公开(公告)号：US08603992B2

公开(公告)日：2013-12-10

申请号：US12504331

申请日：2009-07-16

Applicant: Jianjie Ma ,  Noah Weisleder

Inventor： Jianjie Ma ,  Noah Weisleder

IPC: C12N15/11 ,  A61K48/00 ,  C07H21/02 ,  C07H21/04

CPC classification number: A61K35/34 ,  A01K67/0275 ,  A01K2217/058 ,  A01K2217/203 ,  A01K2217/206 ,  A01K2227/105 ,  A01K2267/03 ,  A61K38/00 ,  A61K38/1709 ,  C07K14/705 ,  C12N15/8509 ,  C12N2800/30 ,  C12N2840/20 ,  C12Q1/6883 ,  C12Q2600/136 ,  C12Q2600/156 ,  C12Q2600/158

Abstract: Disclosed herein are compositions and methods for treatment of muscle dysfunction (including sarcopenia) and other diseases involving skeletal muscle, including age-related muscle dysfunction. In addition, the invention relates to therapeutic compositions comprising nucleotides and/or polypeptides of the invention in combination with a pharmaceutically acceptable carrier, wherein the composition facilitates the treatment of skeletal muscle disorder, including those related to thr normal aging process. Moreover, the invention relates to the treatment and/or prevention of pathological conditions associated with altered intracellular Ca2+ regulation and disrupted membrane structure that occurs when the expression levels of MG29 are reduced.

Abstract translation: 本文公开了用于治疗肌肉功能障碍（包括肌营养不良）和涉及骨骼肌的其它疾病（包括年龄相关的肌肉功能障碍）的组合物和方法。 此外，本发明涉及包含与药学上可接受的载体组合的本发明的核苷酸和/或多肽的治疗组合物，其中所述组合物有助于骨骼肌疾病的治疗，包括与正常老化过程相关的那些。 此外，本发明涉及治疗和/或预防与MG29的表达水平降低时发生的改变的细胞内Ca 2+调节和破坏的膜结构相关的病理状况。

公开(公告)号：US08541548B2

公开(公告)日：2013-09-24

申请号：US12140751

申请日：2008-06-17

Applicant: David B. Rozema

Inventor： David B. Rozema

IPC: A61K38/00 ,  C07K1/00

CPC classification number: C07D495/04 ,  A01K2217/058 ,  A01K2267/0362 ,  A61K48/0041 ,  C07C233/21 ,  C07C233/31 ,  C07C233/39 ,  C07D307/60 ,  C07D475/00 ,  C12N15/87

Abstract: The present invention is directed compounds for reversibly modification of biologically active molecules. Described are polyconjugates systems that incorporate targeting, anti-opsonization, anti-aggregation, and transfection activities into small biocompatible in vivo delivery conjugates. The use of reversible modification provides for physiologically responsive activity modulation.

Abstract translation: 本发明涉及可逆地修饰生物活性分子的化合物。 描述了将靶向，抗调理作用，抗聚集和转染活性结合到小的生物相容性体内递送缀合物中的多偶联体系。 使用可逆修饰提供了生理反应活性调节。

公开(公告)号：US08513209B2

公开(公告)日：2013-08-20

申请号：US12742233

申请日：2008-11-10

Applicant: Eric Olson ,  Eva van Rooij

Inventor： Eric Olson ,  Eva van Rooij

IPC: A61K48/00

CPC classification number: A61K45/06 ,  A01K67/0275 ,  A01K2207/05 ,  A01K2217/058 ,  A01K2227/105 ,  A01K2267/0375 ,  A61K31/7105 ,  C12N15/113 ,  C12N2310/113 ,  C12N2310/14 ,  C12N2330/10 ,  C12N2830/008

Abstract: A family of microRNAs, called the miR-15 family, which includes miR-195, are shown to be up-regulated during pathological cardiac remodeling and repress the expression of mRNAs required for cell proliferation and survival, with consequent loss of cardiomyocytes. Strategies to block expression of the miR-15 family in the heart as a treatment for diverse cardiac disease are provided.

