公开(公告)号：US12129287B2

公开(公告)日：2024-10-29

申请号：US17474535

申请日：2021-09-14

Applicant: President and Fellows of Harvard College ,  The General Hospital Corporation

Inventor： David P. Corey ,  Casey A. Maguire ,  Killian S. Hanlon ,  Maryna V. Ivanchenko

IPC: C07K14/705 ,  A61K48/00 ,  A61P27/16 ,  C12N15/86

CPC classification number: C07K14/705 ,  A61K48/0058 ,  A61P27/16 ,  C12N15/86 ,  C12N2750/14143 ,  C12N2750/14171 ,  C12N2800/90 ,  C12N2840/105

Abstract: Aspects of the disclosure relate to compositions and methods for treating hereditary hearing loss and/or vision loss, for example, due to Usher syndrome, Type 3A. In some embodiments, the disclosure provides a recombinant adeno-associated virus comprising: (i) an AAV-S capsid protein, and (ii) an isolated nucleic acid comprising a transgene (e.g., a transgene for expressing a clarin-1 protein). The present disclosure also provides methods of treating hereditary hearing loss and/or vision loss (e.g., Usher Syndrome, Type 3A) using the same.

公开(公告)号：US12018253B2

公开(公告)日：2024-06-25

申请号：US16495677

申请日：2018-03-23

Applicant: IMBA—INSTITUT FÜR MOLEKULARE BIOTECHNOLOGIE GMBH

Inventor： Oliver Bell ,  Hagar Moussa ,  Ramesh Yelagandula

IPC: C12N15/85 ,  C12N15/10 ,  G01N33/50

CPC classification number: C12N15/1086 ,  C12N15/85 ,  G01N33/5023 ,  C12N2015/859 ,  C12N2800/107 ,  C12N2800/24 ,  C12N2840/002 ,  C12N2840/005 ,  C12N2840/105 ,  C12N2840/85

Abstract: A method of detecting Polycomb Repressive Complex (PRC) activity in a cell providing a cell with a DNA having a protein binding site and at least one reporter gene expression site is operatively connected to the protein binding site, and with a DNA containing a recombinant gene of a binding protein, the binding protein being capable of binding to the protein binding site, wherein the binding protein is fused to a member of the PRC, the method including the step of expressing the recombinant gene, letting the fused binding protein bind to the protein binding site and detecting at least one reporter gene expression.

公开(公告)号：US11845939B2

公开(公告)日：2023-12-19

申请号：US15831230

申请日：2017-12-04

Applicant: Janssen Pharmaceuticals, Inc.

Inventor： Kurt Iver Kamrud ,  Maung Nyan Win ,  Nathaniel Stephen Wang ,  Jason L. DeHart

IPC: C12N15/63 ,  C12N15/86 ,  A61K48/00 ,  C12N15/113 ,  C12N15/85

CPC classification number: C12N15/63 ,  A61K48/005 ,  C12N15/113 ,  C12N15/86 ,  C07K2319/50 ,  C12N2015/8518 ,  C12N2310/531 ,  C12N2770/00042 ,  C12N2770/36062 ,  C12N2770/36111 ,  C12N2770/36143 ,  C12N2830/30 ,  C12N2840/105

Abstract: The present disclosure generally relates to nucleic acid molecules for use in regulating gene expression. Disclosed herein include nucleic acid molecules containing one or more structural elements of the viral capsid enhancer operably linked to a coding sequence of a gene of interest. In some embodiments, the viral capsid enhancer comprises a Downstream Loop (DLP) from a viral capsid protein, or a variant of the DLP.

公开(公告)号：US20180353625A1

公开(公告)日：2018-12-13

申请号：US16106912

申请日：2018-08-21

Applicant: Vrije Universiteit Brussel

Inventor： Carlo Heirman ,  Kristiaan Thielemans

IPC: A61K48/00 ,  C07K14/705 ,  A61K39/00 ,  A61K31/7105 ,  C12N15/85

CPC classification number: A61K48/0066 ,  A61K31/7105 ,  A61K39/0011 ,  A61K2039/53 ,  A61K2039/54 ,  A61K2039/55516 ,  C07K14/70575 ,  C07K14/70596 ,  C12N15/85 ,  C12N2320/52 ,  C12N2830/50 ,  C12N2840/105

Abstract: The present invention in general relates to an improved RNA transcription vector, which is very suitable for the production of mRNA for in vivo therapeutic purposes. The improvements in the vector in particular reside in the presence of a transcription enhancer and a nuclear retention element.

