公开(公告)号：US12077756B2

公开(公告)日：2024-09-03

申请号：US17836458

申请日：2022-06-09

申请人： Dicerna Pharmaceuticals, Inc.

发明人： Bob Dale Brown

IPC分类号： C07H21/02 ,  A61K31/713 ,  C12N15/11 ,  C12N15/113

CPC分类号： C12N15/113 ,  A61K31/713 ,  C12N15/111 ,  C12N15/1135 ,  C12N15/1137 ,  C12N2310/11 ,  C12N2310/14 ,  C12N2310/51 ,  C12N2310/533 ,  C12N2320/53

摘要： The invention provides compositions and methods for reducing expression of a target gene in a cell, involving contacting a cell with an isolated double stranded nucleic acid (dsNA) in an amount effective to reduce expression of a target gene in a cell. The dsNAs of the invention possess a single stranded extension (in most embodiments, the single stranded extension comprises at least one modified nucleotide and/or phosphate back bone modification). Such single stranded extended Dicer-substrate siRNAs (DsiRNAs) were demonstrated to be effective RNA inhibitory agents compared to corresponding double stranded DsiRNAs.

公开(公告)号：US20240150766A1

公开(公告)日：2024-05-09

申请号：US18509130

申请日：2023-11-14

申请人： KIP Co. Ltd.

发明人： Tae Gwan Park ,  Hye Jung Mok ,  Soo Hyeon Lee

IPC分类号： C12N15/113 ,  C12N15/11

CPC分类号： C12N15/1136 ,  C12N15/111 ,  C12N15/113 ,  C12N2310/14 ,  C12N2310/51

摘要： The present invention relates to a multi-conjugate of small interfering RNA (siRNA) and a preparing method of the same, more precisely a multi-conjugate of siRNA prepared by direct binding of double stranded sense/antisense siRNA monomers or indirect covalent bonding mediated by a cross-linking agent or a polymer, and a preparing method of the same. The preparing method of a siRNA multi-conjugate of the present invention is characterized by simple and efficient reaction and thereby the prepared siRNA multi-conjugate of the present invention has high molecular weight multiple times the conventional siRNA, so that it has high negative charge density, suggesting that it has excellent ionic interaction with a cationic gene carrier and high gene delivery efficiency.

公开(公告)号：US11756183B2

公开(公告)日：2023-09-12

申请号：US16625562

申请日：2018-06-23

申请人： CORNELL UNIVERSITY

发明人： Samie R. Jaffrey ,  Jacob L. Litke

IPC分类号： A61K31/7115 ,  G06T7/00 ,  G06T7/11 ,  C12N15/115 ,  C12N15/63 ,  G01N21/88 ,  G01N21/95 ,  G01N21/956

CPC分类号： G06T7/001 ,  A61K31/7115 ,  C12N15/115 ,  C12N15/63 ,  G01N21/8851 ,  G01N21/9501 ,  G01N21/95607 ,  G06T7/11 ,  C12N2310/128 ,  C12N2310/16 ,  C12N2310/3519 ,  C12N2310/51 ,  C12N2310/532 ,  G01N2021/95615 ,  G06T2207/20104 ,  G06T2207/30148

摘要： The present invention relates to a RNA molecule comprising a first ribozyme, a first ligation sequence, an effector molecule, a second ligation sequence, and a second ribozyme. Methods of producing circular RNA molecules and treatment methods are also disclosed.

公开(公告)号：US11702667B2

公开(公告)日：2023-07-18

申请号：US16386903

申请日：2019-04-17

申请人： The Penn State Research Foundation

发明人： Yinong Yang ,  Kabin Xie

IPC分类号： C12N15/11 ,  C12N15/113 ,  C12N15/85 ,  C12N15/82

CPC分类号： C12N15/8213 ,  C12N15/111 ,  C12N15/113 ,  C12N15/8216 ,  C12N15/8218 ,  C12N15/85 ,  C12N2310/10 ,  C12N2310/20 ,  C12N2310/51 ,  C12N2330/31 ,  C12N2330/51

