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1. 发明申请

WO2005070948A1 CORRECTION OF ALPHA-1-ANTITRYPSIN GENETIC DEFECTS USING SPLICEOSOME MEDIATED RNA TRANS SPLICING 审中-公开
标题翻译：使用SPLICEOSOME介导的RNA转移分离的ALPHA-1-抗体基因缺陷的修正
公开(公告)号：WO2005070948A1
公开(公告)日：2005-08-04
申请号：PCT/US2005/002549
申请日：2005-01-21
申请人： INTRONN, INC. , PUTTARAJU, Madaiah , OTTO, Edward , GARCIA-BLANCO, Mariano, A. , MCGARRITY, Gerard, J. , TEMPLE, Gary, F. , COTE, Collette , MITCHELL, Lloyd, G. , MANSFIELD, Gary, S.
发明人： PUTTARAJU, Madaiah , OTTO, Edward , GARCIA-BLANCO, Mariano, A. , MCGARRITY, Gerard, J. , TEMPLE, Gary, F. , COTE, Collette , MITCHELL, Lloyd, G. , MANSFIELD, Gary, S.
IPC分类号： C07H21/02
CPC分类号： C12N15/10 , C12N15/102 , C12P19/34
摘要： The present invention provides methods and compositions for generating novel nucleic acid molecules through targeted spliceosomal mediated RNA trans -splicing. The compositions of the invention include pre-trans -splicing molecules (PTMs) designed to interact with a SERPINAI target precursor messenger RNA molecule (target pre-mRNA) and mediate a trans -splicing reaction resulting in the generation of a novel chimeric RNA molecule (chimeric RNA). In particular, the PTMs of the present invention include those genetically engineered to interact with SERPINA1 target pre-mRNA so as to result in correction of SERPINAI genetic defects responsible for AAT deficiency. The PTMs of the invention may also comprise sequences that are processed out of the PTM to yield duplex siRNA molecules directed specifically to mutant SERPIN Al mRNAs. Such duplexed siRNAs are designed to reduce the accumulation of toxic AAT protein in liver cells. The methods and compositions of the present invention can be used in gene therapy for correction of SERPINAI disorders such as AAT deficiency.
摘要翻译：本发明提供了通过靶向剪接体介导的RNA转接产生新的核酸分子的方法和组合物。本发明的组合物包括设计为与SERPINAI靶前体信使RNA分子（靶前体mRNA）相互作用的转录前分子（PTM），并介导导致产生新型嵌合RNA分子的反式剪接反应（嵌合RNA）。特别地，本发明的PTM包括经遗传工程改造以与SERPINA1靶前体mRNA相互作用，从而导致对负责AAT缺陷的SERPINAI遗传缺陷的校正。本发明的PTM还可以包含从PTM中加工得到的特异性引导突变体SERPIN A1 mRNA的双链体siRNA分子的序列。这样的双链体siRNA被设计为减少肝细胞中有毒的AAT蛋白的积累。本发明的方法和组合物可用于基因治疗以纠正SERPINAI病症如AAT缺陷。

2. 发明申请

WO2005056762A2 TRUNCATED CMV PROMOTERS AND VECTORS CONTAINING SAME 审中-公开
标题翻译：交叉CMV促销者和包含它的向量
公开(公告)号：WO2005056762A2
公开(公告)日：2005-06-23
申请号：PCT/US2004/040717
申请日：2004-12-06
申请人： UNIVERSITY OF IOWA RESEARCH FOUNDATION , THE GOVERNMENT OF THE UNITED STATES OF AMERICA , OSTEDGAARD, Lynda, S. , STINSKI, Mark, F. , CHIORINI, John, A. , WELSH, Michael, J.
发明人： OSTEDGAARD, Lynda, S. , STINSKI, Mark, F. , CHIORINI, John, A. , WELSH, Michael, J.
IPC分类号： C12N
CPC分类号： C12N15/86 , A61K38/1709 , A61K48/00 , A61K48/0066 , C12N7/00 , C12N15/85 , C12N2750/14111 , C12N2750/14143 , C12N2750/14171 , C12N2830/00 , C12N2830/60
摘要： The present invention relates to nucleic acid molecules comprising certain truncated forms of the human cytomegalovirus (CMV) immediate-early enhancer-promoter, either alone or operably linked to transgenes of interest, including those encoding partially-deleted CFTR proteins. This invention further relates to vectors comprising these nucleic acid molecules and host cells transformed by such vectors. The nucleic acid molecules, vectors and transformed host cells of the present invention are useful for treating a variety of genetic, metabolic and acquired diseases, including inter alia cystic fibrosis (CF) airway disease.
摘要翻译：本发明涉及包含某些截短形式的人巨细胞病毒（CMV）立即早期增强子启动子的核酸分子，其单独或可操作地连接到感兴趣的转基因，包括编码部分缺失的CFTR蛋白的转基因。本发明还涉及包含这些核酸分子和由这些载体转化的宿主细胞的载体。本发明的核酸分子，载体和转化的宿主细胞可用于治疗各种遗传，代谢和获得性疾病，包括尤其是囊性纤维化（CF）气道疾病。

