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1. 发明授权

US09598703B2 Capsid-free AAV vectors, compositions, and methods for vector production and gene delivery 有权
公开(公告)号：US09598703B2
公开(公告)日：2017-03-21
申请号：US14004379
申请日：2012-03-12
申请人： Luis Garcia , Cyriaque Beley , Thomas Voit , Robert Michael Kotin , Lina Li
发明人： Luis Garcia , Cyriaque Beley , Thomas Voit , Robert Michael Kotin , Lina Li
IPC分类号： C12N15/11 , A61K35/12 , A61N1/30 , C12N15/86
CPC分类号： C12N15/86 , C12N2750/14143 , C12N2750/14171 , C12N2800/24
摘要： An isolated linear nucleic acid molecule comprising in this order: a first adeno-associated virus (AAV) inverted terminal repeat (ITR), a nucleotide sequence of interest and a second AAV ITR, wherein said nucleic acid molecule is devoid of AAV capsid protein coding sequences. The said nucleic acid molecule can be applied to a host at repetition without eliciting an immune response. Methods for producing and purifying this nucleic acid molecule, and use of the same for therapeutic purposes are also provided.

2. 发明申请

US20140107186A1 CAPSID-FREE AAV VECTORS, COMPOSITIONS, AND METHODS FOR VECTOR PRODUCTION AND GENE DELIVERY 有权
标题翻译：不含CAPSID的AAV矢量，组合物和方法用于矢量生产和基因分送
公开(公告)号：US20140107186A1
公开(公告)日：2014-04-17
申请号：US14004379
申请日：2012-03-12
申请人： Luis Garcia , Cyriaque Beley , Thomas Voit , Robert Michael Kotin
发明人： Luis Garcia , Cyriaque Beley , Thomas Voit , Robert Michael Kotin
IPC分类号： C12N15/86
CPC分类号： C12N15/86 , C12N2750/14143 , C12N2750/14171 , C12N2800/24
摘要： An isolated linear nucleic acid molecule comprising in this order: a first adeno-associated virus (AAV) inverted terminal repeat (ITR), a nucleotide sequence of interest and a second AAV ITR, wherein said nucleic acid molecule is devoid of AAV capsid protein coding sequences. The said nucleic acid molecule can be applied to a host at repetition without eliciting an immune response. Methods for producing and purifying this nucleic acid molecule, and use of the same for therapeutic purposes are also provided.
摘要翻译：一种分离的线性核酸分子，其以以下顺序包含：第一腺相关病毒（AAV）倒置末端重复（ITR），目的核苷酸序列和第二AAV ITR，其中所述核酸分子缺乏AAV衣壳蛋白编码序列。所述核酸分子可以重复施用于宿主而不引发免疫应答。还提供了用于产生和纯化该核酸分子的方法及其用于治疗目的的用途。

3. 发明授权

US09080170B2 Modified U7 snRNAs for treatment of neuromuscular diseases 有权
标题翻译：修饰的U7 snRNA用于治疗神经肌肉疾病
公开(公告)号：US09080170B2
公开(公告)日：2015-07-14
申请号：US13635356
申请日：2011-03-17
申请人： Luis Garcia , Denis Furling , Cyriaque Beley , Thomas Voit
发明人： Luis Garcia , Denis Furling , Cyriaque Beley , Thomas Voit
IPC分类号： C07H21/04 , C12N15/11
CPC分类号： C12N15/111 , C12N2310/11 , C12N2310/531 , C12N2320/33 , C12N2320/34 , C12N2320/50 , C12N2330/51
摘要： The present invention relates to a method to improve the activity of engineered U7 snRNAs used in the context of RNA-based therapeutics; particularly in exon skipping, exon inclusion, and mRNA eradication strategies. The resulting modified U7 snRNAs are useful for treating neuromuscular diseases, in particular Duchenne neuromuscular dystrophy, myotonic dystrophy DM1 and spinal muscular atrophy.
摘要翻译：本发明涉及一种改善在基于RNA的疗法的上下文中使用的工程化U7 snRNA的活性的方法; 特别是在外显子跳跃，外显子包含和mRNA消除策略。所得修饰的U7 snRNA可用于治疗神经肌肉疾病，特别是杜烯神经肌营养不良症，强直性营养不良DM1和脊髓性肌肉萎缩。

