公开(公告)号：US20160244754A1

公开(公告)日：2016-08-25

申请号：US15016096

申请日：2016-02-04

Applicant: California Institute of Technology

Inventor： David Baltimore ,  Ryan M. O'Connell ,  Konstantin Taganov ,  Mark Boldin

IPC: C12N15/113 ,  A61K45/06 ,  A61K31/7105

CPC classification number: C12N15/113 ,  A61K31/56 ,  A61K31/7105 ,  A61K45/06 ,  C12N2310/11 ,  C12N2310/113 ,  C12N2310/141 ,  C12N2320/31

Abstract: The present disclosure relates to the finding that microRNA-155 plays a role in inflammation, hematopoiesis and myeloproliferation, and that dysregulation of microRNA-155 expression is associated with particular myeloproliferative disorders. Disclosed herein are methods and compositions for diagnosing an treating disorders, including inflammation and myeloproliferation, modulating the levels of expression of one or more genes selected from the group consisting of Cutl1, Arnt1, Picalm, Jarid2, PU.1, Csf1r, HIF1α, Sla, Cepbβ, and Bach1, and the like.

Abstract translation: 本公开涉及microRNA-155在炎症，造血和骨髓增生中起作用的发现，并且微小RNA-155表达的调节异常与特定的骨髓增生性疾病相关。 本文公开了用于诊断治疗疾病（包括炎症和骨髓增生）的方法和组合物，调节选自Cutl1，Arnt1，Picalm，Jarid2，PU.1，Csf1r，HIF1α，Sla的一种或多种基因的表达水平 ，Cepbβ和Bach1等。

公开(公告)号：US20150218564A1

公开(公告)日：2015-08-06

申请号：US14695462

申请日：2015-04-24

Applicant: CALIFORNIA INSTITUTE OF TECHNOLOGY ,  The Regents of the University of California

Inventor： Carlos Lois-Caballe ,  David Baltimore ,  Xiao-Feng Qin ,  Irvin S.Y. Chen ,  Dong Sung An

IPC: C12N15/63

CPC classification number: C12N15/1132 ,  A61K48/00 ,  C12N15/111 ,  C12N15/113 ,  C12N15/1138 ,  C12N15/63 ,  C12N15/86 ,  C12N2310/111 ,  C12N2310/14 ,  C12N2330/30 ,  C12N2740/16043 ,  C12N2740/16045 ,  C12N2760/20122 ,  C12N2799/027 ,  C12N2810/609 ,  C12N2830/003 ,  C12N2830/008 ,  C12N2830/48 ,  C12N2840/203

Abstract: In one aspect, the invention provides methods and compositions for the expression of small RNA molecules within a cell using a retroviral vector (FIG. 1A). Small interfering RNA (siRNA) can be expressed using the methods of the invention within a cell. In a further aspect, the invention provides methods for producing siRNA encoding lentivirus where the siRNA activity may interfere with the lentiviral life cycle. In yet a further aspect, the invention provides methods for expression of a small RNA molecule within a cell, such as an siRNA capable of downregulating CCR5, wherein expression of the small RNA molecule is relatively non-cytotoxic to the cell. The invention also includes small RNA molecules, such as an siRNA capable of downregulating CCR5, that are relatively non-cytotoxic to cells.

Abstract translation: 一方面，本发明提供了使用逆转录病毒载体在细胞内表达小RNA分子的方法和组合物（图1A）。 可以使用本发明的方法在细胞内表达小干扰RNA（siRNA）。 另一方面，本发明提供了用于产生编码慢病毒的siRNA的方法，其中siRNA活性可能干扰慢病毒生命周期。 在另一方面，本发明提供了在细胞内表达小RNA分子的方法，例如能够下调CCR5的siRNA，其中小RNA分子的表达对细胞是相对非细胞毒性的。 本发明还包括对细胞相对非细胞毒性的小RNA分子，例如能够下调CCR5的siRNA。

公开(公告)号：US20140220049A1

公开(公告)日：2014-08-07

申请号：US14138472

申请日：2013-12-23

Applicant: California Institute of Technology

Inventor： David Baltimore ,  Ryan M. O'Connell ,  Konstantin Taganov ,  Mark Boldin

IPC: C12N15/113 ,  A61K31/56 ,  A61K45/06

CPC classification number: C12N15/113 ,  A61K31/56 ,  A61K31/7105 ,  A61K45/06 ,  C12N2310/11 ,  C12N2310/113 ,  C12N2310/141 ,  C12N2320/31

Abstract: The present disclosure relates to the finding that microRNA-155 plays a role in inflammation, hematopoiesis and myeloproliferation, and that dysregulation of microRNA-155 expression is associated with particular myeloproliferative disorders. Disclosed herein are methods and compositions for diagnosing an treating disorders, including inflammation and myeloproliferation, modulating the levels of expression of one or more genes selected from the group consisting of Cutl1, Arntl, Picalm, Jarid2, PU.1, Csflr, HIFlα, Sla, Cepbβ, and Bach1, and the like.

