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    Compositions and methods for diagnosing and treating retinal diseases
    1.
    发明申请
    Compositions and methods for diagnosing and treating retinal diseases 审中-公开
    用于诊断和治疗视网膜疾病的组合物和方法

    公开(公告)号：US20060252107A1

    公开(公告)日：2006-11-09

    申请号：US11361027

    申请日：2006-02-22

    申请人： Ryo Kubota ,  Krzysztof Palczewski ,  Anna Gall

    发明人： Ryo Kubota ,  Krzysztof Palczewski ,  Anna Gall

    IPC分类号： G01N33/53 ,  C07K16/18 ,  C07K14/47 ,  C07D213/55 ,  C07D213/20 ,  C12N5/06

    CPC分类号： A61K39/0007 ,  A61K2039/6031 ,  C07D213/20 ,  C07D213/38 ,  C07D213/50 ,  C07D213/56 ,  C07K16/44

    摘要： The present invention relates generally to the preparation and use of retinoid N-retinylidene-N-retinyl-ethanolamine (A2E) conjugated to a carrier polypeptide and to the preparation and use of antibodies that bind specifically to A2E. The invention relates to the use of A2E conjugates as immunogens or vaccines and to the use of A2E specific antibodies for treatment of ophthalmic diseases. Provided herein are methods for enhancing retinal neuronal cell survival, including photoreceptor cell survival, using antibodies that specifically bind to A2E or by inducing an immune response using an A2E immunoconjugate. Enhancing survival of photoreceptor cells or decreasing accumulation of A2E in the eye using the A2E immunoconjugates or A2E specific antibodies is useful for treatment of ophthalmic diseases such as macular degeneration.

    摘要翻译： 本发明一般涉及与载体多肽缀合的类视黄醇N-亚视黄基-N-视黄醇 - 乙醇胺（A2E）的制备和用途，以及制备和使用与A2E特异性结合的抗体。 本发明涉及使用A2E缀合物作为免疫原或疫苗，以及使用A2E特异性抗体治疗眼科疾病。 本文提供了使用特异性结合A2E的抗体或通过使用A2E免疫缀合物诱导免疫应答来增强视网膜神经元细胞存活的方法，包括感光细胞存活。 使用A2E免疫缀合物或A2E特异性抗体提高感光细胞的存活或减少A2E在眼中的积累，可用于治疗眼部疾病如黄斑变性。

    METHODS FOR ASSESSING A PHYSIOLOGICAL STATE OF A MAMMALIAN RETINA
    4.
    发明申请
    METHODS FOR ASSESSING A PHYSIOLOGICAL STATE OF A MAMMALIAN RETINA 有权
    评估MAMMALIAN RETINA生理状态的方法

    公开(公告)号：US20100262016A1

    公开(公告)日：2010-10-14

    申请号：US12722314

    申请日：2010-03-11

    申请人： Yoshikazu Imanishi ,  Krzysztof Palczewski ,  Bryan S. Sires ,  Eric J. Seibel

    发明人： Yoshikazu Imanishi ,  Krzysztof Palczewski ,  Bryan S. Sires ,  Eric J. Seibel

    IPC分类号： A61B6/00

    CPC分类号： A61B5/0059 ,  A61B3/10 ,  A61B3/1025

    摘要： In one aspect the present invention provides methods for assessing a physiological state of a mammalian retina in vivo, the methods of this aspect of the invention each include the steps of (a) irradiating a portion of a mammalian retina, in vivo, with light having a wavelength in the range of from 600 nm to 1000 nm at an intensity sufficient to stimulate two-photon-induced fluorescence in the retina; and (b) assessing a physiological state of the retina by analyzing the fluorescence.

    摘要翻译： 一方面，本发明提供了用于评估哺乳动物视网膜在体内的生理状态的方法，本发明这方面的方法各自包括以下步骤：（a）在体内照射哺乳动物视网膜的一部分， 以足以刺激视网膜中双光子诱导荧光的强度在600nm至1000nm范围内的波长; 和（b）通过分析荧光来评估视网膜的生理状态。

    Methods for assessing a physiological state of a mammalian retina
    5.
    发明授权
    Methods for assessing a physiological state of a mammalian retina 有权
    用于评估哺乳动物视网膜的生理状态的方法

    公开(公告)号：US07706863B2

    公开(公告)日：2010-04-27

    申请号：US10926780

    申请日：2004-08-26

    申请人： Yoshikazu Imanishi ,  Krzysztof Palczewski ,  Bryan S. Sires ,  Eric J. Seibel

    发明人： Yoshikazu Imanishi ,  Krzysztof Palczewski ,  Bryan S. Sires ,  Eric J. Seibel

    IPC分类号： A61B6/00

    CPC分类号： A61B5/0059 ,  A61B3/10 ,  A61B3/1025

    摘要： In one aspect the present invention provides methods for assessing a physiological state of a mammalian retina in vivo, the methods of this aspect of the invention each include the steps of: (a) irradiating a portion of a mammalian retina, in vivo, with light having a wavelength in the range of from 600 nm to 1000 nm at an intensity sufficient to stimulate two-photon-induced fluorescence in the retina; and (b) assessing a physiological state of the retina by analyzing the fluorescence.

