公开(公告)号：US20240352454A1

公开(公告)日：2024-10-24

申请号：US18430788

申请日：2024-02-02

Applicant: THE BROAD INSTITUTE, INC. ,  MASSACHUSETTS INSTITUTE OF TECHNOLOGY ,  PRESIDENT AND FELLOWS OF HARVARD COLLEGE

Inventor： Feng Zhang ,  Jonathan Gootenberg ,  Omar Abudayyeh

IPC: C12N15/113 ,  B01L3/00 ,  C12N9/22 ,  G01N33/53

CPC classification number: C12N15/113 ,  B01L3/5085 ,  C12N9/22 ,  G01N33/5308 ,  B01L2300/123 ,  B01L2300/126 ,  C12N2310/20 ,  C12N2800/80

Abstract: The embodiments disclosed herein utilized RNA targeting effectors to provide a robust CRISPR-based diagnostic with attomolar sensitivity. Embodiments disclosed herein can detect both DNA and RNA with comparable levels of sensitivity and can differentiate targets from non-targets based on single base pair differences. Moreover, the embodiments disclosed herein can be prepared in freeze-dried format for convenient distribution and point-of-care (POC) applications. Such embodiments are useful in multiple scenarios in human health including, for example, viral detection, bacterial strain typing, sensitive genotyping, and detection of disease-associated cell free DNA.

公开(公告)号：US12123033B2

公开(公告)日：2024-10-22

申请号：US17079097

申请日：2020-10-23

Applicant: INTEGRATED DNA TECHNOLOGIES, INC.

Inventor： Jessica Woodley ,  Bernice Thommandru ,  Joseph Dobosy ,  Mark Behlke ,  Adam Clore ,  Garrett Rettig ,  Beimeng Sun

IPC: C12N15/66 ,  C12N9/22 ,  C12N15/113

CPC classification number: C12N9/22 ,  C12N15/113 ,  C12N15/66 ,  C12N2310/122 ,  C12N2310/20 ,  C12N2310/321 ,  C12N2310/531 ,  C12N2800/80

Abstract: Described herein are compositions and methods for improving homology directed repair (HDR) efficiency and reducing homology-independent integration following introduction of double strand breaks with engineered nucleases. Additionally, modifications to double stranded DNA donors to improve the donor potency and efficiency of homology directed repair following introduction of double stranded breaks with programmable nucleases.

公开(公告)号：US12123015B2

公开(公告)日：2024-10-22

申请号：US17481085

申请日：2021-09-21

Applicant: The Regents of the University of California ,  University of Vienna

Inventor： Jennifer A. Doudna ,  Martin Jinek ,  Krzysztof Chylinski ,  Emmanuelle Charpentier

IPC: C12N15/90 ,  A01H6/46 ,  A01K67/027 ,  A61K38/46 ,  A61K48/00 ,  C12N9/22 ,  C12N15/10 ,  C12N15/11 ,  C12N15/113 ,  C12N15/63 ,  C12N15/70 ,  C12N15/74 ,  C12Q1/686 ,  H01L21/02 ,  H01L33/00

CPC classification number: C12N15/907 ,  A01H6/4684 ,  A01K67/027 ,  A61K38/465 ,  C12N9/22 ,  C12N15/102 ,  C12N15/111 ,  C12N15/113 ,  C12N15/63 ,  C12N15/70 ,  C12N15/746 ,  C12N15/90 ,  C12N15/902 ,  C12Q1/686 ,  H01L21/02312 ,  H01L21/02365 ,  H01L33/0075 ,  A61K48/00 ,  C12N2310/11 ,  C12N2310/13 ,  C12N2310/14 ,  C12N2310/20 ,  C12N2310/31 ,  C12N2310/32 ,  C12N2310/33 ,  C12N2310/3519 ,  C12N2310/531 ,  C12N2800/80 ,  C12Y301/04

