公开(公告)号：US20180346899A1

公开(公告)日：2018-12-06

申请号：US16054382

申请日：2018-08-03

Applicant: Whitehead Institute for Biomedical Research

Inventor： Giulia Pasqual ,  Gabriel Victora

IPC: C12N9/52 ,  A01K67/027 ,  G01N33/50

CPC classification number: C12N9/52 ,  A01K67/027 ,  A01K67/0275 ,  A01K2217/072 ,  A01K2227/105 ,  A01K2267/03 ,  A01K2267/0387 ,  C07K2319/70 ,  C12Y304/2207 ,  G01N33/5008 ,  G01N33/5047 ,  G01N2333/70532 ,  G01N2333/70578

Abstract: An sortase-mediated intercellular labeling method allowing for tracking ligand-receptor interaction both in vitro and in vivo; and uses thereof for tracking molecule interactions both in vitro and in vivo, identifying modulators of ligand-receptor interaction, identifying potential binding partners of a protein of interest, identifying B cells expressing high affinity B cell receptors to antigens, and identifying the antigen to which a T cell of interest binds.

公开(公告)号：US20180318322A1

公开(公告)日：2018-11-08

申请号：US15775358

申请日：2016-11-10

Applicant: CITY OF HOPE

Inventor： Defu ZENG

IPC: A61K31/675 ,  A61K31/7056 ,  C07K16/28 ,  A61K35/17 ,  A61K35/14 ,  A61K35/26 ,  A61K35/28 ,  A61P37/06

CPC classification number: A61K31/675 ,  A01K2207/20 ,  A01K2227/105 ,  A01K2267/0387 ,  A61K31/00 ,  A61K31/7056 ,  A61K35/14 ,  A61K35/17 ,  A61K35/26 ,  A61K35/28 ,  A61P37/06 ,  C07K16/2809

Abstract: Disclosed herein are conditioning regimens and methods for inducing MHC- or HLA-mismatched mixed chimerism by conditioning a recipient with radiation-free, low-doses of cyclophosphamide (CY), pentostatin (PT), and anti-thymocyte globulin (ATG) prior to transplantation of donor bone marrow cells. In certain embodiments, the donor bone marrow cells may be CD4+ T-depleted bone marrow cells. The conditioning regimens and methods may also include administering one or more populations of conditioning donor cells selected from donor CD4+ T-depleted spleen cells, donor CD8+ T cells, and donor G-CSF-mobilized peripheral blood mononuclear cells. The conditioning regimen is clinically acceptable and can be used for treating hereditary hematological diseases and autoimmune diseases, as well as for promoting organ transplantation immune tolerance.

公开(公告)号：US20180305714A1

公开(公告)日：2018-10-25

申请号：US15768482

申请日：2016-10-14

Applicant: ASTRAZENECA AB

Inventor： Marcello MARESCA ,  Mohammad BOHLOOLY-YEGANEH ,  Himjyot JAISWAL ,  Lorenz M. MAYR ,  Xiufeng XU

IPC: C12N15/85 ,  A01K67/027 ,  C12N15/86

CPC classification number: C12N15/8509 ,  A01K67/0275 ,  A01K2217/072 ,  A01K2217/075 ,  A01K2217/203 ,  A01K2217/206 ,  A01K2227/105 ,  A01K2267/0393 ,  C12N15/111 ,  C12N15/79 ,  C12N15/86 ,  C12N15/907 ,  C12N2015/8572 ,  C12N2310/20

Abstract: The present disclosure is directed, in some embodiments, to compositions and methods for inducible modification of a cell genome.

公开(公告)号：US20180272001A1

公开(公告)日：2018-09-27

申请号：US15765431

申请日：2016-10-18

Applicant: University of Massachusetts

Inventor： Jack L. Leonard ,  Karl J. Simin ,  Deborah M. Leonard

IPC: A61K48/00 ,  C07K14/47 ,  A61P35/00

CPC classification number: A61K48/005 ,  A01K67/0275 ,  A01K2217/072 ,  A01K2227/105 ,  A01K2267/0331 ,  A61K31/337 ,  A61K31/351 ,  A61K33/24 ,  A61K2300/00 ,  A61P35/00 ,  C07K14/4703 ,  C12N15/86

Abstract: The invention relates to the discovery of a vital new component of the Wnt pathway that regulates trafficking of β-catenin to the cell nucleus and novel therapeutic approaches to cancer treatment. Disclosed herein is a previously unknown, essential component of the Wnt/β-catenin signaling pathway that governs the quantity of β-catenin delivered to the cell nucleus. This intracellular inhibitor of β-catenin signaling (IBS) is transcribed from a second transcriptional start site adjacent to exon 3 of the Dkk3 gene and is required for early mouse development. IBS captures β-catenin destined for the nucleus in a complex with β-TrCP that is bound to the actin cytoskeleton and unavailable for nuclear translocation.

