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1. 发明申请

WO2004075834A2 PHARMACEUTICAL COMPOSITIONS OF SAFINGOL AND METHODS OF USING THE SAME 审中-公开
标题翻译： SAFINGOL的药物组合物及其使用方法
公开(公告)号：WO2004075834A2
公开(公告)日：2004-09-10
申请号：PCT/US2004004960
申请日：2004-02-19
申请人： LOS ANGELES CHILDRENS HOSPITAL , MAURER BARRY JAMES , REYNOLDS PATRICK C , VISHNUVAJJALA RAO B , GUPTA SHANKER
发明人： MAURER BARRY JAMES , REYNOLDS PATRICK C , VISHNUVAJJALA RAO B , GUPTA SHANKER
IPC分类号： A61K20060101 , A61K9/00 , A61K9/08 , A61K9/107 , A61K9/19 , A61K31/133 , A61K31/19 , A61K31/685 , A61K31/739 , A61K47/10 , A61K47/12 , A61K47/26 , A61P35/00 , A61K
CPC分类号： A61K31/133 , A61K9/0019 , A61K9/08 , A61K9/107 , A61K9/19 , A61K31/19 , A61K31/685 , A61K31/739 , A61K47/10 , A61K47/12 , A61K47/26
摘要： The present invention provides stable aqueous solutions consisting essentially of: (a) a sphingolipid; (b) lactic acid; and (c) optionally a stabilizing agent; wherein the solution has a molar ratio of lactic acid to sphingolipid of 1:1 to 10:1. The present invention further provides an emulsion formulation consisting essentially of: (a) lactic acid; (b) a sphingolipid, wherein the sphingolipid is present in an amount of about .1 to about 30 mg/ml of emulsion; (b) optionally an isotonic agent; and (c) a phospholipid present in an amount of about 0.2 to about 200 mg/ml of emulsion. Methods of making and using the compositions are also provided.
摘要翻译：本发明提供基本上由以下组成的稳定的水溶液：（a）鞘脂; （b）乳酸; 和（c）任选的稳定剂; 其中所述溶液的乳酸与鞘脂的摩尔比为1:1至10:1。本发明还提供一种乳液制剂，其主要由以下物质组成：（a）乳酸; （b）鞘脂，其中所述神经鞘脂以约0.1至约30mg / ml的乳剂的量存在; （b）任选的等渗剂; 和（c）以约0.2至约200mg / ml的乳液的量存在的磷脂。还提供了制备和使用组合物的方法。

2. 发明申请

WO2007124486A3 BMP4 INHIBITORS 审中-公开
标题翻译： BMP4抑制剂
公开(公告)号：WO2007124486A3
公开(公告)日：2008-10-23
申请号：PCT/US2007067221
申请日：2007-04-23
申请人： LOS ANGELES CHILDRENS HOSPITAL , SHI WEI , WARBURTON DAVID
发明人： SHI WEI , WARBURTON DAVID
IPC分类号： C12P21/06
CPC分类号： C07K14/475 , A61K38/00
摘要： A fragment of Gremlin having the sequence of amino acids shown in SEQ ID NO:2 or a functional subsequence of the fragment is described. The fragment is capable of altering BMP4 activity. Also provided is a polynucleotide encoding the fragment. Also disclosed is an assay for testing a molecule for Gremlin inhibitory activity. The invention also provides a method of inhibiting BMP4 activity.
摘要翻译：描述了具有SEQ ID NO：2所示氨基酸序列或片段的功能性亚序列的Gremlin片段。该片段能够改变BMP4的活性。还提供了编码该片段的多核苷酸。还公开了一种用于测试分子用于Gremlin抑制活性的测定法。本发明还提供抑制BMP4活性的方法。

