公开(公告)号：US12060558B2

公开(公告)日：2024-08-13

申请号：US17052874

申请日：2019-05-03

Applicant: STOKE THERAPEUTICS, Inc.

Inventor： Isabel Aznarez

IPC: C07H21/02 ,  A61K38/46 ,  C12N15/113

CPC classification number: C12N15/1137 ,  A61K38/465 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/32 ,  C12N2320/11 ,  C12N2320/33 ,  C12Y301/01013

Abstract: Provided herein are methods and compositions for treating a subject in need thereof, such as a subject with deficient LAL protein expression or a subject having Cholesteryl Ester Storage Disease.

公开(公告)号：US20240093241A1

公开(公告)日：2024-03-21

申请号：US18324928

申请日：2023-05-26

Applicant: The Regents of the University of Colorado, a Body Corporate

Inventor： Andrew Garst ,  Ryan T Gill

IPC: C12N15/90 ,  C12N9/22 ,  C12N15/10 ,  C12N15/11 ,  C40B40/08

CPC classification number: C12N15/902 ,  C12N9/22 ,  C12N15/102 ,  C12N15/1024 ,  C12N15/1082 ,  C12N15/1093 ,  C12N15/11 ,  C12N15/111 ,  C40B40/08 ,  C12N2310/10 ,  C12N2310/20 ,  C12N2320/11 ,  C12N2320/12 ,  C12N2330/31

Abstract: Described herein are method for generating a vector for editing a cell. The method comprises ligating into a vector that encodes a portion of a gRNA a cassette comprising at least one editing cassette, a promoter, and a gene encoding another portion of the gRNA. Upon ligation, the portion of the gRNA from the editing cassette and the other portion of the gRNA are ligated and form a functional gRNA.

公开(公告)号：US20230287428A1

公开(公告)日：2023-09-14

申请号：US17927658

申请日：2021-05-27

Applicant: EmendoBio Inc.

Inventor： Rafi Emmanuel ,  Michal Golan Mashiach

IPC: C12N15/113 ,  C12N15/10

CPC classification number: C12N15/1137 ,  C12N15/102 ,  C12N2310/20 ,  C12N2320/11

Abstract: RNA molecules comprising a guide sequence portion having 17-50 contiguous nucleotides containing nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-12105 and compositions, methods, and uses thereof.

公开(公告)号：US11661590B2

公开(公告)日：2023-05-30

申请号：US16324476

申请日：2017-08-09

Applicant: President and Fellows of Harvard College

Inventor： David R. Liu ,  Brian Chaikind ,  Jeffrey L. Bessen

IPC: C12N9/12 ,  C07K4/00 ,  C12N9/22 ,  C12N15/113

CPC classification number: C12N9/1241 ,  C07K4/00 ,  C12N9/22 ,  C12N15/113 ,  C07K2319/09 ,  C07K2319/21 ,  C07K2319/43 ,  C12N2310/20 ,  C12N2320/11 ,  C12Y207/07

Abstract: Some aspects of this disclosure provide a fusion protein comprising a guide nucleotide sequence-programmable DNA binding protein domain (e.g., a nuclease-inactive variant of Cas9 such as dCas9), an optional linker, and a recombinase catalytic domain (e.g., a tyrosine recombinase catalytic domain or a serine recombinase catalytic domain such as a Gin recombinase catalytic domain). This fusion protein can recombine DNA sites containing a minimal recombinase core site flanked by guide RNA-specified sequences. The instant disclosure represents a step toward programmable, scarless genome editing in unmodified cells that is independent of endogenous cellular machinery or cell state.

公开(公告)号：US20180371499A1

公开(公告)日：2018-12-27

申请号：US16056310

申请日：2018-08-06

Applicant: THE REGENTS OF THE UNIVERSITY OF COLORADO, A BODY CORPORATE

Inventor： Ryan T. GILL ,  Andrew GARST

IPC: C12N15/90 ,  C12N15/11 ,  C12N15/10 ,  C12N9/22 ,  C40B40/08

CPC classification number: C12N15/902 ,  C12N9/22 ,  C12N15/102 ,  C12N15/1024 ,  C12N15/1082 ,  C12N15/1093 ,  C12N15/11 ,  C12N15/111 ,  C12N2310/10 ,  C12N2310/20 ,  C12N2320/11 ,  C12N2320/12 ,  C12N2330/31 ,  C40B40/08

Abstract: Described herein are methods and vectors for rational, multiplexed manipulation of chromosomes within open reading frames (e.g., in protein libraries) or any segment of a chromosome in a cell or population of cells, in which various CRISPR systems are used.

公开(公告)号：US20180320182A1

公开(公告)日：2018-11-08

申请号：US16038029

申请日：2018-07-17

Applicant: Thermo Fisher Scientific Inc.

