1 |
用于治疗威尔森病的核酸构建体和基因治疗载体 |
CN201580076009.3 |
2015-12-17 |
CN107208105A |
2017-09-26 |
O·穆里罗索卡; G·冈萨雷斯亚瑟古拉扎; R·赫尔南德斯阿尔克瑟巴 |
本发明涉及用于治疗由铜转运ATPase2缺乏或功能障碍引起的病症,尤其是威尔森病的包含ATP7B变体的核酸构建体和基因治疗载体。根据本发明设计的AAV载体显著降低用该载体处理的威尔森病小鼠中的尿Cu排出和肝Cu含量,同时显著恢复铜蓝蛋白活性。另一方面,施用该载体导致血清转氨酶水平和肝脏组织学正常化,同时明显降低炎症浸润。 |
2 |
Methods and compositions for neuroprotection |
US15228174 |
2016-08-04 |
US09738896B2 |
2017-08-22 |
Elena Feinstein |
Disclosed herein are methods and kits useful for providing neuroprotection to neurons in the inner ear and to methods of treating inner ear diseases and disorders, including tinnitus and Mnire's disease. |
3 |
Methods and Compositions for Neuroprotection |
US15228174 |
2016-08-04 |
US20160362695A1 |
2016-12-15 |
Elena Feinstein |
Disclosed herein are methods and kits useful for providing neuroprotection to neurons in the inner ear and to methods of treating inner ear diseases and disorders, including tinnitus and Ménière's disease. |
4 |
神経保護のための方法および組成物 |
JP2014540054 |
2012-11-01 |
JP2015501634A |
2015-01-19 |
エレナ フェインスタイン |
内耳内のニューロンに神経保護を提供するために有用である方法およびキット、ならびに耳鳴およびメニエール病を含む内耳疾患および障害を治療する方法が本明細書に開示される。【選択図】なし |
5 |
COMPOSITIONS FOR USE IN NEUROPROTECTION |
EP12806730.3 |
2012-11-01 |
EP2773758B1 |
2017-06-07 |
FEINSTEIN, Elena |
|
6 |
ATP-VISUALIZING ANIMAL AND USE THEREOF |
US15111730 |
2015-01-15 |
US20180110880A1 |
2018-04-26 |
Masamichi YAMAMOTO |
Provided are a transgenic non-human mammal expressing a fusion protein, wherein the fusion protein comprises an ε subunit of an ATP synthase and two distinct fluorescent proteins as a donor and an acceptor for FRET, one of the fluorescent proteins being placed at an amino terminal moiety of the ε subunit and the other being placed at a carboxyl terminal moiety of the ε subunit, and a method of screening for an agent for preventing or treating diseases in a mammal in need thereof, comprising using an above transgenic non-human mammal. |
7 |
BIOPRODUCT FORMATION FROM A PLASMID ADDICTION SYSTEM IN THE ABSENCE OF CO-INDUCERS AND ANTIBIOTICS |
US15528301 |
2015-11-19 |
US20170306337A1 |
2017-10-26 |
F. Robert TABITA; Rick Avalos LAGUNA; Sarah Jeanne YOUNG |
Described herein are plasmid addiction systems comprising a host cell comprising one or more inactivated host cell essential genes; and a plasmid comprising one or more plasmid essential genes operably linked to a constitutively active promoter. Also described herein are metabolism-based plasmid addiction systems (PAS) comprising a host cell and a plasmid operably linked to a constitutively active promoter for producing value-based products (e.g., 1-butanol) and methods of generating PASs in microorganisms and producing 1-butanol from a PAS in the absence of antibiotics and/or co-inducers. |
8 |
Methods and compositions for neuroprotection |
US14354596 |
2012-11-01 |
US09422560B2 |
2016-08-23 |
Elena Feinstein |
Disclosed herein are methods and kits useful for providing neuroprotection to neurons in the inner ear and to methods of treating inner ear diseases and disorders, including tinnitus and Mnire's disease. |
9 |
PRODUCTION OF OIL IN VEGETATIVE TISSUES |
US14046504 |
2013-10-04 |
US20140228585A1 |
2014-08-14 |
CHRISTOPH BENNING; SANJAYA; CHANGCHENG XU |