公开(公告)号：US10080354B2

公开(公告)日：2018-09-25

申请号：US14426482

申请日：2013-09-06

Applicant: Children's Medical Center Corporation

Inventor： Derrick J. Rossi ,  Roi Gazit

IPC: A01K67/027 ,  A61K48/00 ,  C07H21/04 ,  C12Q1/6897 ,  G01N33/50

CPC classification number: A01K67/0278 ,  A01K67/0275 ,  A01K2217/072 ,  A01K2227/105 ,  A01K2267/02 ,  C07H21/04 ,  C12N2517/02 ,  C12Q1/6897 ,  G01N33/5073

Abstract: Described herein are nucleic acid constructs, hematopoietic stem cell identifier animals, and methods of using thereof for isolating hematopoietic stem cell populations. Also provided are methods of using the identifier animals and cells isolated from them to screen for agents that affect the growth, proliferation, potency, expansion, or maintenance of the stem cells. Such agents can be used for promoting growth of stem cells in vitro or in vivo, and also for inhibiting cancer cells that have been determined to resemble a stem cell.

公开(公告)号：US20180264038A1

公开(公告)日：2018-09-20

申请号：US15764187

申请日：2016-09-28

Applicant: Regents of the University of Minnesota

Inventor： Bruce R. Blazar ,  Ryan P. Flynn ,  Christopher A. Pennell

IPC: A61K35/17 ,  C07K16/28 ,  A61K48/00 ,  A61K9/00

CPC classification number: A61K35/17 ,  A01K2207/15 ,  A01K2227/105 ,  A01K2267/0387 ,  A61K9/0019 ,  A61K48/005 ,  A61P37/06 ,  C07K16/2803 ,  C07K2317/622 ,  C07K2319/03 ,  C07K2319/33 ,  C12N2740/10043

Abstract: Provided herein are methods and materials for treating autoimmune diseases and alloimmune diseases. Specifically, provided are a pharmaceutical composition comprising a therapeutically effective amount of a population of modified human T cells, wherein the human T cells are modified to comprise a nucleic acid sequence that encodes a chimeric antigen receptor (CAR) construct, wherein the CAR construct comprises an antigen binding domain, wherein the antigen binding domain is specific for a ligand expressed on B cells, plasma cells or plasmablasts in human patients suffering from an autoimmune disease or an alloimmune disease; and a method of treating an autoimmune or an alloimmune disease in a human patient, the method comprising: administering a pharmaceutical composition.

公开(公告)号：US20180255754A1

公开(公告)日：2018-09-13

申请号：US15905068

申请日：2018-02-26

Applicant: Regeneron Pharmaceuticals, Inc.

Inventor： Susannah Brydges ,  Yajun Tang ,  Yang Liu ,  Jingtai Cao ,  Carmelo Romano

IPC: A01K67/027 ,  C12N15/85

CPC classification number: A01K67/0276 ,  A01K67/0278 ,  A01K2217/054 ,  A01K2217/072 ,  A01K2217/075 ,  A01K2217/15 ,  A01K2217/206 ,  A01K2227/105 ,  A01K2267/0306 ,  A01K2267/0393 ,  C07K14/47 ,  C12N15/8509 ,  C12N2015/8527 ,  C12N2015/8536 ,  C12N2510/00 ,  C12N2800/30 ,  C12N2830/008 ,  G01N33/5088 ,  G01N33/6893

Abstract: Non-human animals suitable for use as animal models for Retinoschisis are provided. In some embodiments, provided non-human animals are characterized by a disruption in a Retinoschisin-1 locus. In some embodiments, provided non-human animals are characterized by a mutant Retinoschisin-1 gene. The non-human animals may be described, in some embodiments, as having a phenotype that includes the development of one or more symptoms or phenotypes associated with Retinoschisis. Methods of identifying therapeutic candidates that may be used to prevent, delay or treat Retinoschisis or eye-related diseases, disorders or conditions are also provided.