3. 发明申请

WO2004067608A2 METHOD FOR PRODUCING SUBMICRON POLYTETRAFLUOROETHYLENE POWDER AND PRODUCTS THEREOF 审中-公开
标题翻译：生产亚磷酸三氟乙烯粉末的方法及其制备方法
公开(公告)号：WO2004067608A2
公开(公告)日：2004-08-12
申请号：PCT/US2004/002209
申请日：2004-01-26
申请人： SHAMROCK TECHNOLOGIES, INC. , CODY, Charles, A. , NEUBERG, William , SUI, Manshi , AWAD, Youssef , CAREY, Paul
发明人： CODY, Charles, A. , NEUBERG, William , SUI, Manshi , AWAD, Youssef , CAREY, Paul
IPC分类号： C08J
CPC分类号： C08F6/20 , C08F6/14 , C08F14/26 , C08F214/26 , Y10T428/2982 , C08L27/18 , C08F2/04
摘要： A method for treating polytetrafluoroethylene (PTFE) in its reactor latex form to produce a dry submicron PTFE powder that remains stable without rheology modifiers, surfactants, wetting agents, pH adjusters or other stabilizing additives. Reactor latex PTFE formed during an emulsion polymerization process can be irradiated, with an electron beam or gamma rays, during or after the polymerization to form a product where the dry submicron PTFE powder is free-flowing, tends not to self-agglomerate and tends not to dust into the air upon handling so that the PTFE is readily dispersible when placed in a desired application system or medium.
摘要翻译：一种在其反应器胶乳形式中处理聚四氟乙烯（PTFE）的方法，以产生干燥亚微米PTFE粉末，其在没有流变改性剂，表面活性剂，润湿剂，pH调节剂或其它稳定添加剂的情况下保持稳定。在乳液聚合过程中形成的反应器胶乳PTFE可以在聚合过程中或之后用电子束或γ射线照射，形成干亚微米PTFE粉末自由流动的产品，倾向于不自聚集，并且不趋向于在处理时灰尘进入空气中，使得PTFE在放置在所需的应用系统或介质中时容易分散。

4. 发明申请

WO2002079397A2 VIRUS DERIVED ANTIMICROBIAL PEPTIDES 审中-公开
标题翻译：病毒衍生的抗真菌肽
公开(公告)号：WO2002079397A2
公开(公告)日：2002-10-10
申请号：PCT/US2002/004812
申请日：2002-02-19
申请人： UNIVERSITY OF PITTSBURGH OF THE COMMONWEALTH SYSTEM OF HIGHER EDUCATION
发明人： MONTELARO, Ronald, C. , MIETZNER, Timothy
IPC分类号： C12N
CPC分类号： C07K14/005 , A01N63/00 , A01N63/02 , A61K38/00 , C12N2740/16122
摘要： The invention is directed to peptides having antimicrobial activity (antimicrobial peptides). The antimicrobial peptides of the present invention are analogs of the Lentivirus Lytic Peptide 1 (LLP1) amino acid sequence. The invention is further directed to peptides referred to as the Lytic Base Unit (LBU) peptides derived from the LLP1 analogs, also having antimicrobial activity. In addition, the present invention is also directed to methods of using the peptides in a variety of contexts, including the treatment of prevention of infectious diseases. The antimicrobial LLP1 analog peptides and the LBU peptides (collectively eLLPs) may be highly active under high salt conditions and in biologic fluids. In addition, the eLLPs are effective when presented either in soluble form, or when attached to a solid surface. Furthermore, the peptides of the present invention are selectively active against a wide variety of bacterial pathogens and exhibit minimal toxicity to eukaryotic cells in vitro and in vivo .
摘要翻译：本发明涉及具有抗微生物活性的肽（抗微生物肽）。本发明的抗微生物肽是慢病毒溶血肽1（LLP1）氨基酸序列的类似物。本发明进一步涉及也称为具有抗微生物活性的衍生自LLP1类似物的Lytic Base Unit（LBU）肽的肽。此外，本发明还涉及在各种情况下使用肽的方法，包括预防感染性疾病的治疗。抗菌LLP1类似物肽和LBU肽（统称为eLLP）在高盐条件和生物液体中可能是高度活性的。此外，eLLP在以可溶形式存在或当连接到固体表面时是有效的。此外，本发明的肽对于多种细菌病原体具有选择性的活性，并且在体外和体内对真核细胞表现出极小的毒性。

