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公开(公告)号：US20180015116A1
公开(公告)日：2018-01-18
申请号：US15625648
申请日：2017-06-16
发明人： Michael Heartlein , Braydon Charles Guild , Frank DeRosa , Carsten Rudolph , Christian Plank , Lianne Smith
IPC分类号： A61K31/713 , A61K9/127 , A61K31/7115 , A61K31/7105 , C12N9/14 , C07K14/705 , A61K9/51 , A61K9/00 , A61K48/00 , C12N15/88 , C07H21/02 , C07K14/47 , C12N15/63
摘要： Materials, formulations, production methods, and methods for delivery of CFTR mRNA for induction of CFTR expression, including in the mammalian lung are provided. The present invention is particularly useful for treating cystic fibrosis.
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公开(公告)号：US09668980B2
公开(公告)日：2017-06-06
申请号：US14790562
申请日：2015-07-02
发明人： Frank DeRosa , Shrirang Karve , Michael Heartlein
IPC分类号： A61K31/7105 , A61K9/50 , A61K9/127 , A61K31/713
CPC分类号： A61K9/5089 , A61K9/1271 , A61K9/1272 , A61K9/1277 , A61K9/5015 , A61K31/713
摘要： The present invention provides an improved process for lipid nanoparticle formulation and mRNA encapsulation. In some embodiments, the present invention provides a process of encapsulating messenger RNA (mRNA) in lipid nanoparticles comprising a step of mixing a mRNA solution and a lipid solution, wherein the mRNA solution and/or the lipid solution are at a pre-determined temperature greater than ambient temperature.
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公开(公告)号：US20160201064A1
公开(公告)日：2016-07-14
申请号：US14911849
申请日：2014-08-15
发明人： Fatih Ozsolak
IPC分类号： C12N15/113
CPC分类号： C12N15/1137 , C07K14/47 , C12N15/113 , C12N2310/11 , C12N2310/321 , C12N2310/322 , C12N2310/3231 , C12N2310/34 , C12N2310/351 , C12N2310/3513 , C12N2320/30
摘要： Provided herein are compositions and methods for increasing Frataxin (FXN) expression. Compositions and methods for treating Friedrich's ataxia are also provided.
摘要翻译： 本文提供用于增加Frataxin（FXN）表达的组合物和方法。 还提供了治疗弗里德里希共济失调的组合物和方法。
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4.发明申请公开(公告)号：US20150232847A1
公开(公告)日：2015-08-20
申请号：US14700555
申请日：2015-04-30
发明人： FATIH OZSOLAK
IPC分类号： C12N15/113
CPC分类号： C12N15/113 , A61K48/00 , C12N15/11 , C12N15/111 , C12N15/63 , C12N15/67 , C12N2310/11 , C12N2310/317 , C12N2310/321 , C12N2310/322 , C12N2310/3231 , C12N2310/531 , C12N2320/51 , C12N2830/50
摘要： Aspects of the invention relate to methods for increasing gene expression in a targeted manner. In some embodiments, methods and compositions are provided that are useful for posttranscriptionally altering protein and/or RNA levels in a targeted manner. Aspects of the invention disclosed herein provide methods and compositions that are useful for protecting RNAs from degradation (e.g., exonuclease mediated degradation).
摘要翻译： 本发明的方面涉及以目标方式增加基因表达的方法。 在一些实施方案中，提供了用于以靶向方式转录后改变蛋白质和/或RNA水平的方法和组合物。 本文公开的本发明的方面提供了可用于保护RNA免于降解的方法和组合物（例如，外切核酸酶介导的降解）。
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公开(公告)号：US20150225715A1
公开(公告)日：2015-08-13
申请号：US14700529
申请日：2015-04-30
发明人： FATIH OZSOLAK
IPC分类号： C12N15/11
CPC分类号： C12N15/113 , A61K48/00 , C12N15/11 , C12N15/111 , C12N15/63 , C12N15/67 , C12N2310/11 , C12N2310/317 , C12N2310/321 , C12N2310/322 , C12N2310/3231 , C12N2310/531 , C12N2320/51 , C12N2830/50
摘要： Aspects of the invention relate to methods for increasing gene expression in a targeted manner. In some embodiments, methods and compositions are provided that are useful for posttranscriptionally altering protein and/or RNA levels in a targeted manner. Aspects of the invention disclosed herein provide methods and compositions that are useful for protecting RNAs from degradation (e.g., exonuclease mediated degradation).
摘要翻译： 本发明的方面涉及以目标方式增加基因表达的方法。 在一些实施方案中，提供了用于以靶向方式转录后改变蛋白质和/或RNA水平的方法和组合物。 本文公开的本发明的方面提供了可用于保护RNA免于降解的方法和组合物（例如，外切核酸酶介导的降解）。
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公开(公告)号：US20150152410A1
公开(公告)日：2015-06-04
申请号：US14401237
申请日：2013-05-16
申请人： RaNA Therapeutics, Inc. , The General Hospital Corporation d/b/a Massachusetts General Hospital
IPC分类号： C12N15/113 , A61K31/7115 , A61K31/712 , A61K47/48 , A61K31/7125
CPC分类号： C12N15/113 , A61K31/7115 , A61K31/712 , A61K31/7125 , A61K47/555 , A61K47/61 , A61K47/64 , A61K47/6455 , C07H21/00 , C07H21/02 , C07H21/04 , C12N2310/11 , C12N2310/315 , C12N2310/321 , C12N2310/3231 , C12N2310/3521
摘要： Aspects of the invention provide single stranded oligonucleotides for activating or enhancing expression of MECP2. Further aspects provide compositions and kits comprising single stranded oligonucleotides for activating or enhancing expression of MECP2. Methods for modulating expression of MECP2 using the single stranded oligonucleotides are also provided. Further aspects of the invention provide methods for selecting a candidate oligonucleotide for activating or enhancing expression of MECP2.
