公开(公告)号：US11912994B2

公开(公告)日：2024-02-27

申请号：US17214320

申请日：2021-03-26

Applicant: The General Hospital Corporation

Inventor： Jeannie T. Lee ,  Anand Minajigi ,  Lieselot Carrette

IPC: C12N15/113 ,  C12N9/10 ,  C12Q1/6883 ,  C12N9/90

CPC classification number: C12N15/113 ,  C12N9/1007 ,  C12N9/90 ,  C12Q1/6883 ,  C12Y201/01037 ,  C12Y599/01002 ,  C12N2310/113 ,  C12N2310/14 ,  C12N2310/20 ,  C12N2310/3231 ,  C12N2310/531 ,  C12N2320/34 ,  C12Q2600/156 ,  C12N2310/14 ,  C12N2310/531

Abstract: Methods for reactivating genes on the inactive X chromosome that include administering one or both of a DNA methyltransferase (DNMT) Inhibitor and/or a topoisomerase inhibitor, e.g., etoposide and/or 5′-azacytidine (aza), optionally in combination with an inhibitor of XIST RNA and/or an Xist-interacting protein, e.g., a chromatin-modifying protein, e.g., a small molecule or an inhibitory nucleic acid (such as a small inhibitory RNA (siRNAs) or antisense oligonucleotide (ASO)) that targets XIST RNA and/or a gene encoding an Xist-interacting protein, e.g., a chromatin-modifying protein.

公开(公告)号：US20230183662A1

公开(公告)日：2023-06-15

申请号：US17552076

申请日：2021-12-15

Applicant: Zhejiang University

Inventor： Yuejin Hua ,  Shengjie Li ,  Liangyan Wang ,  Jianling Cai ,  Xiaoting Hua

IPC: C12N9/10

CPC classification number: C12N9/1007 ,  C12Y201/01037

Abstract: A DNA methyltransferase is derived from Deinococcus radiodurans and has a typical conservative structural domain of DNA methyltransferase. The DNA methyltransferase includes: an AdoMet binding region containing a “FxGxG” conservative sequence, a target sequence recognition region and a catalytic region containing a “TSPPY” conservative sequence sequentially from N-terminal to C-terminal; and belongs to α-type DNA methyltransferase category. The recognized substrate DNA conservative sequence is 5′-CCGCGG-3′, a methylation modified position is N4 site of second cytosine to generate a 4mC type modified base, and an optimum temperature for methylated reaction is in a range of 25-37° C. The DNA methyltransferase can specific-recognize the conservative motif of “CCGCGG” and methylate the N4 site of the second cytosine to produce the 4mC modified base, which is a N4-Cytosine DNA methyltransferase.

公开(公告)号：US20180179525A1

公开(公告)日：2018-06-28

申请号：US15821632

申请日：2017-11-22

Applicant: Flagship Pioneering, Inc.

Inventor： Laura Gabriela Lande ,  David Arthur Berry ,  Rahul Karnik

IPC: C12N15/113 ,  C12N15/63 ,  C12N15/90

CPC classification number: C12N15/113 ,  A01K2217/056 ,  A01K2227/706 ,  A01K2267/0318 ,  A61K35/12 ,  C07K2319/00 ,  C07K2319/85 ,  C12N9/00 ,  C12N9/1007 ,  C12N9/22 ,  C12N9/80 ,  C12N15/63 ,  C12N15/85 ,  C12N15/87 ,  C12N15/90 ,  C12N2310/14 ,  C12N2310/20 ,  C12Y201/01037 ,  C12Y201/01043 ,  C12Y305/01098

Abstract: The present disclosure provides compositions with a modulating gene expression and methods for modulating transcription.

公开(公告)号：US20120252072A1

公开(公告)日：2012-10-04

申请号：US13439069

申请日：2012-04-04

Applicant: JANET WESTPHELING ,  DaeHwan Chung ,  Jennifer Huddleston ,  Joel A. Farkas

Inventor： JANET WESTPHELING ,  DaeHwan Chung ,  Jennifer Huddleston ,  Joel A. Farkas

IPC: C12N15/74 ,  C12N15/55 ,  C12P19/34 ,  C12N1/21 ,  C12N9/16 ,  C12N9/10 ,  C12N15/63 ,  C12N15/54

CPC classification number: C12N9/22 ,  C12N9/1007 ,  C12P19/34 ,  C12Y201/01037 ,  C12Y301/21004

Abstract: The present invention relates to the discovery of a novel restriction/modification system in Caldicellulosiruptor bescii. The discovered restriction enzyme is a HaeIII-like restriction enzyme that possesses a thermophilic activity profile. The restriction/modification system also includes a methyltransferase, M.CbeI, that methylates at least one cytosine residue in the CbeI recognition sequence to m4C. Thus, the invention provides, in various aspects, isolated CbeI or M.CbeI polypeptides, or biologically active fragments thereof; isolated polynucleotides that encode the CbeI or M.CbeI polypeptides or biologically active fragments thereof, including expression vectors that include such polynucleotide sequences; methods of digesting DNA using a CbeI polypeptide; methods of treating a DNA molecule using a M.CbeI polypeptide; and methods of transforming a Caldicellulosiruptor cell.

