公开(公告)号：US20240327867A1

公开(公告)日：2024-10-03

申请号：US18605384

申请日：2024-03-14

Applicant: FLAGSHIP PIONEERING INNOVATIONS V, INC.

Inventor： Joseph Michael Cabral ,  Cato Hawkins Prince ,  Bryan Alexander McGuffie ,  Hassana Kia Oyibo ,  Simon Delagrave ,  Dhananjay Maniklal Nawandar ,  Kevin James Lebo ,  Cesar Augusto Arze ,  Agamoni Bhattacharyya ,  Nathan Lawrence Yozwiak ,  Roger Joseph Hajjar ,  Geoffrey Parsons

IPC: C12N15/86 ,  A61K47/64 ,  C07K14/005

CPC classification number: C12N15/86 ,  A61K47/64 ,  C07K14/005 ,  C12N2750/00022 ,  C12N2750/00023 ,  C12N2750/00043 ,  C12N2750/00051 ,  C12N2750/00071 ,  C12N2830/008 ,  C12N2840/203

Abstract: This invention relates generally to modified anellovirus capsid proteins, anellovectors, anelloVLPs, and compositions and uses thereof.

公开(公告)号：US20240175041A1

公开(公告)日：2024-05-30

申请号：US18523237

申请日：2023-11-29

Applicant: RESEARCH & BUSINESS FOUNDATION SUNGKYUNKWAN UNIVERSITY

Inventor： Suk Chan LEE ,  Aamir LAL

IPC: C12N15/82 ,  C12N7/00 ,  C12Q1/6806 ,  C12Q1/686 ,  C12Q1/70

CPC classification number: C12N15/8203 ,  C12N7/00 ,  C12Q1/6806 ,  C12Q1/686 ,  C12Q1/701 ,  C12N2750/00043

Abstract: Disclosed herein are an infectious clone capable of inoculating Nanovirus DNA-C, DNA-M, DNA-N, and DNA-U1, and uses thereof. A recombinant vector comprising the nucleotide sequences of Nanovirus DNA-C, DNA-M, DNA-N, and DNA-U1 represented by SEQ ID NOS: 1 to 8, and E. coli and Agrobacterium tumefaciens, each transformed therewith, can effectively induce the infection of Nanovirus into crops even without any insect vectors. Therefore, the disclosure can be applied to various studies, such as investigating the host range and the correlation with hosts of Nanovirus DNA-C, DNA-M, DNA-N, and DNA-U1. This can help in preemptively preventing economic losses due to virus infections, making it beneficially applicable in the related fields.

公开(公告)号：US20230227849A1

公开(公告)日：2023-07-20

申请号：US18010674

申请日：2021-06-17

Applicant: FLAGSHIP PIONEERING INNOVATIONS V, INC.

Inventor： Avak Kahvejian ,  Erica Gabrielle Weinstein ,  Roger Joseph Hajjar ,  Nathan Lawrence Yozwiak ,  Simon Delagrave ,  Yann Paul Guy Régis Echelard ,  Simeon Ungerleider Springer ,  Cesar A. Arze ,  Kristian Graugaard Andersen

IPC: C12N15/86 ,  C12Q1/70 ,  A61K48/00

CPC classification number: C12N15/86 ,  C12Q1/701 ,  A61K48/0066 ,  A61K48/0041 ,  C12N2750/00043 ,  C12N2750/00071

Abstract: This invention relates generally to compositions and methods for administering an anellovector (e.g., a synthetic anellovector) that can be used as a delivery vehicle, e.g., for delivering genetic material, for delivering an effector, e.g., a payload, or for delivering a therapeutic agent or a therapeutic effector to a eukaryotic cell (e.g., a human cell or a human tissue). Also provided are methods for amplifying circular nucleic acids comprising Anellovirus sequences.