3. 发明申请

WO2004069203A3 ORAL COMPOSITIONS OF FENRETINIDE HAVING INCREASED BIOAVAILABILITY AND METHODS OF USING THE SAME 审中-公开
标题翻译：具有增加生物利用度的烯丙醇酯的口腔组合物及其使用方法
公开(公告)号：WO2004069203A3
公开(公告)日：2005-02-03
申请号：PCT/US2004003562
申请日：2004-01-29
申请人： LOS ANGELES CHILDRENS HOSPITAL , MAURER BARRY JAMES , REYNOLDS C PATRICK , YESAIR DAVID W , MCKEE ROBERT TRAVIS , BURGESS STEPHEN W , SHAW WALTER A
发明人： MAURER BARRY JAMES , REYNOLDS C PATRICK , YESAIR DAVID W , MCKEE ROBERT TRAVIS , BURGESS STEPHEN W , SHAW WALTER A
IPC分类号： A23L1/30 , A61K9/00 , A61K31/167 , A61K47/00
CPC分类号： A61K9/0095 , A23L33/10 , A23V2002/00 , A61K9/1617 , A61K9/1664 , A61K31/167 , A23V2250/30 , A23V2250/186 , A23V2250/192 , A23V2250/1846
摘要： The present invention provides an edible composition for oral delivery of an active agent such as a retinide. The composition comprises, in the form of a dry flowable powder: (a) an active agent such as a retinide; (b) lipid matrix composition; (c) optionally sweetener; (d) flour. Compositions of the invention may be administered per se or mixed with a solid or liquid food carrier, for direct oral consumption by a subject or administration through a feeding tube.
摘要翻译：本发明提供了用于口服递送活性剂如视黄酸盐的可食用组合物。组合物包含干燥可流动粉末的形式：（a）活性剂如视黄酸盐; （b）脂质基质组成; （c）任选的甜味剂; （d）面粉。本发明的组合物本身可以施用或与固体或液体食品载体混合，由受试者或给药通过饲管直接口服消费。

4. 发明申请

WO02060321A2 ASSESSING BLOOD BRAIN BARRIER DYNAMICS OR IDENTIFYING OR MEASURING SELECTED SUBSTANCES OR TOXINS IN A SUBJECT BY ANALYZING RAMAN SPECTRUM SIGNALS OF SELECTED REGIONS IN THE EYE 审中-公开
标题翻译：通过分析眼睛中选择的区域的拉曼光谱信号来评估血液脑屏障动态，或鉴定或测量选择的物质或毒素
公开(公告)号：WO02060321A2
公开(公告)日：2002-08-08
申请号：PCT/US0200649
申请日：2002-01-09
申请人： LOS ANGELES CHILDRENS HOSPITAL , CALIFORNIA INST OF TECHN , LAMBERT JAMES L , BORCHERT MARK S
发明人： LAMBERT JAMES L , BORCHERT MARK S
IPC分类号： A61B5/00 , G01N21/65
CPC分类号： A61B5/1455 , A61B5/14532 , A61B5/14546 , A61B5/4064 , A61B5/411 , G01J3/027 , G01N21/65 , G01N2021/656
摘要： A non-invasive method for analyzing the blood-brain barrier includes obtaining a Raman spectrum of a selected portion of the eye and monitoring the Raman spectrum to ascertain a change to the dynamics of the blood brain barrier. Also, non-invasive methods for determining the brain or blood level of an analyte of interest, such as glucose, drugs, alcohol, poisons, and the like, comprises: generating an excitation laser beam (e.g., at a wavelength of 600 to 900 nanometers); focusing the excitation laser beam into the anterior chamber of an eye of the subject so that aqueous humor, vitreous humor, or one or more conjunctiva vessels in the eye is illuminated; detecting (preferably confocally detecting) a Raman spectrum from the illuminated portion of the eye; and then determining the blood level or brain level (intracranial or cerebral spinal fluid level) of an analyte of interest for the subject from the Raman spectrum. In certain embodiments, the detecting step may be followed by the step of subtracting a confounding fluorescence spectrum from the Raman spectrum to produce a difference spectrum; and determining the blood level and/or brain level of the analyte of interest for the subject from that difference spectrum, preferably using linear or nonlinear multivariate analysis such as partial least squares analysis. Apparatus for carrying out the foregoing methods are also disclosed.
摘要翻译：用于分析血脑屏障的非侵入性方法包括获得眼睛的所选部分的拉曼光谱并监测拉曼光谱以确定血脑屏障动力学的变化。此外，用于确定目标分析物（例如葡萄糖，药物，酒精，毒素等）的脑或血液水平的非侵入性方法包括：产生激发激光束（例如，波长为600至900 纳米）; 将激发激光束聚焦到受试者的眼睛的前房中，使得眼睛中的房水，玻璃体液或一个或多个结膜血管被照亮; 从眼睛的照射部分检测（优选地共同检测）拉曼光谱; 然后从拉曼光谱确定受试者感兴趣的分析物的血液水平或脑水平（颅内或脑脊液水平）。在某些实施方案中，检测步骤之后可以是从拉曼光谱中减去混杂荧光光谱以产生差光谱的步骤; 并且优选使用线性或非线性多变量分析（例如偏最小二乘法分析）从所述差异光谱确定受试者感兴趣分析物的血液水平和/或脑水平。还公开了用于实现上述方法的装置。