4. 发明申请

US20130045538A1 MODIFIED U7 SNRNAS FOR TREATMENT OF NEUROMUSCULAR DISEASES 有权
标题翻译：用于治疗神经性疾病的改良U7 SNRNA
公开(公告)号：US20130045538A1
公开(公告)日：2013-02-21
申请号：US13635356
申请日：2011-03-17
申请人： Luis Garcia , Denis Furling , Cyriaque Beley , Thomas Voit
发明人： Luis Garcia , Denis Furling , Cyriaque Beley , Thomas Voit
IPC分类号： C07H21/02 , C12N15/113 , C12N15/85 , C12N15/63 , C12N5/10
CPC分类号： C12N15/111 , C12N2310/11 , C12N2310/531 , C12N2320/33 , C12N2320/34 , C12N2320/50 , C12N2330/51
摘要： The present invention relates to a method to improve the activity of engineered U7 snRNAs used in the context of RNA-based therapeutics; particularly in exon skipping, exon inclusion, and mRNA eradication strategies. The resulting modified snRNAs are useful for treating neuromuscular diseases, in particular Duchenne neuromuscular dystrophy, myotonic dystrophy DM1 and spinal muscular atrophy.
摘要翻译：本发明涉及一种改善在基于RNA的疗法的上下文中使用的工程化U7 snRNA的活性的方法; 特别是在外显子跳跃，外显子包含和mRNA消除策略。所产生的修饰的snRNA可用于治疗神经肌肉疾病，特别是Duchenne神经肌肉营养不良症，强直性营养不良DM1和脊髓性肌肉萎缩。

5. 发明授权

US09546112B2 Methods of increasing efficiency of vector penetration of target tissue 有权
标题翻译：提高靶组织载体渗透效率的方法
公开(公告)号：US09546112B2
公开(公告)日：2017-01-17
申请号：US13636596
申请日：2011-03-22
申请人： Thomas Voit , Luis Garcia , Jérôme Denard , Fedor Svinartchouk
发明人： Thomas Voit , Luis Garcia , Jérôme Denard , Fedor Svinartchouk
IPC分类号： C12N15/00 , A61K35/12 , A61N1/30 , C04B35/10 , A61K38/17 , A61K38/21 , A61K45/06 , C07K16/08 , C07K16/18 , C12N15/86 , A61K31/522 , A61K38/00 , A61K48/00 , C04B35/14 , C04B35/565 , C04B35/622 , C07K14/005 , C12N7/00
CPC分类号： C04B35/10 , A61K31/522 , A61K38/00 , A61K38/1709 , A61K38/21 , A61K45/06 , A61K48/0008 , A61K48/0058 , C04B35/14 , C04B35/565 , C04B35/622 , C07K14/005 , C07K16/081 , C07K16/18 , C12N7/00 , C12N15/86 , C12N2750/14122 , C12N2750/14143 , C12N2750/14145 , C12N2750/14151 , C12N2810/6027 , A61K2300/00
摘要： Methods for increasing the efficiency of target tissue penetration of an adeno-associated virus (AAV) vector in a patient are provided. In some aspects, the methods involve inhibiting the interaction of the serum protein galectin 3 binding protein (G3BP) with AAV vector. Further provided are methods for reducing tissue distribution of a virus or for neutralizing a virus harbored by an organ destined for transplant, or newly transplanted, by administering a composition comprising G3BP.
摘要翻译：提供了增加患者中腺相关病毒（AAV）载体靶组织穿透效率的方法。在某些方面，该方法涉及抑制血清蛋白半乳凝素3结合蛋白（G3BP）与AAV载体的相互作用。进一步提供的是通过施用包含G3BP的组合物来减少病毒的组织分布或用于中和由待移植或新移植的器官所携带的病毒的方法。

6. 发明申请

US20130189225A1 METHODS OF INCREASING EFFICIENCY OF VECTOR PENETRATION OF TARGET TISSUE 有权
标题翻译：提高靶向组织渗透效率的方法
公开(公告)号：US20130189225A1
公开(公告)日：2013-07-25
申请号：US13636596
申请日：2011-03-22
申请人： Thomas Voit , Luis Garcia , Jérôme Denard , Fedor Svinartchouk
发明人： Thomas Voit , Luis Garcia , Jérôme Denard , Fedor Svinartchouk
IPC分类号： A61K48/00 , A61K38/00 , A61K31/522 , A61K38/21
CPC分类号： C04B35/10 , A61K31/522 , A61K38/00 , A61K38/1709 , A61K38/21 , A61K45/06 , A61K48/0008 , A61K48/0058 , C04B35/14 , C04B35/565 , C04B35/622 , C07K14/005 , C07K16/081 , C07K16/18 , C12N7/00 , C12N15/86 , C12N2750/14122 , C12N2750/14143 , C12N2750/14145 , C12N2750/14151 , C12N2810/6027 , A61K2300/00
摘要： Methods for increasing the efficiency of target tissue penetration of an adeno-associated virus (AAV) vector in a patient are provided. In some aspects, the methods involve inhibiting the interaction of the serum protein galectin 3 binding protein (G3BP) with AAV vector. Further provided are methods for reducing tissue distribution of a virus or for neutralizing a virus harbored by an organ destined for transplant, or newly transplanted, by administering a composition comprising G3BP.
摘要翻译：提供了增加患者中腺相关病毒（AAV）载体靶组织穿透效率的方法。在某些方面，该方法涉及抑制血清蛋白半乳凝素3结合蛋白（G3BP）与AAV载体的相互作用。进一步提供的是通过施用包含G3BP的组合物来减少病毒的组织分布或用于中和由目的地移植或新移植的器官所携带的病毒的方法。

