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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
221	MODIFYING MESSENGER RNA STABILITY IN PLANT TRANSFORMATIONS	US15056544	2016-02-29	US20170081672A1	2017-03-23	Thomas Stoddard; Song Luo
Materials and methods for genome engineering through transient expression of a targeted nuclease are described herein. For example, the methods described herein can include introducing into a cell a messenger RNA (mRNA) that encodes a nuclease targeted to a selected sequence within the cell, where the stability of the mRNA is modified by the addition of untranslated regions (UTRs).
222	CRISPR/CAS-RELATED METHODS AND COMPOSITIONS FOR TREATING USHER SYNDROME AND RETINITIS PIGMENTOSA	US15123576	2015-03-05	US20170073674A1	2017-03-16	Morgan L. MAEDER; David A. BUMCROT
CRISPR/Cas-related compositions and methods for treatment of Usher Syndrome and/or Retinitis Pigmentosa are disclosed herein.
223	Orthogonal Cas9 proteins for RNA-guided gene regulation and editing	US14674895	2015-03-31	US09587252B2	2017-03-07	George M. Church; Kevin Esvelt; Prashant Mali
Methods of modulating expression of a target nucleic acid in a cell are provided including use of multiple orthogonal Cas9 proteins to simultaneously and independently regulate corresponding genes or simultaneously and independently edit corresponding genes.
224	FAD2 PERFORMANCE LOCI AND CORRESPONDING TARGET SITE SPECIFIC BINDING PROTEINS CAPABLE OF INDUCING TARGETED BREAKS	US15292774	2016-10-13	US20170044559A1	2017-02-16	W. Michael Ainley; Steven R. Webb; Jayakumar P. Samuel; Dmitry Y. Guschin; Jeffrey C. Miller; Lei Zhang
Methods and compositions for gene disruption, gene editing or gene stacking within a FAD2 loci by cleaving, in a site directed manner, a location in a FAD2 gene in a soybean cell, to generate a break in the FAD2 gene and then optionally integrating into the break a nucleic acid molecule of interest is disclosed.
225	Stitched polypeptides	US14027064	2013-09-13	US09556227B2	2017-01-31	Gregory L. Verdine; Young-Woo Kim
The present invention provides inventive stitched polypeptides, pharmaceutical compositions thereof, and methods of making and using inventive stitched polypeptides.
226	METHODS AND COMPOSITIONS FOR TREATMENT OF NEURODEGENERATIVE DISEASES	US15125542	2015-03-11	US20170000729A1	2017-01-05	Martin L. Katz; Joan R. Coates; Christopher J. Tracy; Rebecca E.H. Whiting; Jacqueline W. Pearce
The present invention provides methods and compositions for treating a neurodegeneration disease or retinal degenerative disease in a mammal comprising the use of mesenchymal stem cells expressing a therapeutic compound. The invention also provides cells and constructs for use in such methods. Also provided are kits for treatment of a neurodegeneration disease or retinal degenerative disease. The present invention relates in general to the field of retinal degenerative and neurodegenerative diseases. More specifically, the invention relates to methods for treatment of retinal degenerative and neurodegenerative disease.
227	Targeted modification of malate dehydrogenase	US13875992	2013-05-02	US09523098B2	2016-12-20	Vipula Shukla; Manju Gupta; Fyodor Urnov; Dmitry Guschin; Michiel Jan De Both; Paul Bundock; Lakshmi Sastry-Dent
Disclosed herein are methods and compositions for targeted modification of one or more endogenous plant malate dehydrogenase genes.
228	CRISPR HYBRID DNA/RNA POLYNUCLEOTIDES AND METHODS OF USE	US15238464	2016-08-16	US20160362668A1	2016-12-15	Andrew Paul May; Paul Daniel Donohoue
The present disclosure provides DNA-guided CRISPR systems; polynucleotides comprising DNA, RNA and mixtures thereof for use with CRISPR systems; and methods of use involving such polynucleotides and DNA-guided CRISPR systems.
229	ENGINEERING OF SYSTEMS, METHODS AND OPTIMIZED GUIDE COMPOSITIONS WITH NEW ARCHITECTURES FOR SEQUENCE MANIPULATION	US15172636	2016-06-03	US20160355795A1	2016-12-08	Fei Ran; Feng Zhang
The invention provides for systems, methods, and compositions for altering expression of target gene sequences and related gene products. Provided are structural information on the Cas protein of the CRISPR-Cas system, use of this information in generating modified components of the CRISPR complex, vectors and vector systems which encode one or more components or modified components of a CRISPR complex, as well as methods for the design and use of such vectors and components. Also provided are methods of directing CRISPR complex formation in eukaryotic cells and methods for utilizing the CRISPR-Cas system. In particular the present invention comprehends optimized functional CRISPR-Cas enzyme systems. In particular the present invention comprehends engineered new guide architectures to be used in optimized CRISPR-Cas enzyme systems.
