子分类：
| 序号 | 专利名 | 申请号 | 申请日 | 公开（公告）号 | 公开（公告）日 | 发明人 |
|---|---|---|---|---|---|---|
| 21 | AAV-MEDIATED GENE THERAPY FOR RPGR X-LINKED RETINAL DEGENERATION | US15699262 | 2017-09-08 | US20180036385A1 | 2018-02-08 | William A. BELTRAN; Gustavo D. AGUIRRE; Samuel G. JACOBSON; Artur V. CIDECIYAN; Alfred S. LEWIN; Sanford L. BOYE; William W. HAUSWIRTH; Wen-Tao DENG |
| Described herein are methods of preventing, arresting progression of or ameliorating vision loss and other conditions associated with retinitis pigmentosa and x-linked retinitis pigmentosa in a subject. The methods include administering to said subject an effective concentration of a composition comprising a recombinant adeno-associated virus (AAV) carrying a nucleic acid sequence encoding a normal retinitis pigmentosa GTPase regulator (RPGR gene), or fragment thereof, under the control of regulatory sequences which express the product of the gene in the photoreceptor cells of the subject, and a pharmaceutically acceptable carrier. | ||||||
| 22 | METHODS AND COMPOSITIONS FOR INHIBITION OF RAS | US15508387 | 2015-09-25 | US20170247376A1 | 2017-08-31 | Liansheng LI; Jun FENG; Yun Oliver LONG; Pingda REN; Yi LIU |
| Inhibitors of Ras protein, methods to modulate the activity of Ras protein, and methods of treatment of disorders mediated by Ras protein are provided. A method for regulating activity of a K-Ras, H-Ras or N-Ras mutant protein with a compound is described. Disorders that can be treated include cancer, such as hematological cancer, pancreatic cancer, MYH associated polyposis, colorectal cancer, or lung cancer. | ||||||
| 23 | COMPOSITIONS COMPRISING A MITOFUSIN INHIBITOR FOR PROMOTING CELL REPROGRAMMING AND A USE THEREOF | US14442883 | 2015-04-23 | US20170051254A1 | 2017-02-23 | Yee Sook CHO; Myung Jin SON; You Jeong KWON |
| The present invention relates to a composition comprising a repressor of mitofusin gene expression, an inhibitor of mitofusin protein activity, or a mixture thereof as an active ingredient for promoting reprogramming a differentiated cell into a pluripotent stem cell, and a use thereof. The composition according to the present invention increases the efficiency of reprogramming as well as reduces the time required for reprogramming to produce pluripotent stem cells. Therefore, the present composition can be beneficially used to develop the production technology of high efficiency pluripotent stem cell and secure a large-scale culture system. Further, the present composition can be beneficially used to maintain pluripotent stem cells and screen the compounds capable of promoting the reprogramming into pluripotent stem cells. | ||||||
| 24 | Preparations and methods for treating malignancies | US14060260 | 2013-10-22 | US09193800B2 | 2015-11-24 | Geoffrey Schmidt |
| Disclosed are therapeutic formulations comprising antibodies against the PEKRAEKIWK (SEQ ID NO:1) epitope of the monomeric isoform of A-protein and a physiologically acceptable carrier. Methods for the treatment of subjects using these therapeutic formulations are also disclosed. | ||||||
| 25 | Clinical Applications for Recombinant Human MxA Protein | US14741578 | 2015-06-17 | US20150273026A1 | 2015-10-01 | Robert W. VanDine; Robert P. Sambursky; Uma Mahesh Babu |
| Full length MxA constructs and truncated MxA constructs produce human MxA protein in E. coli. The full length MxA and truncated MxA constructs are preferably E. coli codon-optimized to optimize the amount of protein made using the constructs. T5 or T7 promoters can each be used in combination with either the full length MxA or the truncated MxA constructs. In one preferred embodiment, the MxA protein produced by the full length MxA or truncated MxA constructs is used in a control prep or external control. In other preferred embodiments, the MxA protein is used as a therapeutic. | ||||||