公开(公告)号：US10070632B2

公开(公告)日：2018-09-11

申请号：US15442857

申请日：2017-02-27

Applicant: REGENERON PHARMACEUTICALS, INC.

Inventor： Lisa Purcell ,  Alexander O. Mujica ,  Yajun Tang

IPC: A01K67/027 ,  C12N5/0735 ,  C12N9/64

CPC classification number: A01K67/0275 ,  A01K67/0278 ,  A01K2207/15 ,  A01K2217/072 ,  A01K2227/10 ,  A01K2227/105 ,  A01K2267/0337 ,  A61K48/00 ,  C07K14/47 ,  C07K2319/00 ,  C12N5/0606 ,  C12N9/6424 ,  C12N15/09 ,  C12N15/63 ,  C12N15/86 ,  C12N2710/10332

Abstract: Genetically modified rodents such as mice and rats, and methods and compositions for making and using the same, are provided. The rodents comprise a humanization of at least one endogenous rodent Tmprss gene, such as an endogenous rodent Tmprss2, Tmprss4, or Tmprss11d gene.

公开(公告)号：US20180243446A1

公开(公告)日：2018-08-30

申请号：US15756837

申请日：2016-09-01

Applicant: THE HOSPITAL FOR SICK CHILDREN

Inventor： Ronald Cohn ,  Daria Wojtal ,  Dwi Kemaladewi ,  Zhenya Ivakine

IPC: A61K48/00 ,  A01K67/027 ,  A61K38/46 ,  C12N15/113 ,  C12N15/90 ,  C12N9/22

CPC classification number: A61K48/0066 ,  A01K67/0275 ,  A01K2217/072 ,  A01K2227/105 ,  A01K2267/0306 ,  A61K31/713 ,  A61K38/46 ,  A61K48/0016 ,  A61P21/00 ,  C07K19/00 ,  C12N9/22 ,  C12N15/113 ,  C12N15/907 ,  C12N2310/20 ,  C12N2740/16043

Abstract: In one aspect, the Invention relates to methods and compositions to remove replicate genetic material, such as duplicate copy number variations (CNVs) present in genetic material using targeted endonuclease technology with one guide, such as one single guide RNA. In another aspect, the invention relates to methods and compositions useful for removing replicate or duplicate genetic material. In another aspect the invention is useful for the treatment and prevention of conditions that are caused by replicate genetic material, including duplicate CNVs, such as a certain types of Duchenne Muscular Dystrophy and or MECP2 duplication syndrome. In another aspect the invention provides novel genetically engineered animals with duplicate CNVs useful to screen potential therapies.

公开(公告)号：US10060933B2

公开(公告)日：2018-08-28

申请号：US14935024

申请日：2015-11-06

Applicant: UNIVERSITE LAVAL

Inventor： Jean-Pierre Julien ,  Vivek Swarup

IPC: A61K49/00 ,  G01N33/53 ,  A61B5/00 ,  A61K39/395 ,  A61K39/00 ,  A61K38/00 ,  C07K14/00 ,  C07K7/00 ,  G01N33/68 ,  C12Q1/6883 ,  G01N33/50 ,  C12N15/113 ,  A61K31/58 ,  A61K31/7088 ,  A01K67/027 ,  C07K14/47 ,  C12N15/85 ,  C07K16/18

CPC classification number: G01N33/6896 ,  A01K67/0275 ,  A01K2217/052 ,  A01K2227/105 ,  A01K2267/0312 ,  A01K2267/0318 ,  A61K31/58 ,  A61K31/7088 ,  C07K14/4702 ,  C07K16/18 ,  C07K2317/76 ,  C12N15/113 ,  C12N15/8509 ,  C12N2310/14 ,  C12Q1/6883 ,  C12Q2600/112 ,  C12Q2600/158 ,  G01N33/502 ,  G01N2333/4703 ,  G01N2333/4704 ,  G01N2500/00 ,  G01N2800/28 ,  G01N2800/2835 ,  G01N2800/50 ,  G01N2800/56

Abstract: The present invention provides methods and uses for the diagnostic of a subject predisposed or suspected of developing a neurodegenerative disease or suffering from a neurodegenerative disease. The present invention also relates to methods and uses for identifying candidate compounds and to compounds for treating neurodegenerative disease. The present invention also relates to an animal model for neurodegenerative disease.