公开(公告)号：US20170298350A1

公开(公告)日：2017-10-19

申请号：US15515834

申请日：2015-10-03

Applicant: HocusLocus, LLC ,  Universität für Bodenkultur

Inventor： Edward Eveleth ,  Nicole Borth

IPC: C12N15/11 ,  C12N5/10 ,  C12N15/85

CPC classification number: C12N15/111 ,  C12N5/10 ,  C12N15/85 ,  C12N2310/141 ,  C12N2320/10 ,  C12N2330/51 ,  C12N2800/24 ,  C12N2840/102 ,  C12N2840/105 ,  C12N2999/007

Abstract: Described herein are methods and compositions for producing a gene of interest (GOD, which, in certain embodiments, can reduce the metabolic burden on cells and reduce decoupling of GOI production from marker production, as compared to prior art methods. The methods relate to positive selection and negative selection approaches to establishing high GOI-producing cell lines, e.g., CHO lines. In certain embodiments, the methods comprise transfecting a cell with (a) an oligonucleotide comprising a GOI and a non-coding RNA, and (b) an oligonucleotide encoding a selection protein; wherein the non-coding RNA promotes or inhibits production of the selection protein. The cell producing the can be identified and/or selected as a result of or by detecting the absence or the presence of the selection protein.

公开(公告)号：US09644205B2

公开(公告)日：2017-05-09

申请号：US14396967

申请日：2013-04-24

Applicant: The Regents of the University of California ,  BAYLOR COLLEGE OF MEDICINE

Inventor： Francis C. Brunicardi ,  Shi-He Liu

IPC: C07H21/04 ,  C12N15/63 ,  C12N15/113 ,  C12N15/11 ,  C12N15/85 ,  C12N15/70 ,  C12N15/81

CPC classification number: C12N15/113 ,  C12N15/111 ,  C12N15/70 ,  C12N15/81 ,  C12N15/85 ,  C12N2330/51 ,  C12N2830/008 ,  C12N2840/105

Abstract: The present invention provides nucleic acid constructs, expression vectors, transgenic cell and methods of making and using the same, wherein the nucleic acid construct includes a synthetic promoter designed using selected PDX-1 activation sites such as those observed in the human insulin promoter (HIP). In illustrative working embodiments of the invention, an exogenous nucleic acid fragment encoding thymidine kinase is operably linked to the synthetic promoter which is then shown to regulate the expression of this polypeptide.

公开(公告)号：US20160279266A1

公开(公告)日：2016-09-29

申请号：US15034048

申请日：2014-11-12

Applicant: Vrije Universiteit Brussel

Inventor： Carlo HEIRMAN ,  Kristiaan THIELEMANS

IPC: A61K48/00 ,  C07K14/705 ,  A61K39/00 ,  C12N15/85

CPC classification number: A61K48/0066 ,  A61K31/7105 ,  A61K39/0011 ,  A61K2039/53 ,  A61K2039/54 ,  A61K2039/55516 ,  C07K14/70575 ,  C07K14/70596 ,  C12N15/85 ,  C12N2320/52 ,  C12N2830/50 ,  C12N2840/105

Abstract: The present invention relates to an improved RNA transcription vector, which is very suitable for the production of mRNA for in vivo therapeutic purposes. The improvements in the vector reside in the presence of a translation enhancer (TE) and a nuclear retention element (NRS), especially when the latter is the “Expression and Nuclear Retention Element” (ENE) of Kaposi's sarcoma associated Herpes virus (KSHV).