Abstract translation: 被称为miR-15家族的miRNA家族（包括miR-195）在病理性心脏重构过程中被上调，并抑制细胞增殖和存活所需的mRNA的表达，从而导致心肌细胞的丧失。 提供了阻止miR-15家族在心脏中表达作为多种心脏病治疗的策略。

公开(公告)号：US20130131144A1

公开(公告)日：2013-05-23

申请号：US13661155

申请日：2012-10-26

Applicant: The Johns Hopkins University

Inventor： Shanthini Sockanathan ,  Meenakshi Rao

IPC: C12N9/16 ,  C12N15/113 ,  C12Q1/02 ,  C12N15/85

CPC classification number: C12N9/16 ,  A01K67/0275 ,  A01K67/0276 ,  A01K2217/05 ,  A01K2217/058 ,  A01K2227/30 ,  A01K2267/0356 ,  C12N15/1137 ,  C12N15/85 ,  C12Q1/025 ,  G01N2500/00

Abstract: The present invention relates to compositions to treat glycerophosphodiester phosphodiesterase (GDE) related disorders. The invention also relates to methods treating GDE related disorders. The invention further relates to kits for treating GDE related disorders in a subject. The invention further relates to methods of identifying novel treatments for treating GDE related disorders in a subject.

Abstract translation: 本发明涉及治疗甘油磷酸二酯磷酸二酯酶（GDE）相关疾病的组合物。 本发明还涉及治疗GDE相关疾病的方法。 本发明还涉及用于治疗受试者中GDE相关疾病的试剂盒。 本发明还涉及鉴定用于治疗受试者中GDE相关疾病的新疗法的方法。

公开(公告)号：US20130090522A1

公开(公告)日：2013-04-11

申请号：US13645788

申请日：2012-10-05

Applicant: RECOMBINETICS, INC.

Inventor： Scott C. Fahrenkrug ,  Daniel F. Carlson ,  Aron M. Geurts

IPC: A61D19/00

CPC classification number: C12N15/8509 ,  A01K67/0273 ,  A01K67/0275 ,  A01K2217/058 ,  A01K2217/203 ,  A01K2227/101 ,  A01K2227/103 ,  A01K2227/105 ,  A01K2227/108 ,  A01K2267/03 ,  A01K2267/035 ,  A61D19/00 ,  C07K14/4712 ,  C12N15/1138 ,  C12N2015/8527 ,  C12N2310/14 ,  C12N2800/30 ,  C12N2800/90

Abstract: Transgenic artiodactyls are described as well as methods of making and using such artiodactyls.

Abstract translation: 本发明描述了转基因偶蹄菌，以及制备和使用这些偶氮酰基的方法。

公开(公告)号：US20130046079A1

公开(公告)日：2013-02-21

申请号：US13654007

申请日：2012-10-17

Applicant: Crescendo Biologics Limited

Inventor： Marianne BRÜGGEMANN ,  Xiangang ZOU

IPC: A01K67/027 ,  C07K16/00 ,  C12P21/00

CPC classification number: A01K67/0276 ,  A01K2217/058 ,  A01K2217/075 ,  A01K2227/105 ,  A01K2267/01 ,  C07K16/00 ,  C07K16/06 ,  C07K2317/56 ,  C12N2800/30

Abstract: The present invention provides in a first aspect a mouse in which the λ (lambda) light chain locus has been functionally silenced. In one embodiment, the mouse λ light chain locus was functional silenced by deletion of acne segments coding for the λ light chain locus. In a further aspect, a mouse containing functionally silenced λ and κ (kappa) L chain loci was produced. The invention is useful for the production of antibodies, for example heterologous antibodies, including heavy chain only antibodies.

Abstract translation: 本发明在第一方面提供了一种其中λ（λ）轻链基因座功能性沉默的小鼠。 在一个实施方案中，小鼠λ轻链基因座通过缺失编码λ轻链基因座的痤疮区段而被功能性沉默。 在另一方面，包含功能性沉默的λ和＆ （kappa）L链基因座。 本发明可用于生产抗体，例如异源抗体，包括仅重链抗体。

公开(公告)号：US08367888B2

公开(公告)日：2013-02-05

申请号：US12476087

申请日：2009-06-01

Applicant: Marianne Brüggemann ,  Xiangang Zou

Inventor： Marianne Brüggemann ,  Xiangang Zou

IPC: A01K67/00 ,  A01K67/027

CPC classification number: A01K67/0276 ,  A01K2217/058 ,  A01K2217/075 ,  A01K2227/105 ,  A01K2267/01 ,  C07K16/00 ,  C07K16/06 ,  C07K2317/56 ,  C12N2800/30

Abstract: The present invention provides in a first aspect a mouse in which the λ (lambda) light chain locus has been functionally silenced. In one embodiment, the mouse λ light chain locus was functional silenced by deletion of gene segments coding for the λ light chain locus. In a further aspect, a mouse containing functionally silenced λ and κ (kappa) L chain loci was produced. The invention is useful for the production of antibodies, for example heterologous antibodies, including heavy chain only antibodies.

