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序号	专利名	申请号	申请日	公开（公告）号	公开（公告）日	发明人
261	ENGINEERING OF SYSTEMS, METHODS AND OPTIMIZED GUIDE COMPOSITIONS FOR SEQUENCE MANIPULATION	US14704551	2015-05-05	US20150247150A1	2015-09-03	Feng Zhang; Le Cong; Patrick Hsu; Fei Ran
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 or more components of a CRISPR complex, as well as methods for the design and use of such vectors. Also provided are methods of directing CRISPR complex formation in eukaryotic cells and methods for selecting specific cells by introducing precise mutations utilizing the CRISPR-Cas system.
262	COMPOSITIONS AND METHODS COMPRISING SEQUENCES HAVING MEGANUCLEASE ACTIVITY	US14398592	2013-05-01	US20150184173A1	2015-07-02	Ericka Bermudez; Andrew Mark Cigan; James English; Saverio Carl Falco; Huirong Gao; Lu Liu; Zhan-bin Liu; Azalea Ong; Sergei Svitashev; Joshua K. Young
Compositions and methods comprising polynucleotides and polypeptides having meganuclease activity are provided. Further provided are nucleic acid constructs, yeast, plants, plant cells, explants, seeds and grain having the meganuclease sequences. Various methods of employing the meganuclease sequences are provided. Such methods include, for example, methods for producing a meganuclease with increased activity at a wide range of temperatures, methods for producing a yeast, plant, plant cell, explant or seed comprising a meganuclease with increased activity.
263	METHODS FOR CORRECTING VON WILLEBRAND FACTOR POINT MUTATIONS	US14326318	2014-07-08	US20150166985A1	2015-06-18	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 nucleic acid encoding a mutant von Willebrand Factor protein to correct a point mutation associated with a disease or disorder, e.g., with von Willebrand disease. The methods provided are useful for correcting a vWF point mutation 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, 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.
264	METHODS FOR CORRECTING PRESENILIN POINT MUTATIONS	US14326269	2014-07-08	US20150166983A1	2015-06-18	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 nucleic acid encoding a mutant Presenilin1 protein to correct a point mutation associated with a disease or disorder, e.g., with familial Alzheimer's disease. The methods provided are useful for correcting a PSEN1 point mutation 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, 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.
265	FUSIONS OF CAS9 DOMAINS AND NUCLEIC ACID-EDITING DOMAINS	US14325815	2014-07-08	US20150166980A1	2015-06-18	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.
266	BIOCONTROL	US14383515	2013-03-05	US20150143552A1	2015-05-21	Luke Alphey
Provided is an arthropod male germline gene expression system suitable for conditional expression of an effector gene in an Arthropod male germline. The system comprises a first expression unit comprising an effector gene and a promoter therefor operably linked thereto; and a second expression unit. Said second unit comprises a coding sequence for a transcription factor and an upstream regulatory element operably linked thereto, the transcription factor being capable of acting upon the promoter in the first expression unit to drive expression of the effector gene. The upstream regulatory element includes a promoter for the transcription factor; and a 5′ UTR adjacent a start site for the transcription factor coding sequence. The upstream regulatory element driving sufficient expression of the transcription factor such that the transcription factor protein in turn drives transcription of the effector gene before meiosis. Also provided are uses of the system for instance in methods of biocontrol and quality control.
267	RNA-guided human genome engineering	US14319100	2014-06-30	US09023649B2	2015-05-05	Prashant G. Mali; George M. Church; Luhan Yang
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.
268	Method of treating toxemia	US14037696	2013-09-26	US09023344B2	2015-05-05	Joan M. Fallon
A therapeutic agent for the treatment of toxemia, preeclampsia and eclampsia and a method for preparing the therapeutic agent are disclosed. The therapeutic agent is a stable pharmaceutical preparation containing, but not limited to, digestive/pancreatic enzymes. The therapeutic agent may be manufactured by a variety of encapsulation technologies. Delivery of the therapeutic agent may be made orally, through injection, by adherence of a medicated patch or by other methods. Further, a method of using the presence of chymotrypsin in the maternal GI tract as a biomarker, to determine the likelihood of developing preeclampsia, a pregnancy induced hypertension, and eclampsia/toxemia is disclosed.