Abstract: The present disclosure provides a DNA-targeting RNA that comprises a targeting sequence and, together with a modifying polypeptide, provides for site-specific modification of a target DNA and/or a polypeptide associated with the target DNA. The present disclosure further provides site-specific modifying polypeptides. The present disclosure further provides methods of site-specific modification of a target DNA and/or a polypeptide associated with the target DNA The present disclosure provides methods of modulating transcription of a target nucleic acid in a target cell, generally involving contacting the target nucleic acid with an enzymatically inactive Cas9 polypeptide and a DNA-targeting RNA. Kits and compositions for carrying out the methods are also provided. The present disclosure provides genetically modified cells that produce Cas9; and Cas9 transgenic non-human multicellular organisms.

公开(公告)号：US20240336905A1

公开(公告)日：2024-10-10

申请号：US18597260

申请日：2024-03-06

Applicant: Metagenomi, Inc.

Inventor： Brian C. THOMAS ,  Christopher BROWN ,  Cindy CASTELLE ,  Lisa ALEXANDER ,  Liliana GONZALEZ-OSORIO ,  Paula MATHEUS CARNEVALI ,  Dom CASTANZO

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

CPC classification number: C12N9/22 ,  C12N15/11 ,  C12N15/907 ,  C12N2310/20 ,  C12N2800/80

Abstract: Described herein are methods, compositions, and systems derived from uncultivated microorganisms useful for gene editing.

公开(公告)号：US20240327862A1

公开(公告)日：2024-10-03

申请号：US18605149

申请日：2024-03-14

Applicant: THE REGENTS OF THE UNIVERSITY OF COLORADO, A BODY CORPORATE ,  NATIONAL JEWISH HEALTH

Inventor： Brian Freed ,  Kirsten Anderson ,  Christina Roark ,  Jennifer Matsuda

IPC: C12N15/85 ,  A01K67/0275 ,  A61K35/28 ,  A61K48/00 ,  C12N5/0789 ,  C12N9/22 ,  C12N15/11

CPC classification number: C12N15/85 ,  A01K67/0275 ,  A61K35/28 ,  C12N5/0647 ,  C12N9/22 ,  C12N15/11 ,  A01K2217/05 ,  A01K2227/105 ,  A01K2267/0325 ,  A61K48/00 ,  C12N2310/20 ,  C12N2740/15042 ,  C12N2750/14142 ,  C12N2800/80

Abstract: Methods of preventing or treating rheumatoid arthritis (RA) in a subject by introducing the DRB1*04:01K71E mutation that is resistant to RA. The resistant allele is introduced into the subject having or at risk of developing RA, using a HLA CRISPR/Cas9 vector that targets codon 71 in the HLA allele DRB1*04:01, introducing a single A to G point mutation in codon 71 by homology directed repair to alter the lysine at position 71 of the expressed protein to glutamic acid. This modified allele is affected in the subject's hematopoietic stem cells, which are then expanded and transplanted back into the patient. This microgene therapy confers RA-resistance via an autologous transplant. The invention includes isolated nucleic acids, vectors, recombinant viruses, cells, and pharmaceutical compositions to modify the HLA DRB1*04:01 allele.

公开(公告)号：US20240327853A1

公开(公告)日：2024-10-03

申请号：US18743454

申请日：2024-06-14

Applicant: Keygene N.V.

Inventor： Bjorn Alexander KLOOSTERMAN ,  Franck Georges Paul LHUISSIER

IPC: C12N15/82 ,  C12N9/22 ,  C12N15/11

CPC classification number: C12N15/8213 ,  C12N9/22 ,  C12N15/111 ,  C12N15/8203 ,  C12N2310/20 ,  C12N2800/80

Abstract: The invention pertains to a method for producing a shoot of a plant, wherein the shoot comprises one or more cells having a genetic modification. The method preferably comprises the steps of: i) providing a plant, ii) introducing in a cell of the plant a vector expressing at least one of a gRNA and a CRISPR-nuclease, iii) removing the shoot apical meristem of the plant, and iv) regenerating a shoot at a second location in the plant, wherein the shoot comprises one or more cells having a genetic modification. The shoot apical meristem of the plant is preferably removed by decapitating the plant. Similarly, the shoot is preferably regenerated by a second decapitation of the plant at a second location. The invention further related to a plant having a shoot comprising one or more cells having a genetic modification, wherein the plant is obtainable by the method of the invention.