公开(公告)号：US20150218586A1

公开(公告)日：2015-08-06

申请号：US14514606

申请日：2014-10-15

Applicant: PLASMIDFACTORY GMBH & CO. KG

Inventor： MARTIN SCHLEEF

IPC: C12N15/86

CPC classification number: C12N15/86 ,  C12N2740/10052 ,  C12N2750/00043 ,  C12N2750/14152 ,  C12N2770/00043 ,  C12N2800/107 ,  C12N2800/30 ,  C12N2800/40 ,  C12N2800/50

Abstract: The invention relates to a minicircle transfer vector for producing viral vectors comprising a transfer sequence and specific packing signals flanking both sides of the transfer sequence for packaging of the transfer sequence into particles of a viral vector. The invention also relates to minicircle packaging vectors carrying support functions for producing viral vectors. The invention further relates to cells bearing the disclosed minicircles. The invention further relates to methods for producing viral vectors using such minicircles and viral vectors obtained thereby, as well as kits useful in performing the described methods.

Abstract translation: 本发明涉及用于产生病毒载体的小环转移载体，其包含转移序列和转移序列两侧侧翼的特异性包装信号，用于将转移序列包装到病毒载体的颗粒中。 本发明还涉及携带用于产生病毒载体的支持功能的小环包装载体。 本发明还涉及带有所公开的微型圆筒的电池。 本发明还涉及使用这样的小环和由此获得的病毒载体产生病毒载体的方法，以及可用于实施所述方法的试剂盒。

公开(公告)号：US20150167000A1

公开(公告)日：2015-06-18

申请号：US14629859

申请日：2015-02-24

Applicant: Regents of the University of Minnesota

Inventor： Daniel F. Voytas ,  Paul Atkins ,  Nicholas J. Baltes

IPC: C12N15/113 ,  C12N15/52 ,  C12N9/16 ,  C12N15/82

CPC classification number: C12N15/1131 ,  C12N9/16 ,  C12N15/52 ,  C12N15/8203 ,  C12N15/8205 ,  C12N15/8207 ,  C12N15/8213 ,  C12N2750/00043 ,  C12Y301/21

Abstract: Materials and methods for gene targeting using Clustered Regularly Interspersed Short Palindromic Repeats/CRISPR-associated (CRISPR/Cas) systems are provided herein.

Abstract translation: 本文提供了使用聚类经常散布的短回文重复/ CRISPR相关（CRISPR / Cas）系统进行基因靶向的材料和方法。

公开(公告)号：US20150065560A1

公开(公告)日：2015-03-05

申请号：US14242648

申请日：2014-04-01

Applicant: Genepod Therapeutics AB

Inventor： Tomas Björklund ,  Anders Björklund ,  Deniz Kirik

IPC: C12N7/00 ,  C12N9/02 ,  C12N9/78

CPC classification number: C12N7/00 ,  A61K48/00 ,  C12N9/0071 ,  C12N9/78 ,  C12N9/88 ,  C12N15/86 ,  C12N2750/00043 ,  C12N2750/14143 ,  C12N2830/008 ,  C12N2830/34 ,  C12N2830/40 ,  C12N2830/42 ,  C12N2830/48 ,  C12N2830/50 ,  C12N2830/60 ,  C12Y114/16002 ,  C12Y305/04016

Abstract: The present invention relates to a one-vector expression system comprising a sequence encoding two polypeptides, such as tyrosine hydroxylase (TH) and GTP-cyclohydrolase 1 (GCH1). The two polypeptides can be should preferentially be expressed at a ratio between 3:1 and 15:1, such as between 3:1 and 7:1. The invention is useful in the treatment of catecholamine deficient disorders, such as dopamine deficient disorders including but not limited to Parkinson's Disease. Moreover, the present invention provides a method to deliver the vector construct in order to limit the increased production of the catecholamine to the cells in need thereof.

Abstract translation: 本发明涉及包含编码两种多肽（如酪氨酸羟化酶（TH））和GTP-环化水解酶1（GCH1）的序列的单载体表达系统。 这两种多肽可优先以3:1至15:1之间的比例，例如3:1至7:1之间的比例表达。 本发明可用于治疗儿茶酚胺缺乏症，如多巴胺缺乏症，包括但不限于帕金森病。 此外，本发明提供了递送载体构建体的方法，以限制将儿茶酚胺生产增加到有需要的细胞中。