The present invention relates to compositions and methods for providing RNA interference (RNAi) vectors comprising trigalactosyldiacylglycerol (tgd) biosynthesis enzyme constructs for increasing oil content of plants. Further, the use of tgd RNAi silencing vectors in combination with co-expression of heterologous oil regulating transcription factors, such as WRINKLED1, are contemplated to overcome the reduced growth and variable levels of embryonic lethality in plants with reduced TGD protein. Additionally, plants having reduced APS1, a gene encoding a major catalytic isoform of the small subunit of AGPase (AGP(−) plants), co-expressing a heterologous oil modulating transcription factor are contemplated for use in combination with plants having reduced TGD. Oil harvested from vegetative tissues of plants comprising vectors and genes of the present invention is contemplated for use in biofuel and biodiesel products. |
10 |
ウィルソン病の処置に使用するための核酸構築物及び遺伝子治療ベクター |
JP2017532746 |
2015-12-17 |
JP2018500904A |
2018-01-18 |
ムリージョ・サウカ,オイハナ; ゴンサレス・アセグイノラザ,グロリア; エルナンデス・アルコセバ,ルーベン |
本発明は、銅輸送ATPase2の欠損又は機能不全に関連する状態、及び特に、ウィルソン病の処置に使用するための、ATP7B変異体を含む核酸構築物及び遺伝子治療ベクターに関する。本発明に従って発明されたAAVベクターは、このベクターで処置されたウィルソン病マウスにおける尿中Cu排泄及び肝臓Cu含量を顕著に減少させた。一方で、セルロプラスミン活性は、顕著に回復された。一方、ベクターの投与により、炎症性浸潤の顕著な減少を伴って、血清トランスアミナーゼレベル及び肝臓の組織学の正常化がもたらされた。 |
11 |
神経保護のための方法および組成物 |
JP2014540054 |
2012-11-01 |
JP6118331B2 |
2017-04-19 |
フェインスタイン エレナ |
|
12 |
METHODS AND COMPOSITIONS FOR NEUROPROTECTION |
EP12806730.3 |
2012-11-01 |
EP2773758A2 |
2014-09-10 |
FEINSTEIN, Elena |
Disclosed herein are methods and kits useful for providing neuroprotection to neurons in the inner ear and to methods of treating inner ear diseases and disorders, including tinnitus and Mnire's disease. |
13 |
Production of oil in vegetative tissues |
US14046504 |
2013-10-04 |
US10006039B2 |
2018-06-26 |
Christoph Benning; Sanjaya; Changcheng Xu |
The present invention relates to compositions and methods for providing RNA interference (RNAi) vectors comprising trigalactosyldiacylglycerol (tgd) biosynthesis enzyme constructs for increasing oil content of plants. Further, the use of tgd RNAi silencing vectors in combination with co-expression of heterologous oil regulating transcription factors, such as WRINKLED1, are contemplated to overcome the reduced growth and variable levels of embryonic lethality in plants with reduced TGD protein. Additionally, plants having reduced APS1, a gene encoding a major catalytic isoform of the small subunit of AGPase (AGP(−) plants), co-expressing a heterologous oil modulating transcription factor are contemplated for use in combination with plants having reduced TGD. Oil harvested from vegetative tissues of plants comprising vectors and genes of the present invention is contemplated for use in biofuel and biodiesel products. |
14 |
NUCLEIC ACID CONSTRUCTS AND GENE THERAPY VECTORS FOR USE IN THE TREATMENT OF WILSON DISEASE |
US15537781 |
2015-12-17 |
US20170348435A1 |
2017-12-07 |
Oihana MURILLO SAUCA; Gloria GONZÁLEZ ASEGUINOLAZA; Rubén HERNÁNDEZ ALCOCEBA |
The invention relates to nucleic acid constructs and gene therapy vectors that comprise an ATP7B variant for use in the treatment of conditions associated with a deficiency or dysfunction of Copper-transporting ATPase 2, and particularly of Wilson's disease. An AAV vector devised according to the invention significantly reduced urine Cu excretion, and liver Cu content in Wilson's disease mice treated with the vector, while ceruloplasmin activity was significantly restored. On the other hand, the administration of the vector resulted in the normalization of serum transaminases' levels and of liver histology, together with a marked reduction of the inflammatory infiltrate. |