| 26 | AAV-MEDIATED GENE THERAPY FOR RPGR X-LINKED RETINAL DEGENERATION | US14413884 | 2013-01-23 | US20150202269A1 | 2015-07-23 | William A Beltran; Gustavo D Aguirre; Samuel G Jacobson; Artur V Cideciyan; Alfred S Lewin; Sanford L Boye; William W Hauswirth; Wen-Tao Deng |
| Described herein are methods of preventing, arresting progression of or ameliorating vision loss and other conditions associated with retinitis pigmentosa and x-linked retinitis pigmentosa in a subject. The methods include administering to said subject an effective concentration of a composition comprising a recombinant adeno-associated virus (AAV) carrying a nucleic acid sequence encoding a normal retinitis pigmentosa GTPase regulator (RPGR gene), or fragment thereof, under the control of regulatory sequences which express the product of the gene in the photoreceptor cells of the subject, and a pharmaceutically acceptable carrier. | ||||||
| 27 | RHOC-BASED IMMUNOTHERAPY | US12808649 | 2008-12-18 | US20110002954A1 | 2011-01-06 | Mads Hald Andersen; Per Thor Straten |
| The present invention relates generally to the field of prophylaxis and therapy of metastatic cancer. In particular there is provided a protein; Ras Homology gene family, member C (RhoC) or peptide fragments thereof that are capable of eliciting anti-cancer immune responses. Specifically, the invention relates to use of RhoC or peptides derived thereof or RhoC specific T-cells for treatment of metastatic cancer. Hence, the invention in one aspect relates to RhoC specific T-cells adoptively transferred or induced in vivo by vaccination as a treatment of cancer. Also the use of RhoC and immunogenic peptide fragments hereof in cancer treatment, diagnosis and prognosis is provided. | ||||||
| 28 | AAV-MEDIATED GENE THERAPY FOR RPGR X-LINKED RETINAL DEGENERATION | EP13816245 | 2013-01-23 | EP2872183A4 | 2015-12-09 | BELTRAN WILLIAM A; AGUIRRE GUSTAVO D; JACOBSON SAMUEL G; CIDECIYAN ARTUR V; LEWIN ALFRED S; BOYE SANFORD L; HAUSWIRTH WILLIAM W; DENG WEN-TAO |
| Described herein are methods of preventing, arresting progression of or ameliorating vision loss and other conditions associated with retinitis pigmentosa and x-linked retinitis pigmentosa in a subject. The methods include administering to a subject an effective concentration of a composition comprising a recombinant adeno-associated virus (AAV) carrying a nucleic acid sequence encoding a normal retinitis pigmentosa GTPase regulator (RPGR gene), or fragment thereof, under the control of regulatory sequences which express the product of the gene in the photoreceptor cells of the subject, and a pharmaceutically acceptable carrier. | ||||||
| 29 | AAV-MEDIATED GENE THERAPY FOR RPGR X-LINKED RETINAL DEGENERATION | EP13816245.8 | 2013-01-23 | EP2872183A1 | 2015-05-20 | BELTRAN, William, A.; AGUIRRE, Gustavo, D.; JACOBSON, Samuel, G.; CIDECIYAN, Artur, V.; LEWIN, Alfred, S.; BOYE, Sanford, L.; HAUSWIRTH, William, W.; DENG, Wen-Tao |
| Described herein are methods of preventing, arresting progression of or ameliorating vision loss and other conditions associated with retinitis pigmentosa and x-linked retinitis pigmentosa in a subject. The methods include administering to a subject an effective concentration of a composition comprising a recombinant adeno-associated virus (AAV) carrying a nucleic acid sequence encoding a normal retinitis pigmentosa GTPase regulator (RPGR gene), or fragment thereof, under the control of regulatory sequences which express the product of the gene in the photoreceptor cells of the subject, and a pharmaceutically acceptable carrier. | ||||||
| 30 | RHOC-BASED IMMUNOTHERAPY | EP08862897.9 | 2008-12-18 | EP2234635B1 | 2017-01-25 | ANDERSEN, Mads Hald; STRATEN, Per Thor |
