchateau; André Choulika; Laurent Poirot
The present invention relates to methods of developing genetically engineered, preferably non-alloreactive T-cells for immunotherapy. This method involves the use of RNA-guided endonucleases, in particular Cas9/CRISPR system, to specifically target a selection of key genes in T-cells. The engineered T-cells are also intended to express chimeric antigen receptors (CAR) to redirect their immune activity towards malignant or infected cells. The invention opens the way to standard and affordable adoptive immunotherapy strategies using T-Cells for treating cancer and viral infections.
205 DNA cleavage enzyme US14890009 2014-05-09 US09840700B2 2017-12-12 Yoshizumi Ishino; Sonoko Ishino; Miyako Shiraishi
There is provided an enzyme which has an activity of cleaving a phosphodiester bond of deoxyribonucleotide having a damaged base and deoxyribonucleotide adjacent to the 5′ side of the deoxyribonucleotide in DNA strands which contain the damaged base as a reagent or the like for manipulating a gene, and further provided a method of removing a damaged base from DNA strands using the enzyme.
206 Methods for nucleic acid editing US14326109 2014-07-08 US09840699B2 2017-12-12 David R. Liu; Alexis Christine Komor
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 single site within the genome of a cell or subject, e.g., within the human genome. In some embodiments, fusion proteins of Cas9 and nucleic acid editing enzymes or enzyme domains, e.g., deaminase domains, are provided. In some embodiments, methods for targeted nucleic acid editing are provided. In some embodiments, reagents and kits for the generation of targeted nucleic acid editing proteins, e.g., fusion proteins of Cas9 and nucleic acid editing enzymes or domains, are provided.
207 Liquid Detergent Composition Comprising An Encapsulated Enzyme US15484440 2017-04-11 US20170335244A1 2017-11-23 Neil Joseph Lant; Steven George Patterson; Nazarmohammad Gulamhussain Momin; Jordan Courtney Toye
Liquid detergent composition that includes encapsulated enzymes. Related uses and methods.
208 Engineered CRISPR-Cas9 nucleases with Altered PAM Specificity US15664873 2017-07-31 US20170327806A1 2017-11-16 J. Keith Joung; Benjamin Kleinstiver
Engineered CRISPR-Cas9 nucleases with altered and improved PAM specificities and their use in genomic engineering, epigenomic engineering, and genome targeting.
209 Methods, compositions, and devices for supplying dietary fatty acid needs US15587867 2017-05-05 US09775784B2 2017-10-03 Alexey L. Margolin; Robert Gallotto; Bhami Shenoy
Nutritional formulas comprising long-chain polyunsaturated fatty acids (LC-PUFAs) are provided, along with methods and devices for preparing and/or administering nutritional formulas. In some embodiments, a percentage of the LC-PUFAs in the nutritional formula are in the form of monoglycerides and/or free fatty acids. In some embodiments, the nutritional formulas do not comprise added lipase. Also provided are methods for providing nutrition to a subject, methods for improving fat absorption, methods for improving cognitive ability, methods for preventing chronic lung disease, and methods for reducing the length of time a patient requires total parenteral nutrition.
210 Method for treating systemic DNA mutation disease US15157910 2016-05-18 US09770492B2 2017-09-26 Dmitry Dmitrievich Genkin; Georgy Viktorovich Tets; Viktor Veniaminovich Tets
The invention is directed to treatment of systemic DNA mutation diseases accompanied with development of somatic mosaicism and elevation of blood extracellular DNA. The inventive method comprises introducing a DNASE enzyme into the systemic blood circulation of a patient in doses and regimens which are sufficient to decrease average molecular weight of circulating extracellular blood DNA in the blood of said patient.
211 METHODS, COMPOSITIONS, AND DEVICES FOR SUPPLYING DIETARY FATTY ACID NEEDS US15587867 2017-05-05 US20170246089A1 2017-08-31 Alexey L. MARGOLIN; Robert GALLOTTO; Bhami Shenoy
Nutritional formulas comprising long-chain polyunsaturated fatty acids (LC-PUFAs) are provided, along with methods and devices for preparing and/or administering nutritional formulas. In some embodiments, a percentage of the LC-PUFAs in the nutritional formula are in the form of monoglycerides and/or free fatty acids. In some embodiments, the nutritional formulas do not comprise added lipase. Also provided are methods for providing nutrition to a subject, methods for improving fat absorption, methods for improving cognitive ability, methods for preventing chronic lung disease, and methods for reducing the length of time a patient requires total parenteral nutrition.
212 CONTROLLED-RELEASE AND STRATIFIED CYCLODEXTRIN INCLUSION COMPLEX VEHICLES US15285264 2016-10-04 US20170224842A1 2017-08-10 Al Czap
The invention provides cyclodextrin inclusion complex delivery vehicles, in which the cyclodextrin inclusion complex is provided together with enzyme having a cyclodextrin-degrading activity capable of digesting the cyclodextrin, so that upon delivery of the vehicle to a target the enzyme is activated and releases the guest molecule from the cyclodextrin cavity. In alternative aspects, these cyclodextrin inclusion complex delivery vehicles are for example provided in the form of medicaments, food ingredients, medical food ingredients, nutritional supplement ingredients, dietary supplement ingredients, herbicides, insecticides, fungicides, animal repellents, pheromones, plant growth regulators, fragrances, fabrics or packaging materials.