15 |
SIMILAR PERFORMANCE FROM SEEDS WITH EPIGENETIC TRAITS |
US15419700 |
2017-01-30 |
US20170223914A1 |
2017-08-10 |
Michael E Fromm |
Methods for using seeds comprising an epigenetic trait for reproducibly producing seed lots for sale with similar agronomic performance over multiple years and production cycles are provided. |
16 |
ASSAYS AND METHODS FOR CELL PROLIFERATION-TARGETED TREATMENT THERAPIES |
US15074993 |
2016-03-18 |
US20160299149A1 |
2016-10-13 |
Otto Phanstiel, IV; Deborah Altomare; Laurence von Kalm |
Assays are described which measure polyamine transport activity and markers associated with polyamine transport and metabolism. These data are then used for the selection of treatments using therapies which target polyamine transport and polyamine metabolism. The assay includes a substrate to which a protein-containing cell sample can bind, a solution comprising a first antibody specific against ATP13A3, wherein the first antibody is configured to bind to ATP13A3 protein on the substrate, a solution comprising a second antibody specific against the first antibody, the second antibody comprising an enzyme linked thereto, wherein the second antibody is configured to bind to the first antibody. The assay further includes a substrate specific to the enzyme, wherein upon combining the substrate and the enzyme, the amount of enzyme in the solution can be identified, wherein said amount of enzyme identified is proportional to the amount of ATP13A3 protein in the sample, wherein said ATP13A3 protein is indicative of polyamine transport in the cells of the sample. In addition, primary antibodies covalently attached to respective fluorophores can be used to directly measure the relative expression levels of these biomarkers in histological samples. In addition, several biomarkers are described which allow for therapy selection based upon the expression and relative ratios of specific proteins associated with polyamine transport (c-myc, ATP13A3 Cav-2, and Cav-1 as well as c-Raf). |
17 |
METHODS AND COMPOSITIONS FOR NEUROPROTECTION |
US14354596 |
2012-11-01 |
US20150031746A1 |
2015-01-29 |
Elena Feinstein |
Disclosed herein are methods and kits useful for providing neuroprotection to neurons in the inner ear and to methods of treating inner ear diseases and disorders, including tinnitus and Mnire's disease. |
18 |
윌슨병 및 기타 병태의 치료에 사용되기 위한 핵산 구조물 및 유전자 치료용 벡터 |
KR1020177019707 |
2015-12-17 |
KR1020170108951A |
2017-09-27 |
무릴로사우카,오이하나; 곤잘레즈아세귀놀라자,글로리아; 헤르난데즈알코세바,루벤 |
본발명은구리-전달 ATPase 2의결핍또는기능장애에기인하는병태, 좋기로는윌슨병을치료하는데사용되기위한, ATP7B 변이체를포함하는핵산구조물및 유전자치료용벡터에관한것이다. 본발명에따라안출된 AAV 벡터는상기벡터로처리된윌슨병마우스들에있어서소변의 Cu 배설및 간의 Cu 함량을유의적으로감소시키는한편, 세룰로플라스민활성은유의적으로복구시켰다. 다른한편, 상기벡터를투여하자염증성침윤물의현저한감소와함께, 간조직학및 혈청트랜스아미나제수준이정상화되었다. |
19 |
신경보호를 위한 방법 및 조성물 |
KR1020147010978 |
2012-11-01 |
KR1020140091683A |
2014-07-22 |
파인스타인엘레나 |
본 출원에는 내이 내의 신경들에 대하여 그리고 이명 및 메니에르병을 포함하여 내이 질환들 및 장애들을 치료하는 방법에 대하여 신경보호를 제공하는 데 유용한 방법 및 킷트들이 기술된다.
|
20 |
METHODS AND COMPOSITIONS FOR NEUROPROTECTION |
PCT/US2012062894 |
2012-11-01 |
WO2013067076A3 |
2013-07-04 |
FEINSTEIN ELENA |
Disclosed herein are methods and kits useful for providing neuroprotection to neurons in the inner ear and to methods of treating inner ear diseases and disorders, including tinnitus and Mnire's disease. |