| 31 | COMPOSITIONS COMPRISING A MITOFUSIN INHIBITOR FOR PROMOTING CELL REPROGRAMMING AND A USE THEREOF | EP15721111 | 2015-04-23 | EP3041928A4 | 2016-08-17 | CHO YEE SOOK; SON MYUNG JIN; KWON YOU JEONG |
| The present invention relates to a composition comprising a repressor of mitofusin gene expression, an inhibitor of mitofusin protein activity, or a mixture thereof as an active ingredient for promoting reprogramming a differentiated cell into a pluripotent stem cell, and a use thereof. The composition according to the present invention increases the efficiency of reprogramming as well as reduces the time required for reprogramming to produce pluripotent stem cells. Therefore, the present composition can be beneficially used to develop the production technology of high efficiency pluripotent stem cell and secure a large-scale culture system. Further, the present composition can be beneficially used to maintain pluripotent stem cells and screen the compounds capable of promoting the reprogramming into pluripotent stem cells. | ||||||
| 32 | MODULATORS OF ACYL-COA LYSOCARDIOLIPIN ACYLTRANSFERASE 1 ( ALCAT1) AND USES THEREOF | EP13749685 | 2013-02-15 | EP2822571A4 | 2016-03-09 | SHI YUGUANG |
| Compositions of modulators of acyl-CoA lysocardiolipin acyf transferase 1 (ALCAT1) expression, function or activity are provided. In particular, inhibitors of ALCAT1 are useful in treating metabolic diseases, cardiac diseases and, in general diseases associated with mitochondrial dysfunction. Assays for identification of novel ALCAT1 modulators are provided. | ||||||
| 33 | アシル補酵素A:リゾカルジオリピン・アシル基転移酵素1(ALCAT1)の調節物質およびその使用方法 | JP2017113848 | 2017-06-08 | JP6410878B2 | 2018-10-24 | シー、ユーアン |
| 34 | RPGRX連鎖性網膜変性のAAV媒介型遺伝子治療 | JP2015521593 | 2013-01-23 | JP6199965B2 | 2017-09-20 | ベルトラン,ウイリアム・エイ; アギーレ,グスタボ・デイー; ジヤコブソン,サミユエル・ジー; シデシヤン,アーサー・ブイ; リユーイン,アルフレツド・エス; ボーイ,サンフオード・エル; ハウスワース,ウイリアム・ダブリユー; デン,ウエン−タオ |
| 35 | RhoCに基づく免疫療法 | JP2014027157 | 2014-02-17 | JP5813801B2 | 2015-11-17 | マッズ ハルド アンデルセン; ペル ソル ストラテン |
| 36 | ＲＰＧＲＸ連鎖性網膜変性のＡＡＶ媒介型遺伝子治療 | JP2015521593 | 2013-01-23 | JP2015523379A | 2015-08-13 | ベルトラン，ウイリアム・エイ; アギーレ，グスタボ・デイー; ジヤコブソン，サミユエル・ジー; シデシヤン，アーサー・ブイ; リユーイン，アルフレツド・エス; ボーイ，サンフオード・エル; ハウスワース，ウイリアム・ダブリユー; デン，ウエン−タオ |
| 本明細書に記載するのは、個体の網膜色素変性症およびX連鎖性網膜色素変性症に伴う視覚喪失および他の状態を防ぎ、進行を阻止し、または改善する方法である。この方法は、正常網膜色素変性GTPase調節物質(RPGR遺伝子)またはそのフラグメントをコードする核酸配列を、個体の光受容細胞に該遺伝子産物を発現する調節配列の制御下に持つ組換えアデノ随伴ウイルス(AAV)、および製薬学的に許容され得る担体を含んでなる組成物の有効濃度を該個体に投与することを含む。【選択図】図1 | ||||||
| 37 | アシル補酵素Ａ：リゾカルジオリピン・アシル基転移酵素１（ＡＬＣＡＴ１）の調節薬およびその使用方法 | JP2014557806 | 2013-02-15 | JP2015514966A | 2015-05-21 | シー、ユーアン |
| アシル補酵素A:リゾカルジオリピン・アシル基転移酵素1(ALCAT1)の発現、機能または活性の調節剤の組成物が提供される。特にアシル補酵素A:リゾカルジオリピン・アシル基転移酵素1(ALCAT1)阻害薬は代謝性疾患、心臓疾患、およびミトコンドリア機能不全に伴う一般的疾患を処置するのに有用である。新規のアシル補酵素A:リゾカルジオリピン・アシル基転移酵素1(ALCAT1)調節剤の同定のための検定法が提供される。【選択図】図3A | ||||||
| 38 | マイトフュージン抑制剤を含む、細胞のリプログラミング促進用組成物及びその用途 | JP2016560330 | 2015-04-23 | JP6386080B2 | 2018-09-05 | チョ イェスク; ソン ミュンジン; クウォン ユジョン |
| 39 | アシル補酵素A:リゾカルジオリピン・アシル基転移酵素1(ALCAT1)の調節物質およびその使用方法 | JP2017113848 | 2017-06-08 | JP2017198689A | 2017-11-02 | シー、ユーアン |
| 【課題】アシル補酵素A:リゾカルジオリピン・アシル基転移酵素1(ALCAT1)の発現、機能または活性の調節剤の組成物を提供する。 【解決手段】代謝性疾患、心臓疾患、およびミトコンドリア機能不全に伴う一般的疾患を処置するのに有用である、アシル補酵素A:リゾカルジオリピン・アシル基転移酵素1(ALCAT1)阻害薬を提供する。また、アシル補酵素A:リゾカルジオリピン・アシル基転移酵素1(ALCAT1)調節剤の同定のための新規の検定法も提供される。 【選択図】図3A−3E |
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| 40 | マイトフュージン抑制剤を含む、細胞のリプログラミング促進用組成物及びその用途 | JP2016560330 | 2015-04-23 | JP2017502693A | 2017-01-26 | イェスク チョ; ミュンジン ソン; ユジョン クウォン |
| 本発明は、マイトフュージン遺伝子の発現抑制剤、マイトフュージンタンパク質の活性抑制剤、又はその混合物を有効成分として含む、分化した細胞から全分化能幹細胞へのリプログラミング促進用組成物及びその用途に関するものである。本発明の組成物は、全分化能幹細胞を作製するためのリプログラミングの誘導過程において、リプログラミングの誘導効率を向上させるだけでなく、リプログラミングの誘導にかかる所要時間を減少させることにより、全分化能幹細胞の産業化に求められる高効率の全分化能幹細胞の作製技術及び安定的な大量培養システムを開発するのに有効に活用され得る。また、全分化能幹細胞を維持し、全分化能幹細胞へのリプログラミングを促進できる化合物をスクリーニングするのに有効に活用することができる。 | ||||||
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