5. 发明申请

WO02036758A2 RECOMBINANT LIGHT CHAINS OF BOTULINUM NEUROTOXINS AND LIGHT CHAIN FUSION PROTEINS FOR USE IN RESEARCH AND CLINICAL THERAPY 审中-公开
标题翻译：用于研究和临床治疗的嗜碱性神经毒素和光链融合蛋白的重组轻链
公开(公告)号：WO02036758A2
公开(公告)日：2002-05-10
申请号：PCT/US2001/047230
申请日：2001-11-06
申请人： UNITED STATES ARMY MEDICAL RESEARCH AND MATERIAL COMMAND
发明人： SMITH, Leonard, A. , JENSEN, Melody
IPC分类号： C12N9/00
CPC分类号： C07K14/33 , C12N9/6489
摘要： Botulinum neurotoxins, the most potent of all toxins, induce lethal neuromuscular paralysis by inhibiting exocytosis at the neuromuscular junction. The light chains (LC) of these dichain neurotoxins are a new class of zinc-endopeptidases that specifically cleave the synaptosomal proteins, SNAP-25, VAMP, or syntaxin at discrete sites. The present invention relates to the construction, expression, purification, and use of synthetic or recombinant botulinum neutoroxin genes. For example, a synthetic gene for the LC of the botulinum neurotoxin serotype A (BoNT/A) was constructed and overexpressed in Escherichia coli . The gene product was purified from inclusion bodies. The methods of the invention can provide 1.1 g of the LC per liter of culture. The LC product was stable in solution at 4 °C for at least 6 months. This rBoNT/A LC was proteolytically active, specifically cleaving the Glu-Arg bond in a 17-residue synthetic peptide of SNAP-25, the reported cleavage site of BoNT/A. Its calculated catalytic efficiency k cat /K m was higher than that reported for the native BoNT/A dichain. Treating the rBoNT/A LC with mercuric compounds completely abolished its activity, most probably by modifying the cysteine-164 residue located in the vicinity of the active site. About 70 % activity of the LC was restored by adding Zn 2+ -free, apo-LC preparation. The LC was nontoxic to mice and failed to elicit neutralizing epitope(s) when the animals were vaccinated with this protein. In addition, injecting rBoNT/A LC into sea urchin eggs inhibited exocytosis-dependent plasma membrane resealing.
摘要翻译：肉毒杆菌神经毒素是所有毒素中最有效的，通过抑制神经肌肉接头处的胞吐作用诱发致死性神经肌肉麻痹。这些双链神经毒素的轻链（LC）是一类新的锌内肽酶，其在离散位点上特异性切割突触体蛋白，SNAP-25，VAMP或syntaxin。本发明涉及合成或重组肉毒杆菌中毒素基因的构建，表达，纯化和使用。例如，构建了肉毒杆菌神经毒素血清型A（BoNT / A）的LC的合成基因，并在大肠杆菌中过表达。从包涵体纯化基因产物。本发明的方法可以提供每升培养物1.1g的LC。 LC产品在4℃的溶液中稳定至少6个月。这种rBoNT / A LC具有蛋白水解活性，特异性切割了SNAP-25的17位残基合成肽中的Glu-Arg键，这是所报道的BoNT / A切割位点。其计算的催化效率kcat / Km高于天然BoNT / A二酮所报道的催化效率。用汞化合物处理rBoNT / ALC完全消除其活性，最可能的是通过修饰位于活性位点附近的半胱氨酸164残基。通过添加无Zn 2+的载脂蛋白制备，恢复了约70％的LC活性。当对这些蛋白质接种动物时，LC对小鼠无毒，并且不能引发中和表位。另外，将rBoNT / A LC注入海胆蛋抑制胞吐作用依赖性细胞膜再密封。