Abstract translation: 本公开涉及microRNA-155在炎症，造血和骨髓增生中起作用的发现，并且微小RNA-155表达的调节异常与特定的骨髓增生性疾病相关。 本文公开了用于诊断治疗疾病（包括炎症和骨髓增生）的方法和组合物，调节选自Cutl1，Arnt1，Picalm，Jarid2，PU.1，Csflr，HIF1α，Sla的一种或多种基因的表达水平 ，Cepb＆bgr，和Bach1等。

公开(公告)号：US20140127162A1

公开(公告)日：2014-05-08

申请号：US14067786

申请日：2013-10-30

Applicant: California Institute of Technology

Inventor： Alejandro Benjamin Balazs ,  David Baltimore

IPC: A61K48/00 ,  C12N15/86

CPC classification number: A61K48/0066 ,  C12N15/86 ,  C12N2750/14143 ,  C12N2800/30 ,  C12N2800/40 ,  C12N2830/001 ,  C12N2830/42 ,  C12N2840/44

Abstract: According to some embodiments herein, expression systems and methods for reversible gene expression are provided. In some embodiments, adeno-associated viral vectors encoding a gene product of interest and comprising a plurality of recombinase target sites are provided. In some embodiments, a source of recombinase is provided. In some embodiments, the gene product of interest is expressed, and the recombinase then induces recombination events between the recombinase target sites, thus reducing or eliminating expression of the gene product of interest.

Abstract translation: 根据本文的一些实施方案，提供了可逆基因表达的表达系统和方法。 在一些实施方案中，提供编码感兴趣的基因产物并且包含多个重组酶靶位点的腺相关病毒载体。 在一些实施方案中，提供了重组酶的来源。 在一些实施方案中，表达目的基因产物，然后重组酶诱导重组酶靶位点之间的重组事件，从而减少或消除感兴趣的基因产物的表达。

公开(公告)号：US12221465B2

公开(公告)日：2025-02-11

申请号：US16122562

申请日：2018-09-05

Applicant: California Institute of Technology

Inventor： Alok V Joglekar ,  Michael T Leonard ,  Michael T Bethune ,  David Baltimore

IPC: C07K14/705 ,  A61K39/00 ,  A61P37/00 ,  C07K14/725 ,  C07K14/74 ,  C12N5/0783 ,  C40B40/02 ,  G01N33/566 ,  G01N33/68 ,  A61K38/00

Abstract: Described herein are compositions and methods for signaling and antigen-presenting bifunctional receptors (SABRs) comprising one or more antigen presenting domains; and one or more signal transduction domains, wherein the one or more antigen presenting domains comprise a binding fragment of a major histocompatibility complex (MHC) molecule. Various immunological functions of the SABRs are also described.

公开(公告)号：US20180230491A1

公开(公告)日：2018-08-16

申请号：US15944562

申请日：2018-04-03

Applicant: California Institute of Technology

Inventor： Carlos Lois-Caballe ,  David Baltimore ,  Xiao-Feng Qin

IPC: C12N15/86 ,  C12N15/113 ,  C12N7/00

CPC classification number: C12N15/86 ,  A61K38/00 ,  A61K48/00 ,  C12N7/00 ,  C12N15/113 ,  C12N15/1132 ,  C12N15/1138 ,  C12N15/867 ,  C12N2310/111 ,  C12N2310/14 ,  C12N2310/53 ,  C12N2310/531 ,  C12N2330/30 ,  C12N2740/15043 ,  C12N2740/16043 ,  C12N2830/003 ,  C12N2830/006 ,  C12N2830/008 ,  C12N2830/30 ,  C12N2830/48 ,  C12N2830/60 ,  C12N2830/85 ,  C12N2840/20 ,  C12N2840/203 ,  C12Q1/6897 ,  Y02A50/465

Abstract: The invention provides methods and compositions for the expression of small RNA molecules within a cell using a lentiviral vector. The methods can be used to express doubles stranded RNA complexes. Small interfering RNA (siRNA) can be expressed using the methods of the invention within a cell, which is capable of down regulating the expression of a target gene through RNA interference. A variety of cells can be treated according to the methods of the invention including embryos, embryogenic stem cells, allowing for the generation of transgenic animals or animals constituted partly by the transduced cells that have a specific gene or a group of genes down regulated.

公开(公告)号：US10041072B2

公开(公告)日：2018-08-07

申请号：US15395348

申请日：2016-12-30

Applicant: California Institute of Technology

Inventor： Carlos Lois-Caballe ,  David Baltimore ,  Xiao-Feng Qin ,  Irvin S. Y. Chen ,  Dong Sung An

IPC: C12N15/113 ,  C12N15/867 ,  A61K48/00 ,  A61P31/18 ,  C12N15/86 ,  C12N15/11

Abstract: In one aspect, the invention provides methods and compositions for the expression of small RNA molecules within a cell using a retroviral vector (FIG. 1A). Small interfering RNA (siRNA) can be expressed using the methods of the invention within a cell. In a further aspect, the invention provides methods for producing siRNA encoding lentivirus where the siRNA activity may interfere with the lentiviral life cycle. In yet a further aspect, the invention provides methods for expression of a small RNA molecule within a cell, such as an siRNA capable of downregulating CCR5, wherein expression of the small RNA molecule is relatively non-cytotoxic to the cell. The invention also includes small RNA molecules, such as an siRNA capable of downregulating CCR5, that are relatively non-cytotoxic to cells.