Abstract translation: 本发明涉及一种改进的RNA转录载体，其非常适合用于体内治疗目的的mRNA的产生。 载体中的改进位于翻译增强子（TE）和核保留元件（NRS）的存在下，特别是当后者是卡波西肉瘤相关疱疹病毒（KSHV）的“表达和核保留元件”（ENE）时， 。

公开(公告)号：US09249201B2

公开(公告)日：2016-02-02

申请号：US14134169

申请日：2013-12-19

Applicant: Barbara Enenkel

Inventor： Barbara Enenkel

IPC: C12P21/02 ,  C07K14/47 ,  C12N15/62 ,  C12N15/63 ,  C12N15/67 ,  C12N15/85 ,  C07K7/08 ,  C07K16/00

CPC classification number: C07K14/47 ,  C07K7/08 ,  C07K16/00 ,  C07K2317/14 ,  C07K2317/52 ,  C07K2319/00 ,  C12N15/625 ,  C12N15/63 ,  C12N15/67 ,  C12N15/85 ,  C12N2830/42 ,  C12N2840/105

Abstract: The invention concerns the field of recombinant gene engineering. It concerns novel introns and compositions comprising such introns as well as a method to improve expression of polypeptides from nucleic acids such as cloned genes with heterologous introns, especially genes encoding antibodies and antibody derived fragments, and the production of various polypeptides in eukaryotic host cells using said novel intron sequences as heterologous introns.

Abstract translation: 本发明涉及重组基因工程领域。 它涉及包含这种内含子的新颖内含子和组合物以及改善来自核酸例如具有异源内含子的克隆基因，特别是编码抗体和抗体衍生片段的基因的多肽表达的多肽的方法，以及在真核宿主细胞中使用 所述新型内含子序列作为异源内含子。

公开(公告)号：US20100034775A1

公开(公告)日：2010-02-11

申请号：US12520615

申请日：2006-12-20

Applicant: Qingjun Xie

Inventor： Qingjun Xie

IPC: A61K35/76 ,  C12N15/00 ,  C12N7/00 ,  C12N15/87

CPC classification number: C12N15/86 ,  A61K38/00 ,  C07K14/4746 ,  C12N7/00 ,  C12N2710/10332 ,  C12N2710/10343 ,  C12N2830/005 ,  C12N2840/105

Abstract: A recombinant adenovirus vector comprises eukaryotic cell promoter, operator, Kozak sequence, exogenous gene and PolyA signal sequence from 5′ to 3′ in the adenovirus vector backbone. Optionally, introns are inserted between the operator and Kozak sequence, and the exogenous gene is p53 gene. A recombinant adenovirus obtained by co-transfection of the adenovirus vector and adenovirus backbone is also included in the invention. Also disclosed are the preparation methods and uses of the recombinant adenovirus vector and the recombinant adenovirus, respectively. The present invention further discloses a pharmaceutical composition for treating cancers, which comprises the recombinant adenovirus.

Abstract translation: 重组腺病毒载体包含真核细胞启动子，操纵子，Kozak序列，外源基因和腺苷病毒载体骨架中5'至3'的PolyA信号序列。 任选地，将内含子插入操作者和Kozak序列之间，外源基因是p53基因。 通过共转染腺病毒载体和腺病毒骨架获得的重组腺病毒也包括在本发明中。 还公开了重组腺病毒载体和重组腺病毒的制备方法和用途。 本发明还公开了一种用于治疗癌症的药物组合物，其包含重组腺病毒。

公开(公告)号：US07351818B2

公开(公告)日：2008-04-01

申请号：US11167614

申请日：2005-06-27

Applicant: Herve Jean-Clement Coste ,  Jonathan Henry Ellis

Inventor： Herve Jean-Clement Coste ,  Jonathan Henry Ellis

IPC: C07H21/04

CPC classification number: C07K14/47 ,  A61K2039/53 ,  C12N15/85 ,  C12N2830/002 ,  C12N2830/85 ,  C12N2840/105 ,  C12N2840/203

Abstract: Untranslated regions associated with the heat shock response can be used to obtain increased efficiency of translation of polypeptides that are not necessarily normally associated with the heat shock response. This allows the development of greatly improved expression systems. The invention is also useful, for example, in the treatment of a patient suffering from a deficiency in the expression of a polypeptide and in the provision of vaccines.

Abstract translation: 可以使用与热休克反应相关的非翻译区域，以获得不一定通常与热休克反应相关的多肽的翻译效率。 这允许开发大大改进的表达系统。 本发明也可用于例如治疗患有多肽表达缺陷的患者和提供疫苗。