摘要： The invention includes materials and methods to generate numerous small RNAs from one polynucleotide construct (synthetic gene) to facilitate RNA-guided multiplex genome editing, modification, inhibition of expression and other RNA-based technologies. The synthetic gene/polynucleotide construct encodes polycistronic RNA components separated by tRNAs, and preferably also includes regulatory components such as a promoter or terminator to form an expression cassette. Once transcribed in a cell, the transcript is processed by the cell to multiple RNA molecules by the endogenous tRNA processing system. The system can be sued for any RNA based gene manipulation method including RNA-mediated genome editing, artificial microRNA mediated gene silencing, small RNA mediated genetic manipulation, double-stranded RNA mediated gene silencing, antisense mechanisms and the like.

公开(公告)号：US20230193285A1

公开(公告)日：2023-06-22

申请号：US17926067

申请日：2021-05-19

申请人： Aummune Ltd.

发明人： Guy NEEV ,  Irit Carmi-Levy ,  Yaniv Amir ,  Erez Lavi ,  Nir Skalka ,  Neta Zilony-Hanin ,  Zohar Pode

IPC分类号： C12N15/115 ,  A61P35/00 ,  A61K45/06 ,  A61K31/7088

CPC分类号： C12N15/115 ,  A61K31/7088 ,  A61K45/06 ,  A61P35/00 ,  C12N2310/16 ,  C12N2310/17 ,  C12N2310/51 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/335 ,  C12N2310/351 ,  C12N2310/3231 ,  C12N2310/3515 ,  C12N2310/3519 ,  C12N2330/31

摘要： Provided herein are bispecific personalized aptamers that induce the cell death of cancer cells and methods of use thereof.

公开(公告)号：US20230159935A1

公开(公告)日：2023-05-25

申请号：US17797217

申请日：2021-03-01

申请人： UNIVERSITY OF FLORIDA RESEARCH FOUNDATION, INCORPORATED ,  GRYPHON BIO THERAPEUTICS, INC.

发明人： Kevin K-Wang WANG ,  Xiaowei LI ,  Weihong TAN ,  Bang WANG ,  William HASKINS

IPC分类号： C12N15/115

CPC分类号： C12N15/115 ,  C12N2310/16 ,  C12N2310/3513 ,  C12N2310/3517 ,  C12N2310/3519 ,  C12N2310/51 ,  C12N2310/532

摘要： The lack of blood-brain barrier (BBB) penetrating ability has hindered the delivery of many therapeutic agents for tauopathy therapeutic treatment. A circular bifunctional aptamer reported here has been able to enhance the in vivo BBB penetration for improved therapy. The circular aptamer includes one transferrin receptor (TfR) aptamer to facilitate TfR-aptamer recognition-induced transcytosis across BBB endothelial cells, and one Tau protein aptamer selected to inhibit Tau phosphorylation and other tauopathy-related pathological events in the brain. This bispecific construct exhibits strong specificity towards Tau and enhanced plasma stability in comparison to linear Tau aptamer. In vivo administration of circular Tau-TfR aptamer results in a rapid uptake into relevant brain regions after crossing the BBB, such as hippocampus and cortex. A Y-shaped trispecific aptamer including one aptamer for L1CAM, one aptamer for Tau and one aptamer for TfR reported here has enhanced BBB and neuron cell membrane permeation. Bispecific and trispecific Tau aptamer coupled to a signaling moiety (such as dodecane tetraacetic acid (DOTA) or DOTA complexed to Gd+3) for neuroimaging, and bispecific or trispecific Tau aptamer coupled to protein aggregate binding moiety (such as methylene blue) for enhanced ability to disrupt tau aggregation are also contemplated in this invention.

公开(公告)号：US20180171334A1

公开(公告)日：2018-06-21

申请号：US15725843

申请日：2017-10-05

申请人： Regulus Therapeutics Inc.