Inventor： Anastasia Khvorova ,  Angela Reynolds ,  Devin Leake ,  William Marshall ,  Steven Read ,  Stephen Scaringe

IPC: C12N15/113 ,  C12N15/10 ,  A61K31/713 ,  C12N15/11

CPC classification number: C12N15/113 ,  A61K31/713 ,  C12N15/1048 ,  C12N15/111 ,  C12N15/1135 ,  C12N15/1136 ,  C12N15/1137 ,  C12N15/1138 ,  C12N2310/14 ,  C12N2320/10 ,  C12N2320/11 ,  C12Y113/12007 ,  C12Y502/01008

Abstract: Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to nucleotide sequences for TTR.

公开(公告)号：US20180230493A1

公开(公告)日：2018-08-16

申请号：US15948789

申请日：2018-04-09

Applicant: THE REGENTS OF THE UNIVERSITY OF COLORADO, A BODY CORPORATE

Inventor： Ryan T. GILL ,  Andrew GARST

IPC: C12N15/90 ,  C12N15/10 ,  C12N15/11 ,  C12N9/22

CPC classification number: C12N15/902 ,  C12N9/22 ,  C12N15/102 ,  C12N15/1024 ,  C12N15/1082 ,  C12N15/1093 ,  C12N15/11 ,  C12N15/111 ,  C12N2310/10 ,  C12N2310/20 ,  C12N2320/11 ,  C12N2320/12 ,  C12N2330/31 ,  C40B40/08

Abstract: Described herein are methods and vectors for rational, multiplexed manipulation of chromosomes within open reading frames (e.g., in protein libraries) or any segment of a chromosome in a cell or population of cells, in which various CRISPR systems are used.

公开(公告)号：US20180201934A1

公开(公告)日：2018-07-19

申请号：US15862479

申请日：2018-01-04

Applicant: Thermo Fisher Scientific Inc.

Inventor： Anastasia Khvorova ,  Angela Reynolds ,  Devin Leake ,  William Marshall ,  Steven Read ,  Stephen Scaringe

IPC: C12N15/113 ,  A61K31/713 ,  C12N15/10 ,  C12N15/11 ,  C12N15/82

CPC classification number: C12N15/1137 ,  A61K31/713 ,  C12N15/1048 ,  C12N15/111 ,  C12N15/113 ,  C12N15/1135 ,  C12N15/1136 ,  C12N15/1138 ,  C12N15/8218 ,  C12N2310/14 ,  C12N2310/31 ,  C12N2310/322 ,  C12N2320/10 ,  C12N2320/11 ,  C12N2320/13 ,  C12Y113/12007 ,  C12Y502/01008

Abstract: Efficient sequence specific gene silencing is possible through the use of siRNA technology. Be selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods compositions, and kits generated through rational design of siRNAs are disclosed, including those directed to the nucleotide sequences for HAO1.

公开(公告)号：US10011836B2

公开(公告)日：2018-07-03

申请号：US14936403

申请日：2015-11-09

Applicant: Thermo Fisher Scientific Inc.

Inventor： Anastasia Khvorova ,  Angela Reynolds ,  Devin Leake ,  William Marshall ,  Steven Read ,  Stephen Scaringe

IPC: C12N15/11 ,  C12N15/113 ,  A61K31/713 ,  C12N15/10 ,  C12N15/82

CPC classification number: C12N15/1138 ,  A61K31/713 ,  C12N15/1048 ,  C12N15/111 ,  C12N15/113 ,  C12N15/1135 ,  C12N15/1136 ,  C12N15/1137 ,  C12N15/8218 ,  C12N2310/14 ,  C12N2320/10 ,  C12N2320/11 ,  C12N2320/13 ,  C12N2330/31 ,  C12Y113/12007 ,  C12Y502/01008

Abstract: Efficient sequence specific gene silencing is possible through the use of siRNA technology. Be selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods compositions, and kits generated through rational design of siRNAs are disclosed, including those directed to the nucleotide sequences for F12.

公开(公告)号：US09982256B2

公开(公告)日：2018-05-29

申请号：US14886378

申请日：2015-10-19

Applicant: The Trustees of the University of Pennsylvania

Inventor： Robert B. Wilson ,  Yongping Wang

IPC: C12N15/113 ,  C40B50/00 ,  C12N15/10 ,  C12N15/11 ,  C12N15/65 ,  C12N15/66

CPC classification number: C12N15/1086 ,  C12N15/111 ,  C12N15/113 ,  C12N15/65 ,  C12N15/66 ,  C12N2310/14 ,  C12N2310/53 ,  C12N2320/11 ,  C12N2330/31

Abstract: This invention provides expression vectors for a ribonucleic acid (RNA) molecule comprising a double-stranded region of random sequence, sets and libraries of same, methods of generating same, and methods for identifying an RNA therapeutic or RNA molecule that has an ability to affect a biological parameter, for identifying a drug target for a disease or disorder of interest, and for identifying a variant of an RNA molecule that has an altered ability to affect a biological parameter of interest.