5. 发明申请

WO0142425A3 LUNG STEM CELLS AND LUNG REGENERATION 审中-公开
标题翻译：肺癌细胞和肺再生
公开(公告)号：WO0142425A3
公开(公告)日：2002-05-10
申请号：PCT/US0042646
申请日：2000-12-07
申请人： LOS ANGELES CHILDRENS HOSPITAL , SCHWARZ MARGARET , WARBURTON DAVID , DRISCOLL BARBARA , BUCKLEY SUSAN A
发明人： SCHWARZ MARGARET , WARBURTON DAVID , DRISCOLL BARBARA , BUCKLEY SUSAN A
IPC分类号： A61K35/12 , A61K35/42 , C12N5/071 , C12N5/074 , A01N63/00 , A61K48/00 , C12N5/08
CPC分类号： C12N5/0689 , A61K35/12 , A61K35/42
摘要： A method of stimulating the growth of lung alveolar surface in a lung comprises providing progenitor or stem cells capable of regenerating lung alveolar surface, and then administering the progenitor or stem cells to the lung in an amount sufficient to stimulate the growth of lung alveolar surface therein.
摘要翻译：刺激肺肺泡表面在肺中的生长的方法包括提供能够再生肺泡表面的祖细胞或干细胞，然后以足以刺激肺泡表面生长的量将祖细胞或干细胞给予肺。

6. 发明申请

WO2015184187A2 METHOD, SYSTEM AND SOFTWARE FOR ASSESSING EXTUBATION FAILURE 审中-公开
标题翻译：方法，系统和软件评估放射性失败
公开(公告)号：WO2015184187A2
公开(公告)日：2015-12-03
申请号：PCT/US2015033052
申请日：2015-05-28
申请人： LOS ANGELES CHILDRENS HOSPITAL
发明人： KHEMANI ROBINDER , FLINK RUTGER , NEWTH CHRISTOPHER JL
IPC分类号： A61B5/0205 , G06F19/00
CPC分类号： A61B5/0816 , A61B5/0205 , A61B5/037 , A61B5/0806 , A61B5/085 , A61B5/091 , A61B5/4233 , G06F19/3481 , Y02A90/26
摘要： A method, system and software for monitoring a signal from a living subject to measure a function of the living subject. Specifically, a computer method, computer system and software for accurately calibrating respiratory inductance plethysmography and esophageal manometry signals to measure effort of breathing before and after extubation of a patient to provide an automated real-time interpretation for a clinician of whether or not the patient has upper airway obstruction, and severity of the same. The method, system and software can be adapted specifically for a child or infant.
摘要翻译：一种用于监测来自活体的信号以测量活体的功能的方法，系统和软件。具体地说，一种计算机方法，计算机系统和用于精确校准呼吸电感体积描记法和食管测压信号的软件，以测量患者拔管之前和之后的呼吸努力，以便为临床医生提供患者是否具有自动实时解释上呼吸道阻塞，严重程度相同。该方法，系统和软件可以专门适用于儿童或婴儿。