269	METHOD FOR TREATING SYSTEMIC DNA MUTATION DISEASE	US14309363	2014-06-19	US20150010523A1	2015-01-08	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.
270	Engineering of systems, methods and optimized guide compositions for sequence manipulation	US14290575	2014-05-29	US08906616B2	2014-12-09	Feng Zhang; Le Cong; Patrick Hsu; Fei Ran
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 or more components of a CRISPR complex, as well as methods for the design and use of such vectors. Also provided are methods of directing CRISPR complex formation in eukaryotic cells and methods for selecting specific cells by introducing precise mutations utilizing the CRISPR-Cas system.
271	RNA-Guided Human Genome Engineering	US14319255	2014-06-30	US20140342458A1	2014-11-20	Prashant G. MALI; George M. CHURCH; Luhan Yang
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.
272	Using Truncated Guide RNAs (tru-gRNAs) to Increase Specificity for RNA-Guided Genome Editing	US14213723	2014-03-14	US20140295557A1	2014-10-02	J. Keith Joung; Jeffry D. Sander; Morgan Maeder; Yanfang Fu
Methods for increasing specificity of RNA-guided genome editing, e.g., editing using CRISPR/Cas9 systems, using truncated guide RNAs (tru-gRNAs).
273	ENGINEERING OF SYSTEMS, METHODS AND OPTIMIZED GUIDE COMPOSITIONS FOR SEQUENCE MANIPULATION	US14290575	2014-05-29	US20140273232A1	2014-09-18	Feng Zhang; Le Cong; Patrick Hsu; Fei Ran
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 or more components of a CRISPR complex, as well as methods for the design and use of such vectors. Also provided are methods of directing CRISPR complex formation in eukaryotic cells and methods for selecting specific cells by introducing precise mutations utilizing the CRISPR-Cas system.
274	C1-C2 Organic Acid Treatment of Lignocellulosic Biomass to Produce Acylated Cellulose Pulp, Hemicellulose, Lignin and Sugars and Fermentation of the Sugars	US14342634	2012-09-21	US20140227742A1	2014-08-14	Wuli Bao; Thomas Binder; Charles Abbas; Lucas Loveless
A process for production of C5 and C6 sugar enriched syrups from lignocellulosic biomass and fermentation products therefrom is described. A lignocellulosic biomass is treated with a C1-C2 acid (e.g., acetic acid) with washing thereof with a C1-C2 acid miscible organic solvent, (e.g., ethyl acetate). A soluble hemicellulose and lignin enriched fraction is obtained separately from a cellulose pulp enriched fraction and lignin is removed from the soluble hemicellulose fraction. These fractions contain acylated (e.g., acetylated) cellulose and hemicellulose, which are deacylated by treatment with an alkali and/or with an acetyl esterase enzyme. The deacylated fractions are then digested with suitable cellulolytic and/or hemicellulolytic enzymes, preferably in the presence of non-ionic detergent to yield the C5 and C6 enriched syrups. Also described are method of fermentation of the syrups to make ethanol to at least 7% w/vol by separate hydrolysis and fermentation (SHF) or simultaneous hydrolysis and fermentation (SSF) methods.
