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1.发明授权Mannose receptor C type 1 (MRC1) codon optimized cell line and uses thereof 有权甘露糖受体C 1型（MRC1）密码子优化细胞系及其用途公开(公告)号：US09453847B2
公开(公告)日：2016-09-27
申请号：US13811122
申请日：2011-07-19
申请人： Juan Ruiz , Marcia Sellos-Moura , Michael F. Concino , Pan Luying , Paolo Martini , Bettina Strack-Logue , Scott Alderucci
发明人： Juan Ruiz , Marcia Sellos-Moura , Michael F. Concino , Pan Luying , Paolo Martini , Bettina Strack-Logue , Scott Alderucci
CPC分类号： G01N33/6854 , A61K38/47 , C07K14/705 , C12N9/2402 , C12Y302/01045 , G06F19/00 , G06Q50/22 , Y02A90/22 , Y02A90/26
摘要： Described herein are isolated nucleic acid molecules comprising nucleotide sequence encoding mannose receptor, C type 1 (MRC1) wherein the 5′ region of the nucleotide sequence encoding MRC1 is codon optimized; cells comprising such nucleic acid molecules; and methods of detecting antibody production, e.g., neutralizing antibody production, in a subject being treated for Gaucher disease using such cells.
摘要翻译： 本文描述了包含编码甘露糖受体，C型1（MRC1）的核苷酸序列的分离的核酸分子，其中编码MRC1的核苷酸序列的5'区被密码子优化; 包含这种核酸分子的细胞; 以及在使用这种细胞治疗戈谢病的对象中检测抗体产生的方法，例如中和抗体产生。
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2.发明申请MANNOSE RECEPTOR C TYPE 1 (MRC1) CODON OPTIMIZED CELL LINE AND USES THEREOF 有权MANNOSE受体C型1（MRC1）编码细胞优化细胞系及其用途公开(公告)号：US20130195836A1
公开(公告)日：2013-08-01
申请号：US13811122
申请日：2011-07-19
申请人： Juan A. Ruiz , Marcia Sellos-Moura , Michael F. Concino , Luying Pan , Paolo Martini , Bettina Strack-Logue , Scott Alderucci
发明人： Juan A. Ruiz , Marcia Sellos-Moura , Michael F. Concino , Luying Pan , Paolo Martini , Bettina Strack-Logue , Scott Alderucci
CPC分类号： G01N33/6854 , A61K38/47 , C07K14/705 , C12N9/2402 , C12Y302/01045 , G06F19/00 , G06Q50/22 , Y02A90/22 , Y02A90/26
摘要： Described herein are isolated nucleic acid molecules comprising nucleotide sequence encoding mannose receptor, C type 1 (MRC1) wherein the 5′ region of the nucleotide sequence encoding MRC1 is codon optimized; cells comprising such nucleic acid molecules; and methods of detecting antibody production, e.g., neutralizing antibody production, in a subject being treated for Gaucher disease using such cells.
摘要翻译： 本文描述了包含编码甘露糖受体，C型1（MRC1）的核苷酸序列的分离的核酸分子，其中编码MRC1的核苷酸序列的5'区被密码子优化; 包含这种核酸分子的细胞; 以及在使用这种细胞治疗戈谢病的对象中检测抗体产生的方法，例如中和抗体产生。
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公开(公告)号：US20140135275A1
公开(公告)日：2014-05-15
申请号：US14127074
申请日：2012-06-15
CPC分类号： C07K14/47 , A01K2217/075 , A01K2227/105 , A01K2267/0306 , A61K38/00 , A61K38/1709 , A61K47/645 , A61K49/0008 , C07K7/08 , C07K2319/07 , C07K2319/10
摘要： The present invention provides, among other things, compositions and methods for treatment of Friedrich's Ataxia based on effective targeting of a therapeutic moiety to mitochondria that can substitute for natural FXN protein activity or rescue one or more phenotypes or symptoms associated with frataxin-deficiency. In some embodiments, the present invention provides a targeted therapeutic comprising a therapeutic moiety, which is a polypeptide having an N-terminus and a C-terminus, a mitochondrial targeting sequence associated with the therapeutic moiety at the N-terminus, and a mitochondrial membrane-penetrating peptide associated with the therapeutic moiety at the C-terminus, wherein the therapeutic moiety is targeted to mitochondria upon cellular entry.