7. 发明申请

WO2012178184A3 REMOVABLE PROTECTIVE SHELL FOR IMAGING AGENTS AND BIOACTIVE SUBSTANCES 审中-公开
标题翻译：用于成像剂和生物物质的可拆卸保护壳
公开(公告)号：WO2012178184A3
公开(公告)日：2014-05-08
申请号：PCT/US2012044052
申请日：2012-06-25
申请人： LOS ANGELES CHILDRENS HOSPITAL , UNIV SOUTHERN CALIFORNIA , WILLIAMS TRAVIS J , CHANG ANDY Y
发明人： WILLIAMS TRAVIS J , CHANG ANDY Y
IPC分类号： A61K9/00
CPC分类号： A61K49/1881 , A61K9/0009 , A61K9/5123
摘要： The invention provides nanoparticles for delivery of imaging agents, drugs, and other molecules, such as genetic material. The nanoparticles have a core structure comprising the imaging agent and/or drug, and a shell structure that allows for water solubility. The shell structure further provides a barrier with limited water permeability that protects the core. The nanoparticles can be induced to release their cargo by treatment with ultrasound. Methods of delivering drugs and imaging agents are also provided, whereby the nanoparticles are delivered to tissues of interest in a substantially inert form, then activated using ultrasound to release the drugs or imaging agents.
摘要翻译：本发明提供用于递送成像剂，药物和其它分子（例如遗传物质）的纳米颗粒。纳米颗粒具有包含成像剂和/或药物的核心结构以及允许水溶性的壳结构。壳体结构进一步提供了保护芯的有限水渗透性的屏障。通过用超声波处理可以诱导纳米颗粒释放其货物。还提供递送药物和成像剂的方法，由此将纳米颗粒以基本上惰性的形式递送至感兴趣的组织，然后使用超声波激活以释放药物或成像剂。

8. 发明申请

WO0147518A8 METHODS OF FACILITATING VASCULAR GROWTH IN CARDIAC MUSCLE AND METHODS FOR THE PRODUCTION OF RECOMBINANT EMAP II 审中-公开
标题翻译：促进心脏肌肉中血管生长的方法及生产重组EMAP II的方法
公开(公告)号：WO0147518A8
公开(公告)日：2001-11-15
申请号：PCT/US0033467
申请日：2000-12-08
申请人： LOS ANGELES CHILDRENS HOSPITAL , SCHWARZ MARGARET
发明人： SCHWARZ MARGARET
IPC分类号： A61K31/4178 , A61K38/00 , C07K14/52 , C07K16/24 , A61K48/00
CPC分类号： C07K14/52 , A61K31/4178 , A61K38/00 , A61K2039/505 , C07K16/24
摘要： A method of facilitating vascular growth in cardiac muscle of a subject in need of such treatment comprises inhibiting EMAP II activity in said subject by an amount effective to stimulate vascular growth in said cardiac muscle. The inhibiting step may be carried out by any suitable means, such as: By administering a compound (e.g., an antibody) that specifically binds to EMAP II to said subject in an amount effective to stimulate vascular growth in said cardiac muscle; by downregulating EMAP II expression in said subject by an amount effective to stimulate vascular growth in said cardiac muscle (e.g., by administration of an antisense olgionucleotide); or by administering an EMAP II receptor antagonist to said subject in an amount effective to stimulate vascular growth in said cardiac muscle.
摘要翻译：促进需要这种治疗的受试者的心肌中血管生长的方法包括通过有效刺激所述心肌中的血管生长的量来抑制所述受试者中的EMAP II活性。抑制步骤可以通过任何合适的手段进行，例如：通过以有效刺激所述心肌中的血管生长的量向所述受试者施用特异性结合EMAP II的化合物（例如抗体）通过下调有效刺激所述心肌中血管生长的量（例如，通过给予反义寡核苷酸），将所述受试者中的EMAP II表达下调; 或者通过以有效刺激所述心肌中血管生长的量向所述受试者施用EMAP II受体拮抗剂。