Abstract translation: 本发明涉及在Caldicellulosiruptor bescii中发现新的限制/修饰系统。 所发现的限制酶是具有嗜热活性分布的HaeIII样限制酶。 限制/修饰系统还包括甲基转移酶M.CbeI，其将CbeI识别序列中的至少一个胞嘧啶残基甲基化为m4C。 因此，本发明在各个方面提供了分离的CbeI或M.CbeI多肽或其生物活性片段; 编码CbeI或M.CbeI多肽或其生物活性片段的分离的多核苷酸，包括包括这种多核苷酸序列的表达载体; 使用CbeI多肽消化DNA的方法; 使用M.CbeI多肽处理DNA分子的方法; 和转化Caldicellulosiruptor细胞的方法。

公开(公告)号：US20240279623A1

公开(公告)日：2024-08-22

申请号：US18444590

申请日：2024-02-16

Applicant: Whitehead Institute for Biomedical Research

Inventor： Edwin Neumann ,  Tessa Bertozzi ,  Jonathan Weissman

IPC: C12N9/10 ,  A61K38/46 ,  C12N9/22 ,  C12N15/86

CPC classification number: C12N9/1007 ,  A61K38/465 ,  C12N9/22 ,  C12N15/86 ,  C12Y201/01037 ,  C07K2319/80 ,  C12N2750/14143

Abstract: The disclosure provides, in various embodiments, fusion proteins comprising a DNA-binding domain, a DNMT3A-binding domain, and a H3K4me0; and polynucleotides and vectors encoding one or more of the fusion proteins. The disclosure also provides, in various embodiments, gene-delivery systems, cells, compositions (e.g., pharmaceutical compositions) and kits comprising one or more of the fusion proteins polynucleotides, or vectors; methods of epigenetically modifying a genomic locus in a cell; and methods of treating a subject (e.g., a human) in need thereof.

公开(公告)号：US20240132855A1

公开(公告)日：2024-04-25

申请号：US18473990

申请日：2023-09-25

Applicant: Chroma Medicine, Inc.

Inventor： Aron Brandon Jaffe ,  Noorussahar Abubucker ,  Yesseinia Anglero-Rodriguez ,  Vic Myer ,  Angelo Leone Lombardo ,  Martino Alfredo Cappelluti

IPC: C12N7/00 ,  C07K14/47 ,  C12N9/10 ,  C12N9/22 ,  C12N15/11

CPC classification number: C12N7/00 ,  C07K14/4703 ,  C12N9/1007 ,  C12N9/22 ,  C12N15/11 ,  C07K2319/09 ,  C12N2310/20 ,  C12N2710/00021 ,  C12Y201/01037

Abstract: This invention relates to compositions, methods, strategies, and treatment modalities related to the epigenetic modification of hepatitis B virus (HBV) genes.

公开(公告)号：US20240123089A1

公开(公告)日：2024-04-18

申请号：US18516840

申请日：2023-11-21

Applicant: Scribe Therapeutics Inc.

Inventor： Jason FERNANDES ,  Sean HIGGINS ,  Sarah DENNY ,  Ross WHITE ,  Emeric Jean Marius CHARLES ,  Addison WRIGHT ,  Benjamin DEMAREE ,  Manuel MOHR ,  Wenyuan ZHOU ,  Benjamin OAKES

IPC: A61K48/00 ,  C12N9/10 ,  C12N9/22 ,  C12N15/113 ,  C12N15/88 ,  C12N15/90

CPC classification number: A61K48/005 ,  C12N9/1007 ,  C12N9/22 ,  C12N15/1137 ,  C12N15/88 ,  C12N15/907 ,  C12Y201/01037 ,  C07K2319/80 ,  C12N2310/20 ,  C12N2310/531

Abstract: Provided herein are gene repressor systems comprising fusion proteins, such as fusion proteins comprising a DNA binding domain such as a TALE, zinc finger or catalytically-dead CRISPR protein and guide nucleic acid (gRNA), which are useful in the repression of a proprotein convertase subtilisin kexin Type 9 (PCSK9) gene. Also provided are methods of using such systems to repress transcription of PCSK9.