Abstract translation: 本发明在第一方面提供了一种其中λ（λ）轻链基因座功能性沉默的小鼠。 在一个实施方案中，小鼠λ轻链基因座通过缺失编码λ轻链基因座的基因片段而被功能性沉默。 在另一方面，包含功能性沉默的λ和＆ （kappa）L链基因座。 本发明可用于生产抗体，例如异源抗体，包括仅重链抗体。

公开(公告)号：US08309532B2

公开(公告)日：2012-11-13

申请号：US12803118

申请日：2010-06-17

Applicant: Elena Feinstein ,  Jorg Kaufmann ,  Klaus Giese

Inventor： Elena Feinstein ,  Jorg Kaufmann ,  Klaus Giese

IPC: A61K31/70 ,  C07H21/02 ,  C07H21/04

CPC classification number: C07H21/02 ,  A01K2217/058 ,  A01K2267/0362 ,  A61K31/713 ,  A61K39/39541 ,  A61K45/06 ,  C12N15/113 ,  C12N2310/14 ,  C12N2310/321 ,  A61K2300/00 ,  C12N2310/3521

Abstract: The present invention provides novel molecules, compositions, methods and uses for treating microvascular disorders, eye diseases and respiratory conditions based upon inhibition of the RTP801 gene and/or protein.

Abstract translation: 本发明提供了基于RTP801基因和/或蛋白质的抑制来治疗微血管病症，眼部疾病和呼吸病症的新型分子，组合物，方法和用途。

公开(公告)号：US20120030782A1

公开(公告)日：2012-02-02

申请号：US13252638

申请日：2011-10-04

Applicant: Lisa S. D. Emmett ,  Theresa Gratsch ,  K. Sue O'Shea ,  J. Matthew Velkey ,  Michael J. Welsh ,  William Wu

Inventor： Lisa S. D. Emmett ,  Theresa Gratsch ,  K. Sue O'Shea ,  J. Matthew Velkey ,  Michael J. Welsh ,  William Wu

IPC: A01K67/027 ,  C12N5/10 ,  C12N15/85

CPC classification number: A01K67/0275 ,  A01K2217/058 ,  A01K2227/105 ,  A01K2267/03 ,  C12N15/873

Abstract: The present invention provides methods of altering gene expression of embryos to provide compositions and methods for efficient generation of transgenic animals. In particular, the present invention provides compositions and methods for generating germline transgenic animals by direct injection of nucleic acid molecules into animals.

Abstract translation: 本发明提供了改变胚胎基因表达以提供用于有效产生转基因动物的组合物和方法的方法。 特别地，本发明提供了通过将核酸分子直接注入动物来产生种系转基因动物的组合物和方法。

公开(公告)号：US08017109B2

公开(公告)日：2011-09-13

申请号：US12140703

申请日：2008-06-17

Applicant: Darren H. Wakefield ,  David B. Rozema ,  Sean D. Monahan ,  Lori Higgs ,  Jon A. Wolff ,  James E. Hagstrom

Inventor： Darren H. Wakefield ,  David B. Rozema ,  Sean D. Monahan ,  Lori Higgs ,  Jon A. Wolff ,  James E. Hagstrom

IPC: A61K31/74

CPC classification number: C12N15/87 ,  A01K2217/058 ,  A01K2267/0362 ,  A61K47/58

Abstract: The present invention is directed to membrane active polymers useful for cellular delivery of compounds. Described are polyconjugates systems that incorporate targeting, anti-opsonization, anti-aggregation, and transfection activities into small biocompatible in vivo delivery conjugates. The use of reversible modification provides for physiologically responsive activity modulation.

Abstract translation: 本发明涉及可用于细胞递送化合物的膜活性聚合物。 描述了将靶向，抗调理作用，抗聚集和转染活性结合到小的生物相容性体内递送缀合物中的多偶联体系。 使用可逆修饰提供了生理反应活性调节。