公开(公告)号：US12065642B2

公开(公告)日：2024-08-20

申请号：US16793272

申请日：2020-02-18

申请人： Sigma-Aldrich Co. LLC

发明人： Fuqiang Chen ,  Xiao Ding ,  Yongmei Feng ,  Gregory Davis

IPC分类号： C12N9/22 ,  C12N15/10 ,  C12N15/113 ,  C12N15/85 ,  C12N15/90 ,  C12Q1/6897 ,  A61K48/00

CPC分类号： C12N15/102 ,  C12N9/22 ,  C12N15/113 ,  C12N15/85 ,  C12N15/907 ,  C12Q1/6897 ,  A61K48/005 ,  C07K2319/09 ,  C07K2319/10 ,  C07K2319/80 ,  C07K2319/81 ,  C12N2310/20 ,  C12N2800/80

摘要： Compositions and methods for using nucleosome interacting protein domains to increase accessibility of programmable DNA modification proteins to target chromosomal sequences, thereby increasing efficiency of targeted genome/epigenetic modification in eukaryotic cells.

公开(公告)号：US20240271163A1

公开(公告)日：2024-08-15

申请号：US18441806

申请日：2024-02-14

申请人： Versitech Limited ,  Centre for Oncology and Immunology Limited

发明人： Siu Lun Wong ,  Hoi Yee Chu ,  Peng Zhou ,  Hoi Chun Fong

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

CPC分类号： C12N15/907 ,  C12N9/22 ,  C12N15/11 ,  C12N15/111 ,  C12N2310/20 ,  C12N2310/531 ,  C12N2800/80

摘要： Engineered guide RNAs having enhanced stability of interaction with Cas enzymes are disclosed. The variant sgRNAs include engineered nucleic acids in or around the stem-loop 2 region which enhance interaction with the Cas9 enzyme and impart enhanced specificity and on-target editing activity. Compositions and methods of engineered guide RNAs are provided for enhanced genomic engineering with increased on-off target specificity and on-target editing efficacy.

公开(公告)号：US20240271162A1

公开(公告)日：2024-08-15

申请号：US18568638

申请日：2022-06-10

申请人： LifeEDIT Therapeutics, Inc.

发明人： Tyson D. Bowen ,  Alexandra Briner Crawley ,  Tedd D. Elich

IPC分类号： C12N15/90 ,  C12N9/22 ,  C12N9/78 ,  C12N15/11

CPC分类号： C12N15/90 ,  C12N9/22 ,  C12N9/78 ,  C12N15/11 ,  C12Y305/04 ,  C12N2310/20

摘要： Compositions and methods useful for expressing an RNA are provided. Compositions comprise nucleic acid molecules comprising an RNA polymerase III promoter and expression constructs comprising the novel promoters operably linked to a coding sequence. The promoters can initiate transcription of RNA-coding nucleotide sequences through the recruitment of RNA polymerase III (pol III) or, in some embodiments, polypeptide-encoding nucleotide sequences through the recruitment of RNA polymerase II. The promoters thus find use in transcribing nucleotide sequences, including but not limited to, those encoding guide RNAs, and polypeptide-encoding sequences. Such expression constructs comprising the novel promoters operably linked to nucleotide sequences encoding guide RNAs find use in binding and/or modifying the sequence of a target nucleic acid molecule, modifying the expression of a target gene, or detecting a target nucleotide sequence in cells comprising an RNA-guided nuclease (RGN) or RNA-guided nucleotide-binding polypeptide (RGNBP).

公开(公告)号：US20240271128A1

公开(公告)日：2024-08-15

申请号：US18247523

申请日：2020-11-11

申请人： Temple University - of the Commonwealth System of Higher Education

发明人： Kamel KHALILI

IPC分类号： C12N15/11 ,  C12N9/22 ,  C12N15/86

CPC分类号： C12N15/111 ,  C12N9/22 ,  C12N15/86 ,  C12N2310/20 ,  C12N2750/14143

摘要： The present disclosure relates to compositions and methods for the inhibition of the infectivity of a herpesvirus. The present disclosure relates in general to compositions and methods of treating or eradicating Herpes Simplex virus infections. The disclosure relates in particular to targeting of Herpes Simplex virus genes by gene editing complexes.

公开(公告)号：US20240271116A1

公开(公告)日：2024-08-15

申请号：US18545977

申请日：2023-12-19

申请人： President and Fellows of Harvard College

发明人： Juan Pablo Maianti ,  David R. Liu

IPC分类号： C12N9/78 ,  A61K9/00 ,  A61K31/7088 ,  A61K38/46 ,  A61K38/50 ,  A61K48/00 ,  C12N9/22 ,  C12N9/96 ,  C12N15/11

CPC分类号： C12N9/78 ,  A61K9/0029 ,  A61K9/0053 ,  A61K31/7088 ,  A61K38/465 ,  A61K38/50 ,  C12N9/22 ,  C12N9/96 ,  C12N15/11 ,  C12Y305/04 ,  A61K48/00 ,  C07K2319/00 ,  C12N2310/20 ,  C12N2320/31 ,  C12N2320/32 ,  C12N2800/80

摘要： Provided herein are systems, compositions, kits, and methods for the suppression of pain (e.g., chronic pain). Genes encoding ion channels (e.g., SCN9A) responsible for the propagation pain signals in neurons (e.g., DRG neurons) may be edited using a genome editing agent (e.g., a nucleobase editor). In some embodiments, loss-of-function ion channel mutants are generated, leading to pain suppression. In some embodiments, the genome editing agent is administered locally to the site of pain or to the nerves responsible for propagation of the pain signal.