摘要翻译： 本发明的方面提供用于激活或增强MECP2表达的单链寡核苷酸。 其它方面提供了包含用于激活或增强MECP2表达的单链寡核苷酸的组合物和试剂盒。 还提供了使用单链寡核苷酸调节MECP2表达的方法。 本发明的其它方面提供了选择用于激活或增强MECP2表达的候选寡核苷酸的方法。
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公开(公告)号：US20170211065A1
公开(公告)日：2017-07-27
申请号：US15416100
申请日：2017-01-26
IPC分类号： C12N15/113
CPC分类号： C12N15/113 , C12N2310/11 , C12N2310/113 , C12N2310/315 , C12N2310/3231 , C12N2310/341 , C12N2310/346 , C12N2310/51 , C12N2310/52
摘要： The disclosure provides multimeric oligonucleotide compounds, comprising two or more target-specific oligonucleotides (e.g., antisense oligonucleotides (ASOs)), each being resistant to cleavage, and linked together by a cleavable linker. In particular, two or more linked target-specific oligonucleotides, each to a different target, allows concomitant inhibition of multiple genes' expression levels, while exhibiting favorable pharmacokinetic and pharmacodynamic properties. Methods of making and uses of the described compounds are also provided
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8.发明申请公开(公告)号：US20150252364A1
公开(公告)日：2015-09-10
申请号：US14401194
申请日：2013-05-16
申请人： RaNA Therapeutics, Inc. , The General Hospital Corporation d/b/a Massachusetts General Hospital
IPC分类号： C12N15/113
CPC分类号： C12N15/113 , A61K31/7088 , C07H21/00 , C12N2310/11 , C12N2310/315 , C12N2310/3231 , C12N2310/343 , C12N2310/3517 , C12N2310/321 , C12N2310/3521 , C12N2310/322 , C12N2310/3533
摘要： Aspects of the invention provide single stranded oligonucleotides for activating or enhancing expression of SMN1 or SMN2. Further aspects provide compositions and kits comprising single stranded oligonucleotides for activating or enhancing expression of SMN1 or SMN2 that comprises exon 7. Methods for modulating expression of SMN1 or SMN2 using the single stranded oligonucleotides are also provided. Further aspects of the invention provide methods for selecting a candidate oligonucleotide for activating or enhancing expression of SMN1 or SMN2.
摘要翻译： 本发明的方面提供用于激活或增强SMN1或SMN2表达的单链寡核苷酸。 另外的方面提供了包含用于激活或增强包含外显子7的SMN1或SMN2表达的单链寡核苷酸的组合物和试剂盒。还提供了使用单链寡核苷酸调节SMN1或SMN2表达的方法。 本发明的其它方面提供了选择用于激活或增强SMN1或SMN2表达的候选寡核苷酸的方法。
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公开(公告)号：US20150232858A1
公开(公告)日：2015-08-20
申请号：US14700269
申请日：2015-04-30
发明人： Fatih Ozsolak
IPC分类号： C12N15/113
CPC分类号： C12N15/1137 , A61K9/19 , A61K31/7088 , C12N15/111 , C12N2310/11 , C12N2310/113 , C12N2310/141 , C12N2310/321 , C12N2310/3231 , C12N2320/30
摘要： Provided herein are oligonucleotides complementary to euchromatic regions of target genes that are useful for increasing expression of the target genes; related compositions and methods are also provided.
摘要翻译： 本文提供了与用于增加靶基因表达有用的靶基因的染色体区互补的寡核苷酸; 还提供了相关的组合物和方法。
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10.发明申请METHODS FOR SELECTIVE TARGETING OF HETEROCHROMATIN FORMING NON-CODING RNA 审中-公开选择性取代形成非编码RNA的异黄酮的方法公开(公告)号：US20150225722A1
公开(公告)日：2015-08-13
申请号：US14699130
申请日：2015-04-29
发明人： Fatih Ozsolak
IPC分类号： C12N15/113
CPC分类号： C12N15/113 , C12N2310/113 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/3231 , C12N2310/341 , C12N2310/346 , C12N2310/3521 , C12N2310/322 , C12N2310/3533
摘要： Provided herein are oligonucleotides that are useful for modulating the heterochromatin state of genes; related compositions and methods are also provided. In some embodiments, methods are provided for treating a disease associated with heterochromatin formation, including diseases associated with repeat expansion within genes.
摘要翻译： 本文提供了可用于调节基因的异染色质状态的寡核苷酸; 还提供了相关的组合物和方法。 在一些实施方案中，提供了用于治疗与异染色质形成相关的疾病的方法，包括与基因内重复扩增相关的疾病。
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