213 Detergents and Compositions with Enzymatic Polymer Particles US15500738 2015-07-07 US20170218315A1 2017-08-03 Tue Rasmussen; Katarina Jacobson; Kim Bruno Andersen; Martin Noerby; Morten Foverskov; Ole Simonsen; Robert Neil Hay; David John Duncalf; David Brian Young; David Alan Pears
The present invention relates to an enzyme composition comprising enzyme containing polymer particles, which is useful for liquid laundry detergent compositions. In these enzyme containing particles, the particles comprise at least one enzyme, and at least one polymer, which is a hydrophobic modified polyvinyl alcohol.
214 Methods, compositions, and devices for supplying dietary fatty acid needs US15240596 2016-08-18 US09687420B2 2017-06-27 Alexey L. Margolin; Robert Gallotto; Bhami Shenoy
Nutritional formulas comprising long-chain polyunsaturated fatty acids (LC-PUFAs) are provided, along with methods and devices for preparing and/or administering nutritional formulas. In some embodiments, a percentage of the LC-PUFAs in the nutritional formula are in the form of monoglycerides and/or free fatty acids. In some embodiments, the nutritional formulas do not comprise added lipase. Also provided are methods for providing nutrition to a subject, methods for improving fat absorption, methods for improving cognitive ability, methods for preventing chronic lung disease, and methods for reducing the length of time a patient requires total parenteral nutrition.
215 METHODS FOR USING A 5'-EXONUCLEASE TO INCREASE HOMOLOGOUS RECOMBINATION IN EUKARYOTIC CELLS US15378609 2016-12-14 US20170175140A1 2017-06-22 Aaron W. Hummel; Javier Gil Humanes; Daniel F. Voytas
Provided herein are materials and methods for gene editing in eukaryotic cells (e.g., plant cells) by homologous recombination, including materials and methods for boosting the frequency of homologous recombination through the application of a 5′-exonuclease for end-processing of DNA double-strand breaks.
216 Novel Recombinant Adeno-Associated Virus Capsids with Enhanced Human Skeletal Muscle Tropism US15367734 2016-12-02 US20170159026A1 2017-06-08 Mark A. KAY; Nicole K. PAULK
The present invention relates to variant AAV capsid polypeptides, wherein the variant AAV capsid polypeptides exhibit increased transduction and/or tropism in human muscle tissue or cells as compared non-variant parent capsid polypeptides.
217 Methods, compositions, and devices for supplying dietary fatty acid needs US14378856 2013-02-14 US09668942B2 2017-06-06 Alexey L. Margolin; Robert Gallotto; Bhami Shenoy
Nutritional formulas comprising long-chain polyunsaturated fatty acids (LC-PUFAs) are provided, along with methods and devices for preparing and/or administering nutritional formulas. In some embodiments, a percentage of the LC-PUFAs in the nutritional formula are in the form of monoglycerides and/or free fatty acids. In some embodiments, the nutritional formulas do not comprise added lipase. Also provided are methods for providing nutrition to a subject, methods for improving fat absorption, methods for improving cognitive ability, methods for preventing chronic lung disease, and methods for reducing the length of time a patient requires total parenteral nutrition.
218 NUCLEIC ACID ENRICHMENT USING CAS9 US15335139 2016-10-26 US20170107560A1 2017-04-20 Brian Jon Peter; Robert A. Ach
A method of enriching for a fragment of a genome, as well as corresponding compositions and kits, are provided. In certain embodiments, the method comprises: (a) contacting a sample comprising fragmented DNA with a Cas9-gRNA complex comprising mutant Cas9 protein that has inactivated nuclease activity and a Cas9-associated guide RNA that is complementary to a site in the DNA, to produce a Cas9-fragment complex that comprises a fragment of the fragmented DNA; and (b) isolating the complex. In addition, other methods and compositions for Cas9/CRISPR-mediated nucleic acid manipulation are also provided.
219 Substituted-6,8-dioxabicyclo[3.2.1]octane-2,3-diol compounds as targeting agents of ASGPR US15093178 2016-04-07 US09617293B2 2017-04-11 Spiros Liras; Vincent Mascitti; Benjamin Thuma
Compounds of Formula (A) are described herein and the uses thereof for the treatment of diseases, conditions and/or disorders mediated by pharmaceutical compositions and the uses thereof as asialoglycoprotein receptor (ASGPR) targeting agents.
220 DELIVERY METHODS AND COMPOSITIONS US15277595 2016-09-27 US20170087224A1 2017-03-30 Stephen R. Quake
The invention provides methods and compositions that remove target genetic material from a subject by delivery of an enzyme that degrades the target genetic material. The methods include delivering a composition of a nucleic acid to a tissue, such as skin, of a subject along with various types of energy to enhance permeability of the tissue and cause the nucleic acid to enter cells of the tissue, wherein the nucleic acid comprises a gene for an enzyme that cuts target genetic material. The nucleic acid may be a plasmid comprising a cas9 gene and at least one gene for a short guide RNA (sgRNA) and the target genetic material may be viral genome, i.e., with the sgRNA complementary to a portion of the viral genome.
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