发明人： Balkrishen Bhat ,  Neil W. Gibson ,  Diedre MacKenna ,  Brandee Wagner ,  David P. Bartel

IPC分类号： C12N15/113 ,  A61K31/7088 ,  A61K31/713 ,  A61K45/06 ,  A61K47/54

CPC分类号： C12N15/113 ,  A61K31/7088 ,  A61K31/713 ,  A61K45/06 ,  A61K47/549 ,  C12N2310/113 ,  C12N2310/141 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/3231 ,  C12N2310/345 ,  C12N2310/346 ,  C12N2310/351 ,  C12N2310/51 ,  C12N2320/30

摘要： Described herein are compounds comprising modified oligonucleotides that are complementary to miR-103 and/or miR-107 and methods of treating diseases and disorders using the compounds.

公开(公告)号：US20180119120A1

公开(公告)日：2018-05-03

申请号：US15787705

申请日：2017-10-18

申请人： Caribou Biosciences, Inc.

发明人： Paul Daniel Donohoue ,  Andrew Paul May

IPC分类号： C12N9/22 ,  C12N15/113 ,  C12N15/90

CPC分类号： C12N9/22 ,  C12N9/96 ,  C12N15/102 ,  C12N15/113 ,  C12N15/902 ,  C12N15/907 ,  C12N2310/20 ,  C12N2310/3519 ,  C12N2310/51

摘要： The present disclosure provides engineered cross-type-nucleic-acid targeting nucleic acids and compositions thereof. Nucleic acid sequences encoding the engineered cross-type-nucleic-acid targeting nucleic acids, as well as expression cassettes, vectors and cells comprising such nucleic acid sequences, are described. Also, methods are disclosed for making and using the engineered cross-type-nucleic-acid targeting nucleic acids and compositions thereof.

公开(公告)号：US09957490B1

公开(公告)日：2018-05-01

申请号：US15787705

申请日：2017-10-18

申请人： Caribou Biosciences, Inc.

发明人： Paul Daniel Donohoue ,  Andrew Paul May

IPC分类号： C12N15/113 ,  C12N15/90 ,  C12N9/22

CPC分类号： C12N9/22 ,  C12N9/96 ,  C12N15/102 ,  C12N15/113 ,  C12N15/902 ,  C12N15/907 ,  C12N2310/20 ,  C12N2310/3519 ,  C12N2310/51

摘要： The present disclosure provides engineered cross-type-nucleic-acid targeting nucleic acids and compositions thereof. Nucleic acid sequences encoding the engineered cross-type-nucleic-acid targeting nucleic acids, as well as expression cassettes, vectors and cells comprising such nucleic acid sequences, are described. Also, methods are disclosed for making and using the engineered cross-type-nucleic-acid targeting nucleic acids and compositions thereof.

公开(公告)号：US09932590B2

公开(公告)日：2018-04-03

申请号：US14902505

申请日：2014-07-02

申请人： THE UNIVERSITY COURT OF THE UNIVERSITY OF ABERDEEN ,  THE SECRETARY OF STATE FOR ENVIRONMENT, FOOD AND RURAL AFFAIRS

发明人： Alan Stuart Bowman ,  Ewan McInnes Campbell ,  Giles Elliott Budge

IPC分类号： C12N15/113 ,  A01N57/10 ,  A23K20/153 ,  A23K50/90 ,  A01N57/16

CPC分类号： C12N15/1137 ,  A01N57/16 ,  A23K20/153 ,  A23K50/90 ,  C12N15/113 ,  C12N15/1136 ,  C12N15/1138 ,  C12N2310/11 ,  C12N2310/14 ,  C12N2310/51 ,  C12N2320/30 ,  C12N2320/31 ,  C12Y104/03004 ,  C12Y204/01016 ,  C12Y205/01018 ,  C12Y207/0104 ,  C12Y301/01007 ,  C12Y306/01003 ,  C12Y306/03014

摘要： Nucleic acid agents for reducing or removing infestations of the Varroa destructor mite are described. Compositions comprising the nucleic acid agents and methods for controlling mite infestations using the nucleic acid agents and compositions are also disclosed.