275	COMPOSITIONS AND METHODS COMPRISING SEQUENCES HAVING MEGANUCLEASE ACTIVITY	US13886317	2013-05-03	US20140223606A1	2014-08-07	ERICKA BERMUDEZ; ANDREW MARK CIGAN; JAMES ENGLISH; SAVERIO CARL FALCO; HUIRONG GAO; LU LIU; ZHAN-BIN LIU; AZALEA ONG; SERGEI SVITASHEV; JOSHUA K. YOUNG
Compositions and methods comprising polynucleotides and polypeptides having meganuclease activity are provided. Further provided are nucleic acid constructs, yeast, plants, plant cells, explants, seeds and grain having the meganuclease sequences. Various methods of employing the meganuclease sequences are provided. Such methods include, for example, methods for producing a meganuclease with increased activity at a wide range of temperatures, methods for producing a yeast, plant, plant cell, explant or seed comprising a meganuclease with increased activity.
276	SOLID MEDIUM FOR THE STORAGE OF BIOLOGICAL MATERIAL	US13798483	2013-03-13	US20140212880A1	2014-07-31	MARTIN D. JAMES; JEFFREY K. HORTON; PETER J. TATNELL
This invention relates to flat solid media for the storage of samples of biological materials and methods of analysing biomolecules contained within the samples following storage. In particular, the invention relates to the storage and further analysis of biomolecules present in the biological materials, such as proteins, enzymes and nucleic acids. The invention finds particular utility in the dry, room temperature storage of biological materials.
277	Use of meganucleases for inducing homologous recombination ex vivo and in toto in vertebrate somatic tissues and application thereof	US13556206	2012-07-24	US08697395B2	2014-04-15	Sylvain Arnould; Patrick Chames; Phillippe Duchateau; Jean-Charles Epinat; Emmanuel Lacroix; Frederic Paques
Use of meganucleases for cleaving DNA in a non-human or an isolated human cell and, in some instances, inducing homologous recombination in said cells and to its application for genome engineering and gene therapy.
278	Zinc finger proteins for DNA binding and gene regulation in plants	US11511106	2006-08-28	US08680021B2	2014-03-25	Andrew Jamieson; Guofu Li
Disclosed herein are modified plant zinc finger proteins; compositions comprising modified plant zinc finger proteins and methods of making and using modified plant zinc finger proteins. The modified plant zinc finger proteins, in contrast to naturally-occurring plant zinc finger proteins, have a binding specificity that is determined by tandem arrays of modular zinc finger binding unit.
279	Compositions and use thereof for treating symptoms of preeclampsia	US12047818	2008-03-13	US08658163B2	2014-02-25	Joan M. Fallon
A therapeutic agent for the treatment of toxemia, preeclampsia and eclampsia and a method for preparing the therapeutic agent is disclosed. The therapeutic agent is a stable pharmaceutical preparation containing, but not limited to, digestive/pancreatic enzymes. The therapeutic agent may be manufactured by a variety of encapsulation technologies. Delivery of the therapeutic agent may be made orally, through injection, by adherence of a medicated patch or by other methods. Further, a method of using the presence of chymotrypsin in the maternal GI tract as a biomarker to determine the likelihood of developing preeclampsia, a pregnancy induced hypertension, and eclampsia/toxemia is disclosed.
280	Use of meganucleases for inducing homologous recombination ex vivo and in toto in vertebrate somatic tissues and application thereof	US13485469	2012-05-31	US08624000B2	2014-01-07	Sylvain Arnould; Patrick Chames; Phillippe Duchateau; Jean-Charles Epinat; Emmanuel Lacroix; Frederic Paques
A monomer of an I-CreI meganuclease variant wherein said monomer comprises mutations in the amino acid sequence of SEQ ID NO: 34, wherein said mutations include, (i) at least one and up to five amino acid substitutions from residue Q44 to residue R70, said substitutions selected from the group consisting of substitutions at positions Q44, T46, Y66, R68 and R7; and (ii) at least one and up to six amino acid substitutions from residue Q26 to residue Q38 said substitutions selected from the group consisting of substitutions at positions Q26, K28, N30, S32, Y33 and Q38, and wherein said monomer when in dimeric form binds and cleaves a DNA target sequence. Said dimeric forms include homodimeric, heterodimeric and single-chain I-CreI meganuclease variants.

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