9. 发明申请

WO0000207A9 TREATMENT OF HYPERPROLIFERATIVE DISORDERS 审中-公开
标题翻译：治疗高血压病
公开(公告)号：WO0000207A9
公开(公告)日：2000-05-18
申请号：PCT/US9914591
申请日：1999-06-28
申请人： LOS ANGELES CHILDRENS HOSPITAL , MAURER BARRY JAMES , REYNOLDS C PATRICK , CABOT MYLES
发明人： MAURER BARRY JAMES , REYNOLDS C PATRICK , CABOT MYLES
IPC分类号： A61K45/00 , A61K31/07 , A61K31/133 , A61K31/165 , A61K31/167 , A61K31/535 , A61K31/5375 , A61K31/6615 , A61K31/70 , A61K31/7032 , A61K45/06 , A61P35/00 , A61P43/00 , A61K31/13 , A61K31/20 , A61K31/66
CPC分类号： A61K45/06 , A61K31/07 , A61K31/165 , A61K31/535 , A61K31/70 , Y10S424/15 , A61K2300/00
摘要： A method of treating a hyperproliferative disorder in a subject in need of such treatment, comprising administering to said subject, in combination, a treatment effective amount of: (a) a ceramide-generating retinoid such as fenretinide or a pharmaceutically acceptable salt thereof; and (b) at least one (and in certain embodiments at least two) ceramide degredation inhibitor, such as compounds selected from the group consisting of (i) glucosylceramide synthesis inhibitors, (ii) sphingosine-1-phosphate synthesis inhibitors, and (iii) protein kinase C inhibitors. A preferred glucosyl ceramide synthesis inhibitor is 1-phenyl-2-palmitoylamino-3-morpholino-1-propanol. A preferred sphingosine-1-phosphate synthesis inhibitor is D-erytho-N,N-dimethylsphingosine. A preferred protein kinase C inhibitor is L-threo-dihydrosphingosine.
摘要翻译：一种治疗需要这种治疗的受试者的过度增殖性疾病的方法，包括给所述受试者组合施用治疗有效量的：（a）产生神经酰胺的类视黄醇例如芬维A胺或其药学上可接受的盐; 和（b）至少一种（和在某些实施方案中至少两种）神经酰胺降解抑制剂，例如选自（i）葡糖基神经酰胺合成抑制剂，（ii）鞘氨醇-1-磷酸合成抑制剂和（iii））蛋白激酶C抑制剂。优选的葡糖基神经酰胺合成抑制剂是1-苯基-2-棕榈酰氨基-3-吗啉代-1-丙醇。优选的鞘氨醇-1-磷酸合成抑制剂是D-erytho-N，N-二甲基鞘氨醇。优选的蛋白激酶C抑制剂是L-苏式 - 二氢鞘氨醇。

10. 发明申请

WO2015153805A3 TARGETED POLYMERIZED NANOPARTICLES FOR CANCER TREATMENT 审中-公开
标题翻译：用于癌症治疗的靶向聚合纳米颗粒
公开(公告)号：WO2015153805A3
公开(公告)日：2015-12-03
申请号：PCT/US2015023943
申请日：2015-04-01
申请人： LOS ANGELES CHILDRENS HOSPITAL , NANOVALENT PHARMACEUTICALS INC
发明人： NAGY JON O , TRICHE TIM , KANG HYUNGGYOO
IPC分类号： A61K47/34 , A61K9/127 , A61K47/48
CPC分类号： A61K9/1273 , A61K9/1271 , A61K9/51 , A61K31/136 , A61K31/337 , A61K31/407 , A61K31/4745 , A61K31/475 , A61K31/573 , A61K31/704 , A61K31/7068 , A61K31/713 , A61K33/24 , A61K45/06 , A61K2300/00
摘要： The invention relates to a novel drug delivery vehicle. Various embodiments of the invention provide a hybrid polymerized liposomal nanoparticle comprising both polymerizable lipids and non-polymerizable lipids. Therapeutic agents can be loaded into the polymerized liposomal nanoparticle and targeting agents can be conjugated to the surface of the polymerized liposomal nanoparticle. Also described in the invention are methods, compositions and kits that utilize the hybrid polymerized liposomal nanoparticle to treat disease conditions such as various cancers.
摘要翻译：本发明涉及新型药物递送载体。本发明的各种实施方案提供了包含可聚合脂质和不可聚合脂质的杂化聚合脂质体纳米颗粒。可以将治疗剂加载到聚合的脂质体纳米颗粒中，并且靶向剂可以与聚合的脂质体纳米颗粒的表面缀合。本发明还描述了利用杂化聚合脂质体纳米颗粒治疗诸如各种癌症的疾病状况的方法，组合物和试剂盒。

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