摘要翻译： 本发明尤其提供了用于治疗弗里德里希共济失调的组合物和方法，其基于可以替代天然FXN蛋白活性或拯救一种或多种与frataxin缺陷相关的表型或症状的线粒体的治疗性部分的有效靶向。 在一些实施方案中，本发明提供靶向治疗剂，其包含治疗部分，其为具有N末端和C末端的多肽，与N末端的治疗部分相关的线粒体靶向序列，以及线粒体膜 与C-末端的治疗部分相关的穿透肽，其中治疗部分在细胞进入时靶向线粒体。
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公开(公告)号：US20070269870A1
公开(公告)日：2007-11-22
申请号：US11254250
申请日：2005-10-18
申请人： George Church , Noubar Afeyan , Joseph Jacobson , Brian Baynes , Kenneth Nesmith , Brad Chapman , Bettina Strack-Logue
发明人： George Church , Noubar Afeyan , Joseph Jacobson , Brian Baynes , Kenneth Nesmith , Brad Chapman , Bettina Strack-Logue
IPC分类号： C12P19/34
CPC分类号： C12N15/66 , C12N15/10 , C12Q1/6844 , C12Q2531/113 , C12Q2521/514 , C12Q2533/107 , C12Q2521/307 , C12Q2533/101
摘要： Disclosed are methods of manufacturing synthetic DNAs, that is, DNAs made at least in significant part by chemical synthesis of nucleic acid polymers. Also provided are methods for assembling plural DNAs in the same pool by multiplexed assembly of synthetic oligonucleotides. In exemplary embodiments, the methods involve pre-amplification of one or more oligonucleotides using “universal” primers, reduction of the error rate in oligonucleotide and/or nucleic acid products, and sequence optimization and oligonucleotides design. Also provided are low-purity arrays of nucleic acids and methods for assembling nucleic acids using oligonucleotides obtained from low-purity arrays.
摘要翻译： 公开了制造合成DNA的方法，即通过核酸聚合物的化学合成至少大部分制备的DNA。 还提供了通过合成寡核苷酸的多重组装在同一池中组装多个DNA的方法。 在示例性实施方案中，所述方法包括使用“通用”引物预先扩增一种或多种寡核苷酸，降低寡核苷酸和/或核酸产物中的错误率，以及序列优化和寡核苷酸设计。 还提供了低纯度核酸阵列和使用从低纯度阵列获得的寡核苷酸组装核酸的方法。
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公开(公告)号：US09260495B2
公开(公告)日：2016-02-16
申请号：US14127074
申请日：2012-06-15
CPC分类号： C07K14/47 , A01K2217/075 , A01K2227/105 , A01K2267/0306 , A61K38/00 , A61K38/1709 , A61K47/645 , A61K49/0008 , C07K7/08 , C07K2319/07 , C07K2319/10
摘要： The present invention provides, among other things, compositions and methods for treatment of Friedrich's Ataxia based on effective targeting of a therapeutic moiety to mitochondria that can substitute for natural FXN protein activity or rescue one or more phenotypes or symptoms associated with frataxin-deficiency. In some embodiments, the present invention provides a targeted therapeutic comprising a therapeutic moiety, which is a polypeptide having an N-terminus and a C-terminus, a mitochondrial targeting sequence associated with the therapeutic moiety at the N-terminus, and a mitochondrial membrane-penetrating peptide associated with the therapeutic moiety at the C-terminus, wherein the therapeutic moiety is targeted to mitochondria upon cellular entry.
摘要翻译： 本发明尤其提供了用于治疗弗里德里希共济失调的组合物和方法，其基于可以替代天然FXN蛋白活性或拯救一种或多种与frataxin缺陷相关的表型或症状的线粒体的治疗性部分的有效靶向。 在一些实施方案中，本发明提供靶向治疗剂，其包含治疗部分，其为具有N末端和C末端的多肽，与N末端的治疗部分相关的线粒体靶向序列，以及线粒体膜 与C-末端的治疗部分相关的穿透肽，其中治疗部分在细胞进入时靶向线粒体。
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公开(公告)号：US20110318327A1
公开(公告)日：2011-12-29
申请号：US13168969
申请日：2011-06-25
申请人： Michael F. Concino , Pericles Calias , Jing Pan , Kevin Holmes , Paolo Martini , Alla Romashko , Muthuraman Meiyappan , Bohong Zhang , Andrea Iskenderian , Dianna Lundberg , Angela Norton , Bettina Strack-Logue , Yan Huang , Mary Alessandrini , Richard Pfeifer
发明人： Michael F. Concino , Pericles Calias , Jing Pan , Kevin Holmes , Paolo Martini , Alla Romashko , Muthuraman Meiyappan , Bohong Zhang , Andrea Iskenderian , Dianna Lundberg , Angela Norton , Bettina Strack-Logue , Yan Huang , Mary Alessandrini , Richard Pfeifer
CPC分类号： A61K9/0085 , A61K9/0019 , A61K9/19 , A61K38/465 , A61K47/02 , A61K47/26 , C12Y301/06013
摘要： Among other things, the present invention provides methods and compositions of treating Sanfilippo syndrome type B (Sanfilippo B) by, e.g., intrathecal (IT) administration of a Naglu protein. A suitable Naglu protein can be a recombinant, gene-activated or natural protein. In some embodiments, a suitable Naglu protein is a recombinant Naglu protein. In some embodiments, a recombinant Naglu protein is a fusion protein containing a Naglu domain and a lysosomal targeting moiety. In some embodiments, the lysosomal targeting domain is an IGF-II moiety.
摘要翻译： 除此之外，本发明提供了通过例如鞘内（IT）施用Naglu蛋白来治疗Sanfilippo综合征B型（Sanfilippo B）的方法和组合物。 合适的Naglu蛋白可以是重组的，基因激活的或天然的蛋白质。 在一些实施方案中，合适的Naglu蛋白是重组Naglu蛋白。 在一些实施方案中，重组Naglu蛋白是含有Naglu结构域和溶酶体靶向部分的融合蛋白。 在一些实施方案中，溶酶体靶向结构域是IGF-II部分。
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