7. 发明申请

US20120149756A1 TRICYCLO-DNA ANTISENSE OLIGONUCLEOTIDES, COMPOSITIONS, AND METHODS FOR THE TREATMENT OF DISEASE 审中-公开
标题翻译： TRICYCLO-DNA抗体寡核苷酸，组合物和治疗疾病的方法
公开(公告)号：US20120149756A1
公开(公告)日：2012-06-14
申请号：US13263949
申请日：2010-04-09
申请人： Daniel Schümperli , Christian Leumann , Denis Furling , Luis Garcia , Thomas Voit
发明人： Daniel Schümperli , Christian Leumann , Denis Furling , Luis Garcia , Thomas Voit
IPC分类号： A61K31/712 , A61P21/00 , A61P25/00 , C07H21/04 , C12N5/071
CPC分类号： C12N15/113 , A61K31/711 , A61K31/712 , C12N15/111 , C12N2310/11 , C12N2310/3231 , C12N2320/33
摘要： Provided are tricyclo-DNA (tc-DNA) AON and methods employing tc-DNA AON for modifying splicing events that occur during pre-mRNA processing. Tricyclo-DNA (tc-DNA) AON are described that may be used to facilitate exon skipping or to mask intronic silencer sequences and/or terminal stem-loop sequences during pre-mRNA processing and to target RNase-mediated destruction of processed mRNA. Tc-DNA AON described herein may be used in methods for the treatment of Duchenne Muscular Dystrophy by skipping a mutated exon 23 or exon 51 within a dystrophin gene to restore functionality of a dystrophin protein; in methods for the treatment of Spinal Muscular Atrophy by masking an intronic silencing sequence and/or a terminal stem-loop sequence within an SMN2 gene to yield modified functional SMN2 protein, including an amino acid sequence encoded by exon 7, which is capable of at least partially complementing a non-functional SMN1 protein; and in methods for the treatment of Steinert's Myotonic Dystrophy by targeting the destruction of a mutated DM1 mRNA comprising 3′-terminal CUG repeats.
摘要翻译：提供三环DNA（tc-DNA）AON和使用tc-DNA AON来改变在mRNA前处理期间发生的剪接事件的方法。描述了Tricyclo-DNA（tc-DNA）AON，其可用于促进外显子跳跃或在mRNA前处理期间掩盖内含子沉默子序列和/或末端茎环序列，并靶向核糖核酸酶介导的加工mRNA的破坏。本文所述的Tc-DNA AON可用于通过跳过肌营养不良蛋白基因内突变外显子23或外显子51来恢复肌营养不良蛋白功能的方法来治疗杜氏肌营养不良症; 在通过掩蔽SMN2基因内的内含子沉默序列和/或末端茎环序列来治疗脊髓性肌萎缩的方法中，以产生修饰的功能性SMN2蛋白，包括由外显子7编码的氨基酸序列，其能够在最少部分补充非功能性SMN1蛋白; 以及用于通过靶向包含3'-末端CUG重复序列的突变的DM1mRNA的破坏来治疗Steinert's强直性营养不良的方法。

8. 发明申请

US20120172415A1 Exon Skipping Therapy for Functional Amelioration of Semifunctional Dystrophin in Becker and Duchenne Muscular Dystrophy 审中-公开
标题翻译： Becker和Duchenne肌营养不良功能改善半功能肌营养不良蛋白的外显子跳过疗法
公开(公告)号：US20120172415A1
公开(公告)日：2012-07-05
申请号：US13393004
申请日：2010-08-31
申请人： Thomas Voit , Luis Garcia , Valerie Robin , Patrick Dreyfus
发明人： Thomas Voit , Luis Garcia , Valerie Robin , Patrick Dreyfus
IPC分类号： A61K31/7088 , A61P9/00 , A61P21/00 , C12N15/113 , C07K14/47
CPC分类号： C12N15/113 , C12N2310/11 , C12N2310/321 , C12N2310/3231 , C12N2320/33 , C12N2310/3521
摘要： Methods for stabilizing unstable proteins or for restoring functionality to non-functional or poorly functioning (semi-functional) proteins using exon skipping technology are provided. The methods involve the administration of antisense oligonucleotides to cause exon skipping, thereby removing one or more exons responsible for protein instability or lack of functionality. For example, exons encoding protease recognition sites may be removed. The method is useful for treating diseases caused by protein instability, such as Becker Muscular Dystrophy, or for treating Duchenne Muscular Distrophy patients with semi-functional dystrophin due to treatment with other exon skipping or stop codon readthrough therapies.
摘要翻译：提供了使用外显子跳过技术来稳定不稳定蛋白质或恢复非功能或功能不良（半功能）蛋白功能的方法。所述方法包括给予反义寡核苷酸引起外显子跳跃，从而去除负责蛋白质不稳定或缺乏功能的一个或多个外显子。例如，可以去除编码蛋白酶识别位点的外显子。该方法可用于治疗由蛋白质不稳定性引起的疾病，例如贝克肌营养不良症，或用于治疗具有半功能性肌营养不良蛋白的Duchenne肌肉萎缩症患者，这是由于其他外显子跳过或终止密码子阅读疗法治疗。