公开(公告)号：US08945918B2

公开(公告)日：2015-02-03

申请号：US12297958

申请日：2007-08-24

Applicant: Haifeng Chen

Inventor： Haifeng Chen

IPC: C12N15/00 ,  C12N15/85 ,  C12N7/00 ,  C07K14/005

CPC classification number: C12N15/86 ,  C07K14/005 ,  C12N7/00 ,  C12N15/85 ,  C12N2750/00043 ,  C12N2750/00051 ,  C12N2750/14151 ,  C12N2750/14152 ,  C12N2800/105 ,  C12N2800/50 ,  C12N2830/42 ,  C12N2840/20

Abstract: The present teachings disclose nucleic acid cassettes for expressing in an insect cell a plurality of polypeptides encoded by a gene comprising overlapping open reading frames (ORFs). A cassette comprises, in 5′ to 3′ order, a) a first insect cell-operable promoter, b) a 5′ portion of a gene comprising a first ORF of the gene, c) an intron comprising a second insect cell-operable promoter, and d) a 3′ portion of the gene comprising at least one additional ORF. Vectors and insect cells comprising the cassettes are also disclosed, as well as methods for production of recombinant adeno-associated virus in insect cells using the cassettes.

Abstract translation: 本教导公开了用于在昆虫细胞中表达由包含重叠开放阅读框（ORFs）的基因编码的多个多肽的核酸盒。 一个盒子以5'至3'的顺序包括a）第一个昆虫细胞可操作的启动子，b）包含该基因的第一个ORF的基因的5'部分，c）包含第二个昆虫细胞可操作的启动子 启动子，和d）包含至少一个额外ORF的基因的3'部分。 还公开了包含盒的载体和昆虫细胞，以及在使用盒的昆虫细胞中产生重组腺相关病毒的方法。

公开(公告)号：US20240279679A1

公开(公告)日：2024-08-22

申请号：US18170296

申请日：2023-02-16

Applicant: The University of Hong Kong

Inventor： Martin Cheung ,  Tianyuan Shi ,  Jessica Aijia Liu

IPC: C12N15/86 ,  A61P21/00 ,  C12N15/11

CPC classification number: C12N15/86 ,  A61P21/00 ,  C12N15/11 ,  C12N2750/00043

Abstract: It has been discovered that overexpression of DLC1-i1 partly rescues neuronal formation and apoptosis of neuromuscular cells in subjects with spinal muscular atrophy (SMA). Since loss of DLC1-i1 confers specific MN defects in SMA, compositions and methods thereof for the treatment of SMA by delivery of AAV-SYN-DLC1-i1 into the neuronal population of subject with SMA are provided. The methods improve survival and restore locomotion ability to a greater extent than that of commercially-available compositions and methods. Methods including administration of both DLC1-i1 and SMN1 transgenes provide synergistic effects in improving the survival and enhancing locomotion ability in subject with SMA.

公开(公告)号：US20240011005A1

公开(公告)日：2024-01-11

申请号：US18331431

申请日：2023-06-08

Applicant: HuidaGene Therapeutics Co., Ltd.

Inventor： Xiangfeng Kong ,  Lecong Wang ,  Xuqiang Kong

IPC: C12N9/22 ,  C12N15/86

CPC classification number: C12N9/22 ,  C12N15/86 ,  C12N2310/20 ,  C12N2750/00043

Abstract: The disclosure provides Cas12f polypeptides, fusion proteins comprising such Cas12f polypeptides, CRISPR-Cas12f systems comprising such Cas12f polypeptides or fusion proteins, and methods of using the same.

公开(公告)号：US20230365991A1

公开(公告)日：2023-11-16

申请号：US18181952

申请日：2023-03-10

Applicant: AAVNERGENE INC.

Inventor： Qizhao WANG ,  Daozhan YU ,  Yu LIU ,  Cuiping CHEN ,  Rongze YANG

IPC: C12N15/86

CPC classification number: C12N15/86 ,  C12N2750/00043 ,  C12N2710/10043 ,  C12N2750/14143

Abstract: Expression constructs and methods for recombinant parvovirus production are disclosed. In some embodiments, the expression construct encodes (1) adenovirus E4 and E2a proteins, (2) parvovirus proteins necessary for the production of the recombinant parvovirus, and (3) a recombinant parvovirus genome, thus allowing production of the recombinant parvovirus by transfecting a host cell with a single expression construct.