公开(公告)号：US12104179B2

公开(公告)日：2024-10-01

申请号：US18474786

申请日：2023-09-26

Applicant: EMPYREAN NEUROSCIENCE, INC.

Inventor： Thomas Henley ,  Modassir Choudhry ,  Jose Fernandez-Gomez ,  Antoine Larrieu ,  Beata Orman-Ligeza ,  Umar Mohammed ,  Emma McKechnie-Welsh

IPC: C12N9/10 ,  A61K35/66 ,  C12N9/22 ,  C12N15/11

CPC classification number: C12N9/1007 ,  A61K35/66 ,  C12N9/22 ,  C12N15/11 ,  C12N2310/20 ,  C12N2800/80

Abstract: This disclosure provides genetically engineered organisms with genetic modifications that are useful for producing desirable alkaloids. This disclosure also provides methods of making genetically engineered organisms that can produce desirable alkaloids. The organisms described herein produce alkaloids in amounts beyond that produced from comparable wild-type organisms. In certain embodiments, the genetically engineered organisms are fungi from the Basidiomycota division.

公开(公告)号：US20240318200A1

公开(公告)日：2024-09-26

申请号：US18444368

申请日：2024-02-16

Applicant: CRISPR THERAPEUTICS AG

Inventor： Ante Sven Lundberg ,  Samarth Kulkarni ,  Lawrence Klein ,  Hari Kumar Padmanabhan ,  Yvonne Sarah Aratyn

IPC: C12N15/86 ,  C12N7/00 ,  C12N9/22 ,  C12N15/11

CPC classification number: C12N15/86 ,  C12N7/00 ,  C12N9/22 ,  C12N15/111 ,  C12N2310/20 ,  C12N2750/14143 ,  C12N2800/80

Abstract: The present application provides materials and methods for treating a patient with one or more conditions associated with ANGPTL3 whether ex vivo or in vivo. In addition, the present application provides materials and methods for editing and/or modulating the expression of ANGPTL3 gene in a cell by genome editing.

公开(公告)号：US20240318155A1

公开(公告)日：2024-09-26

申请号：US18613420

申请日：2024-03-22

Applicant: Inscripta, Inc.

Inventor： Juhan Kim ,  Benjamin Mijts

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

CPC classification number: C12N9/22 ,  C12N15/11 ,  C12N15/907 ,  C12Y301/21004 ,  C12N2310/20 ,  C12N2800/80

Abstract: The present disclosure provides engineered nucleic acid-guided nickases and optimized scaffolds for making rational, direct edits to nucleic acids in live cells.

公开(公告)号：US12098363B2

公开(公告)日：2024-09-24

申请号：US16964461

申请日：2018-07-20

Applicant: THE CHILDREN'S MEDICAL CENTER CORPORATION ,  UNIVERSITY OF MASSACHUSETTS

Inventor： Daniel E. Bauer ,  Scot Wolfe ,  Mehmet Fatih Bolukbasi ,  Benjamin Roscoe ,  Pengpeng Liu ,  Kevin Luk ,  Yuxuan Wu ,  Jing Zeng

IPC: C12N15/01 ,  A61K38/46 ,  C12N9/22 ,  C12N15/113

CPC classification number: C12N15/01 ,  A61K38/465 ,  C12N9/22 ,  C12N15/113 ,  C12N2310/20 ,  C12N2510/00 ,  C12N2800/80

Abstract: Provided herein are methods and compositions for increasing fetal hemoglobin levels in a cell by disrupting BCL11A expression at the genomic level. Also provided herein are methods and compositions relating to the treatment of hemoglobinopathies by reinduction of fetal hemoglobin levels.