公开(公告)号：US20180179555A1

公开(公告)日：2018-06-28

申请号：US15820741

申请日：2017-11-22

Applicant: CALIFORNIA INSTITUTE OF TECHNOLOGY

Inventor： Pin Wang ,  Lili Yang ,  David Baltimore

IPC: C12N15/86 ,  A61K38/20 ,  A61K35/76 ,  A61K38/17 ,  A61K38/19 ,  C12N7/00 ,  C07K14/005 ,  A61K39/21 ,  A61K31/70 ,  A61K39/00

CPC classification number: C12N15/86 ,  A61K31/70 ,  A61K35/76 ,  A61K38/177 ,  A61K38/1774 ,  A61K38/178 ,  A61K38/1793 ,  A61K38/191 ,  A61K38/193 ,  A61K38/20 ,  A61K38/2013 ,  A61K38/2026 ,  A61K38/204 ,  A61K38/2046 ,  A61K38/2086 ,  A61K39/21 ,  A61K2039/5256 ,  C07K14/005 ,  C12N7/00 ,  C12N2740/13043 ,  C12N2740/13045 ,  C12N2740/15041 ,  C12N2740/15043 ,  C12N2740/15045 ,  C12N2770/36122 ,  C12N2770/36134 ,  C12N2810/609 ,  C12N2810/855 ,  Y02A50/386 ,  Y02A50/396 ,  Y02A50/401 ,  Y02A50/403 ,  Y02A50/407 ,  Y02A50/41 ,  Y02A50/412 ,  Y02A50/423 ,  Y02A50/466 ,  Y02A50/48 ,  Y02A50/489 ,  Y02A50/492 ,  A61K2300/00

Abstract: Methods and compositions are provided for delivery of a polynucleotide encoding a gene of interest, typically an antigen, to a dendritic cell (DC). The virus envelope comprises a DC-SIGN specific targeting molecule. The methods and related compositions can be used to treat patients suffering from a wide range of conditions, including infection, such as HIV/AIDS, and various types of cancers.

公开(公告)号：US09840721B2

公开(公告)日：2017-12-12

申请号：US15051907

申请日：2016-02-24

Applicant: CALIFORNIA INSTITUTE OF TECHNOLOGY

Inventor： Pin Wang ,  Lili Yang ,  David Baltimore

IPC: A01N63/00 ,  C12N15/86 ,  A61K31/70 ,  A61K38/17 ,  A61K38/19 ,  A61K38/20 ,  C07K14/005 ,  A61K35/76 ,  A61K39/21 ,  C12N7/00 ,  A61K39/00

CPC classification number: C12N15/86 ,  A61K31/70 ,  A61K35/76 ,  A61K38/177 ,  A61K38/1774 ,  A61K38/178 ,  A61K38/1793 ,  A61K38/191 ,  A61K38/193 ,  A61K38/20 ,  A61K38/2013 ,  A61K38/2026 ,  A61K38/204 ,  A61K38/2046 ,  A61K38/2086 ,  A61K39/21 ,  A61K2039/5256 ,  C07K14/005 ,  C12N7/00 ,  C12N2740/13043 ,  C12N2740/13045 ,  C12N2740/15041 ,  C12N2740/15043 ,  C12N2740/15045 ,  C12N2770/36122 ,  C12N2770/36134 ,  C12N2810/609 ,  C12N2810/855 ,  Y02A50/386 ,  Y02A50/396 ,  Y02A50/401 ,  Y02A50/403 ,  Y02A50/407 ,  Y02A50/41 ,  Y02A50/412 ,  Y02A50/423 ,  Y02A50/466 ,  Y02A50/475 ,  Y02A50/48 ,  Y02A50/481 ,  Y02A50/492 ,  A61K2300/00

Abstract: Methods and compositions are provided for delivery of a polynucleotide encoding a gene of interest, typically an antigen, to a dendritic cell (DC). The virus envelope comprises a DC-SIGN specific targeting molecule. The methods and related compositions can be used to treat patients suffering from a wide range of conditions, including infection, such as HIV/AIDS, and various types of cancers.

公开(公告)号：US20170192011A1

公开(公告)日：2017-07-06

申请号：US15301930

申请日：2015-04-03

Applicant: The Board of Trustees of the Leland Stanford Junior University ,  CALIFORNIA INSTITUTE OF TECHNOLOGY

Inventor： Michael Edward Birnbaum ,  Juan Luis Mendoza ,  Michael Thomas Bethune ,  David Baltimore ,  Kenan Christopher Garcia

IPC: G01N33/68 ,  C07K14/00

CPC classification number: G01N33/6845 ,  C07K14/00 ,  G01N2333/7051 ,  G01N2333/70539

Abstract: Compositions and methods are provided for the identification of peptide sequences that are ligands for a T cell receptor (TCR) of interest, in a given MHC context.