230	COMPOSITIONS AND METHODS TO TREAT HERPES SIMPLEX VIRUS INFECTIONS	US15167204	2016-05-27	US20160346361A1	2016-12-01	Stephen R. Quake; Jianbin Wang
Herpes simplex virus (HSV) including herpes simplex virus 1 and 2 (HSV-1 and HSV-2) are a persistent cause of human disease with no known cure. Guided nuclease systems target specific regions of the HSV-1 and HSV-2 genomes, disrupting the virus' nucleic acid and rendering even latent viruses incapacitated.
231	ENGINEERING OF SYSTEMS, METHODS AND OPTIMIZED GUIDE COMPOSITIONS FOR SEQUENCE MANIPULATION	US15230025	2016-08-05	US20160340662A1	2016-11-24	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.
232	DNA VECTOR PRODUCTION SYSTEM	US15047029	2016-02-18	US20160333361A1	2016-11-17	Roderick Slavcev; Nafiseh Nafissi
A vector production system is provided. The system comprises recombinant cells designed to encode at least a first recombinase under the control of an inducible promoter and the cells include an expression vector encoding a nucleic acid of interest within the regulatory elements of the expression vector which are flanked on either side by a target sequence for at least the first recombinase. The vector production system provides an efficient one-step process for producing linear or circular covalently closed vectors that incorporate a nucleic acid sequence of interest.
233	FAD2 performance loci and corresponding target site specific binding proteins capable of inducing targeted breaks	US14019293	2013-09-05	US09493779B2	2016-11-15	William Michael Ainley; Steven R. Webb; Pon Samuel; Dmitry Y. Guschin; Jeffrey C. Miller; Lei Zhang
Methods and compositions for gene disruption, gene editing or gene stacking within a FAD2 loci by cleaving, in a site directed manner, a location in a FAD2 gene in a soybean cell, to generate a break in the FAD2 gene and then optionally integrating into the break a nucleic acid molecule of interest is disclosed.
234	TEMPLATE SWITCH-BASED METHODS FOR PRODUCING A PRODUCT NUCLEIC ACID	US15194414	2016-06-27	US20160304946A1	2016-10-20	Craig Betts; Andrew Alan Farmer
Provided are methods of producing a product nucleic acid. The methods include combining a template deoxyribonucleic acid (DNA), a polymerase, a template switch oligonucleotide, and dNTPs into a reaction mixture. The components are combined into the reaction mixture under conditions sufficient to produce a product nucleic acid that includes the template DNA and the template switch oligonucleotide each hybridized to adjacent regions of a single product nucleic acid that includes a region polymerized from the dNTPs by the polymerase. Aspects of the invention further include compositions and kits.
235	CAS VARIANTS FOR GENE EDITING	US15103608	2014-12-12	US20160304846A1	2016-10-20	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.
236	METHODS AND COMPOSITIONS FOR TREATING HUNTINGTON'S DISEASE	US15036020	2014-11-11	US20160296605A1	2016-10-13	H. Steve Zhang
Disclosed herein are methods and compositions for diagnosing, treating or preventing Huntington's Disease.
237	Method for monitoring development of somatic mosaicism	US14309363	2014-06-19	US09463223B2	2016-10-11	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.
238	OPTIMIZED SMALL GUIDE RNAS AND METHODS OF USE	US15025217	2014-09-29	US20160289673A1	2016-10-06	Bo HUANG; Baohui CHEN; Lei QI
Methods, compositions, and kits are provided herein for CRISPER/Cas-mediated nucleic acid detection or modification.
239	Stable Enzymes by Glycation Reduction	US15035666	2014-11-14	US20160289653A1	2016-10-06	Mark S. Gebert; Soren Dalsgaard; Mariliz Ortiz Johnson; Adam L. Garske; Douglas A. Dale
The present teachings provide enzymes with improved stability arising from reduced glycation. Glycation reduction can be achieved by a variety of means, including protein engineering, heating to remove background ground starch hydrolytic activity, diafiltration afiltration, and various formulation approaches including non-inclusion of problematic sugars, and inclusion of resistant starches.
240	METHOD FOR TARGETED MODIFICATION OF ALGAE GENOMES	US14442323	2013-11-18	US20160272980A1	2016-09-22	Philippe DUCHATEAU; Fayza DABOUSSI
The invention relates to a method for modifying genetic material in algal cells that includes the use of rare-cutting endonuclease to target specific genomic sequences. In particular, the invention relates to a method for modifying genetic material in algal cells wherein rare-cutting endonuclease, especially a homing endonuclease or a TALE-Nuclease, is expressed over several generations to efficiently modify said target genome sequences.

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