    摘要翻译： 一方面，本发明提供了用于评估体内哺乳动物视网膜的生理状态的方法，本发明的这个方面的方法各自包括以下步骤：（a）在体内用光照射哺乳动物视网膜的一部分 具有在600nm至1000nm范围内的波长，其强度足以刺激视网膜中的双光子诱导荧光; 和（b）通过分析荧光来评估视网膜的生理状态。

    Expression of polypeptides in rod outer segment membranes
    7.
    发明申请
    Expression of polypeptides in rod outer segment membranes 审中-公开
    多肽在杆外段膜中的表达

    公开(公告)号：US20050124792A1

    公开(公告)日：2005-06-09

    申请号：US10495994

    申请日：2002-11-21

    申请人： Krzysztof Palczewski ,  Ning Li ,  Juan Ballesteros

    发明人： Krzysztof Palczewski ,  Ning Li ,  Juan Ballesteros

    IPC分类号： A01K67/027 ,  A61K48/00 ,  C07H21/04 ,  C07K14/47 ,  C07K14/705 ,  C07K19/00 ,  C12N5/06 ,  C12N5/10 ,  C12N15/09 ,  C12N15/85

    CPC分类号： A01K67/0275 ,  A01K2207/15 ,  A01K2217/00 ,  A01K2217/05 ,  A01K2217/072 ,  A01K2227/105 ,  A01K2227/50 ,  A01K2267/01 ,  A01K2267/03 ,  C07K14/705 ,  C07K2319/01 ,  C07K2319/03 ,  C07K2319/40 ,  C12N15/8509 ,  C12N2800/30 ,  C12N2830/008

    摘要： The invention provides a transgene construct, containing a nucleic acid encoding a photoreceptor specific regulatory sequence, a membrane-associated polypeptide, and a photoreceptor outer segment targeting signal. For example, the invention pro-vides a transgene construct, containing a nucleic acid encoding a rhodopsin promoter, a membrane-associated polypeptide, and a rod outer segment (ROS) targeting signal. In addition, the invention provides a gene targeting construct containing a transgene encoding a polypeptide that contains a rod outer segment (ROS) targeting signal. The transgene is flanked by 5′ and 3′ DNA sequences that are homologous to the rhodopsin gene. Homologous recombination between the construct and a rhodopsin gene results in operable association between the transgene and a rod-specific regulatory sequence. The invention also provides cells and animals whose genome contain a functional disruption of one or both endogenous rhodopsin gene alleles, and a transgene encoding a polypeptide that contains a ROS targeting signal operably associated with a rod-specific regulatory sequence. The invention constructs, cell and animals can be used to isolate transgenic polypeptides from the ROS membrane.

    摘要翻译： 本发明提供了转基因构建体，其含有编码感光体特异性调节序列的核酸，膜相关多肽和感光体外部区段靶向信号。 例如，本发明提供了转基因构建体，其含有编码视紫红质启动子，膜相关多肽和杆外区段（ROS）靶向信号的核酸。 此外，本发明提供了含有编码含有杆外节段（ROS）靶向信号的多肽的转基因的基因靶向构建体。 转基因侧面是与视紫红质基因同源的5'和3'DNA序列。 构建体和视紫红质基因之间的同源重组导致转基因和杆特异性调节序列之间的可操作的关联。 本发明还提供其基因组含有一种或两种内源性视紫红质基因等位基因的功能性破坏的细胞和动物，以及编码含有与杆特异性调节序列可操作地相关的ROS靶向信号的多肽的转基因。 本发明构建体，细胞和动物可用于从ROS膜分离转基因多肽。

    METHODS FOR THE TREATMENT AND PREVENTION OF AGE-RELATED RETINAL DYSFUNCTION
    10.
    发明申请
    METHODS FOR THE TREATMENT AND PREVENTION OF AGE-RELATED RETINAL DYSFUNCTION 有权
    用于治疗和预防与年龄相关的肾功能衰竭的方法

    公开(公告)号：US20130072557A1

    公开(公告)日：2013-03-21

    申请号：US13619947

    申请日：2012-09-14

    申请人： Tadao Maeda ,  David Saperstein ,  Krzysztof Palczewski

    发明人： Tadao Maeda ,  David Saperstein ,  Krzysztof Palczewski

    IPC分类号： A61K31/215

    CPC分类号： A61K31/215 ,  A61K9/0053 ,  A61K31/07 ,  A61K31/22 ,  A61K31/4709 ,  Y10S514/912

    摘要： A method of treating or preventing age-related retinal dysfunction involves administering to a patient a pharmaceutically effective amount of a synthetic retinal derivative repeatedly over a duration of at least about 3 months. Effective synthetic retinal derivatives include 9-cis-retinyl esters, 11-cis-retinyl esters, derivatives and congeners thereof, and combinations thereof. Suitable ester substituents include carboxylates of C1-C10 monocarboxylic acids and C2-C22 polycarboxylic acids.

    摘要翻译： 治疗或预防年龄相关性视网膜功能障碍的方法包括在至少约3个月的持续时间内重复给患者药物有效量的合成的视黄醛衍生物。 有效的合成视黄醛衍生物包括9-顺 - 视黄酯，11-顺 - 视黄酯，其衍生物和同系物及其组合。 合适的酯取代基包括C1-C10单羧酸和C2-C22多元羧酸的羧酸盐。