公开(公告)号：US20240271111A1

公开(公告)日：2024-08-15

申请号：US18424277

申请日：2024-01-26

申请人： PRECISION BIOSCIENCES, INC.

发明人： Derek Jantz ,  James Jefferson Smith

IPC分类号： C12N9/22 ,  A61K9/51 ,  A61K38/00 ,  A61K38/43 ,  A61P31/20 ,  C12N15/52 ,  C12N15/86

CPC分类号： C12N9/22 ,  A61K9/51 ,  A61K38/43 ,  A61P31/20 ,  C12N15/52 ,  C12N15/86 ,  A61K38/00 ,  C12N2730/10122 ,  C12N2750/14141

摘要： The present invention encompasses engineered meganucleases which recognize and cleave a recognition sequence within an open reading frame (ORF) of the genome of at least two genotypes of the Hepatitis B virus (HBV). The present invention also encompasses methods of using such engineered meganucleases in a pharmaceutical composition and in methods for treating or reducing the symptoms of a HBV infection, or treating hepatocellular carcinoma (HCC). Further, the invention encompasses pharmaceutical compositions comprising engineered meganuclease proteins, nucleic acids encoding engineered meganucleases, and the use of such compositions for treating HBV infections or HCC.

公开(公告)号：US12060589B2

公开(公告)日：2024-08-13

申请号：US18343131

申请日：2023-06-28

申请人： Novozymes A/S

发明人： Lars Henrik Oestegaard ,  Gernot J. Abel ,  Dorte Marie Koefoed Klitgaard ,  Annette Helle Johansen

IPC分类号： C12N9/22 ,  C11D3/386

CPC分类号： C12N9/22 ,  C11D3/38636 ,  C12Y301/21001

摘要： The present invention relates to polypeptide variants and methods for obtaining variants. The present invention also relates to polynucleotides encoding the variants; nucleic acid constructs, vectors, and host cells comprising the polynucleotides; and methods of using the variants.

公开(公告)号：US20240263170A1

公开(公告)日：2024-08-08

申请号：US18412210

申请日：2024-01-12

申请人： Inscripta, Inc.

发明人： Jorge Bernate ,  Kevin Ness ,  Phillip Belgrader ,  Don Masquelier ,  Ryan Gill

IPC分类号： C12N15/10 ,  C12M1/00 ,  C12M1/12 ,  C12M1/26 ,  C12M1/34 ,  C12M1/36 ,  C12M1/42 ,  C12M3/00 ,  C12N9/22 ,  C12N15/11 ,  C12Q1/6865 ,  C40B40/02

CPC分类号： C12N15/1082 ,  C12M23/44 ,  C12M33/14 ,  C12M35/00 ,  C12M37/04 ,  C12M41/36 ,  C12M41/48 ,  C12M43/00 ,  C12N9/22 ,  C12N15/11 ,  C12Q1/6865 ,  C40B40/02 ,  C12N2310/20 ,  C12N2800/80

摘要： In an illustrative embodiment, automated multi-module cell editing instruments are provided to automate multiple edits into nucleic acid sequences inside one or more cells.

公开(公告)号：US12054757B1

公开(公告)日：2024-08-06

申请号：US18424759

申请日：2024-01-27

申请人： Hubei University

发明人： Lixin Ma ,  Longyu Wang ,  Fei Wang ,  Xiaochen Xie ,  Wanping Chen ,  Fuyong Huang

IPC分类号： C12N9/22 ,  C12N15/11 ,  C12N15/90 ,  C12Q1/6823

CPC分类号： C12N9/22 ,  C12N15/11 ,  C12N15/90 ,  C12Q1/6823 ,  C12Y301/00 ,  C12N2310/20 ,  C12N2800/80

摘要： A Pyrococcus furiosus Argonaute (PfAgo) mutant protein with mesophilic target nucleic acid cleavage activity and an application thereof are provided. The PfAgo mutant protein has an amino acid mutation on at least one of a 617th position and a 618th position relative to the sequence shown in SEQ ID NO: 1, specifically, K617E/G and/or L618Y/F/W/G. Compared with a wild-type PfAgo protein, the provided PfAgo mutant protein has activity in the temperature range of 30-95 Celsius degree (° C.), effectively expanding application range of prokaryotic Argonaute (pAgo) protein.

公开(公告)号：US12054739B2

公开(公告)日：2024-08-06

申请号：US18472824

申请日：2023-09-22

申请人： ReNAgade Therapeutics Management Inc.

发明人： Alim Ladha ,  Vladimir Presnyak ,  Inna Shcherbakova ,  Brian Goodman ,  Mario Rodriguez Mestre ,  Devin Scott Quinlan ,  Muthusamy Jayaraman ,  Stephen Scully

IPC分类号： C12N9/22 ,  C12N9/12 ,  C12N15/11 ,  C12N15/90

CPC分类号： C12N15/907 ,  C12N9/1276 ,  C12N9/22 ,  C12N15/11 ,  C12N2310/20 ,  C12N2310/531 ,  C12N2800/80

摘要： Disclosed are engineered retrons and methods of use such as to modify the genome of a host (e.g., mammalian) cell by delivering the engineered retron or the encoded ncRNA in vitro or in vivo to the host (e.g., mammalian) cell.