9. 发明授权

US09303078B2 Nucleic acid molecules and methods for exchanging exon(s) by transsplicing 有权
标题翻译：核酸分子和通过transsplicing交换外显子的方法
公开(公告)号：US09303078B2
公开(公告)日：2016-04-05
申请号：US13500612
申请日：2010-10-08
申请人： Luis Garcia , Stéphanie Lorain
发明人： Luis Garcia , Stéphanie Lorain
IPC分类号： C12N15/00 , C12N5/00 , C07K14/47 , A61K48/00 , C12N15/85
CPC分类号： C07K14/4708 , A61K48/005 , C12N15/85 , C12N2840/445 , C12N2840/85
摘要： The present invention provided methods and compositions for generating novel nucleic acid molecules through targeted spliceosome mediated simple or double trans-splicing. The compositions of the invention include pre-trans-splicing molecules (PTMs) designed to interact with a target precursor messenger RNA molecule (target pre-mRNA) and to mediate a simple or double trans-splicing reaction resulting in the generation of a novel chimeric RNA molecule (Chimeric RNA).
摘要翻译：本发明提供了通过靶向剪接体介导的简单或双转录产生新型核酸分子的方法和组合物。本发明的组合物包括被设计为与靶前体信使RNA分子（靶前mRNA）相互作用并且介导简单或双重反式剪接反应的预转录分子（PTM），导致产生新的嵌合 RNA分子（嵌合RNA）。

10. 发明授权

US09198185B2 Mechanism for aggregating uplink interference information for use in multi-carrier systems 有权
标题翻译：用于聚合用于多载波系统的上行链路干扰信息的机制
公开(公告)号：US09198185B2
公开(公告)日：2015-11-24
申请号：US13577979
申请日：2010-02-10
申请人： Istvan Zsolt Kovacs , Frank Frederiksen , Klaus Ingemann Pedersen , Luis Garcia
发明人： Istvan Zsolt Kovacs , Frank Frederiksen , Klaus Ingemann Pedersen , Luis Garcia
IPC分类号： H04B7/216 , H04W72/08 , H04B1/707 , H04W24/02 , H04W72/04 , H04W84/04 , H04L5/00
CPC分类号： H04W72/082 , H04B1/707 , H04L5/001 , H04L5/0073 , H04L5/0091 , H04W24/02 , H04W72/0413 , H04W84/045
摘要： There is proposed a mechanism by means of which information can be generated and used for estimating and identifying at each base transceiver station of a communication network the set of other base transceiver stations generating interference on certain monitored component carriers used by the base transceiver station, i.e. information for the generation of the incoming uplink background interference matrix. For this purpose, a specific communication information set, referred to as uplink radio load map, is determined which informs about an average (time/frequency) and aggregated (over all or group of served UEs) uplink load measure per component carrier. Based on the uplink radio load map received via suitable signaling from other base transceiver stations, each base transceiver station can determine the incoming uplink background interference matrix and initiate, if necessary, suitable measures for reducing interference per component carrier.
摘要翻译：提出了一种机制，通过该机制可以产生信息并用于在通信网络的每个基站收发台处估计和识别该基站收发信机所使用的特定监视分量载波上的干扰的其他基站收发信机的集合，即用于生成输入上行背景干扰矩阵的信息。为此，确定称为上行链路无线电负载映射的特定通信信息集，其通知关于每个分量载波的上行链路负载测量的平均（时间/频率）和聚合（在全部或一组被服务UE上）。基于通过来自其他基站收发台的适当信令接收的上行链路无线电负载图，每个基站收发台可以确定输入的上行链路背景干扰矩阵，并且如有必要，启动减少每个分量载波干扰的适当措施。

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