公开(公告)号：US20230374549A1

公开(公告)日：2023-11-23

申请号：US18028975

申请日：2021-09-29

Applicant: FLAGSHIP PIONEERING INNOVATIONS V, INC.

Inventor： Lauren Marie BEECH ,  Jesse Jerome SMITH ,  Rahul KARNIK ,  Kendrick Alan GOSS ,  Adam Walter SCHEIDEGGER ,  Jodi Michelle KENNEDY ,  Jeremiah Dale FARELLI ,  Houda BELAGHZAL ,  Laura Anh NGUYEN ,  Charles W. O'DONNELL

IPC: C12N15/90 ,  C12N9/22 ,  C12N15/11 ,  C12N9/10 ,  C12N9/80 ,  A61P37/06 ,  A61K35/12 ,  A61K38/46 ,  A61K31/7088 ,  C07K14/47

CPC classification number: C12N15/907 ,  C12N9/22 ,  C12N15/11 ,  C12N9/1007 ,  C12Y201/01043 ,  C12N9/80 ,  C12Y305/01098 ,  A61P37/06 ,  A61K35/12 ,  A61K38/465 ,  A61K31/7088 ,  C07K14/4703 ,  C12Y201/01037 ,  C12Y201/01072 ,  C12N2310/20 ,  A61K2035/122 ,  C07K2319/00

Abstract: The present disclosure relates to site-specific disrupting agents for modulating, e.g., decreasing, expression of a target plurality of genes in a cell. In some embodiments, the target plurality of genes comprises pro-inflammatory genes, e.g., CXCL1, CXCL2, CXCL3, CXCL4, CXCL5, CXCL6, CXCL7, and IL-8. In some embodiments, the method comprises using a first site-specific disrupting agent that targets a first anchor sequence and a second site-specific disrupting agents that disrupts a second anchor sequence.

公开(公告)号：US20180256749A1

公开(公告)日：2018-09-13

申请号：US15760765

申请日：2016-09-16

Applicant: University of Massachusetts

Inventor： Michael R. Green ,  Minggang Fang ,  Walter Kowtoniuk

IPC: A61K48/00 ,  A61K31/496 ,  A61K31/713 ,  A61K31/549 ,  A61K31/706 ,  A61K31/5377 ,  A61K39/395 ,  A61K38/45 ,  A61K38/53 ,  A61K31/711 ,  A61K31/7105 ,  A61K35/30 ,  C12N15/113 ,  C12Q1/6883 ,  A61P25/14 ,  A61P15/02

CPC classification number: A61K48/005 ,  A61K31/167 ,  A61K31/404 ,  A61K31/437 ,  A61K31/4406 ,  A61K31/496 ,  A61K31/497 ,  A61K31/506 ,  A61K31/522 ,  A61K31/5377 ,  A61K31/546 ,  A61K31/549 ,  A61K31/706 ,  A61K31/7088 ,  A61K31/7105 ,  A61K31/711 ,  A61K31/713 ,  A61K35/30 ,  A61K38/45 ,  A61K38/53 ,  A61K39/3955 ,  A61K48/0016 ,  A61P15/02 ,  A61P25/14 ,  C12N15/113 ,  C12N2310/11 ,  C12N2310/14 ,  C12N2310/141 ,  C12N2310/531 ,  C12Q1/6883 ,  C12Q2600/154 ,  C12Q2600/158 ,  C12Y201/01037 ,  C12Y201/01043 ,  C12Y603/02019

Abstract: The disclosure relates to methods and compositions for reactivating a silenced FMR1 gene. In some aspects, methods described by the disclosure are useful for treating a FMR1-inactivation-associated disorder (e.g., fragile X syndrome).

公开(公告)号：US09963705B2

公开(公告)日：2018-05-08

申请号：US15034092

申请日：2014-11-10

Applicant: New England Biolabs, Inc.

Inventor： Sriharsa Pradhan ,  Pierre O. Esteve ,  Guoqiang Zhang

IPC: C12N15/113 ,  A61K31/7105

CPC classification number: C12N15/1137 ,  A61K31/7105 ,  C12N2310/11 ,  C12N2310/141 ,  C12N2310/151 ,  C12N2320/30 ,  C12Y201/01037

Abstract: RNA molecules inhibiting a DNMT and methods and compositions incorporating or generating the RNA molecules are described.