6. 发明申请

WO2004038380A3 SCREENING METHODS FOR IDENTIFICATION OF EFFICIENT PRE-TRANS-SPLICING MOLECULES 审中-公开
公开(公告)号：WO2004038380A3
公开(公告)日：2004-05-06
申请号：PCT/US2003/034102
申请日：2003-10-23
申请人： INTRONN, INC. , MITCHELL, Lloyd, G. , PUTTARAJU, Madaiah , GARCIA-BLANCO, Mariano , OTTO, Edward , YANG, Yanping
发明人： MITCHELL, Lloyd, G. , PUTTARAJU, Madaiah , GARCIA-BLANCO, Mariano , OTTO, Edward , YANG, Yanping
IPC分类号： C12Q1/68
摘要： The present invention provides methods and compositions for rapid high capacity functional screening to identify optimal pre- trans -splicing molecules (PTMs). The compositions of the invention include PTM expression libraries capable of encoding candidate PTMs designed to interact with a target precursor messenger RNA molecule (target pre-mRNA) and mediate a trans-splicing reaction resulting in the generation of a novel chimeric RNA molecule (chimeric RNA). The candidate PTMs of the invention encode a portion of a first reporter molecule and may encode one or more other reporter molecules, which can be used to select for cells expressing optimal PTMs (efficient and specific). The compositions of the invention also include cells that express a target pre-mRNA encoding the remaining portion of the first reporter molecule. The screening methods of the invention encompass (i) contacting a PTM expression library with cells expressing a target pre-mRNA under conditions in which a trans -splicing reaction will occur in the presence of an optimal PTM (expressed by the library vector) resulting in the formation of a chimeric repaired RNA molecule capable of encoding at least one reporter molecule; (ii) selecting for cells expressing the repaired reporter molecule wherein expression of the reporter molecule indicates the presence of an optimal PTM in the selected cell; and (iii) identifying the optimal PTM expressed in the selected cell(s). The additional reporter molecule(s) can be used to assess both specific and non-specific trans -splicing, as well direct PTM expression.

7. 发明申请

WO2004018636A2 METHODS FOR MODULATING TRANSCRIPTIONAL ACTIVATION USING MINT PROTEINS 审中-公开
标题翻译：使用薄荷蛋白调节转录激活的方法
公开(公告)号：WO2004018636A2
公开(公告)日：2004-03-04
申请号：PCT/US2003/026436
申请日：2003-08-22
申请人： BOARC OF REGENTS, THE UNIVERSITY OF TEXAS SYSTEM , SUDHOF, Thomas, C. , BIEDERER, Thomas , HO, Angela , LIU, Xinran
发明人： SUDHOF, Thomas, C. , BIEDERER, Thomas , HO, Angela , LIU, Xinran
IPC分类号： C12N
CPC分类号： G01N33/5008 , A01K2217/05 , A01K2217/075 , C07K14/4702 , C07K14/4711 , C07K14/70567 , C07K2319/00 , C12Q1/6897 , G01N33/5023 , G01N33/5035 , G01N33/6896 , G01N2333/4709 , G01N2500/20 , G01N2800/2821 , C12Q2565/201
摘要： The present invention is directed to isolated nucleic acids encoding Mint protein variants having enhanced abilities to modulate the transcriptional activation mediated by the cytoplasmic tail of the amyloid precursor protein (APP) relative to wild-type Mint proteins. The present invention is further directed toward purified Mint protein variants having enhanced abilities to modulate the transcriptional activation mediated by the cytoplasmic tail of APP relative to wild-type Mint proteins. The present invention also encompasses methods of modulating transcriptional activation and methods of identifying compounds that modulate transcriptional activation, and vectors, as well as transfected cells and kits useful for modulating transcriptional activation or for the identification of compounds that can modulate transcriptional activation. The present invention further encompasses transgenic knockout mice with little or no expression of Mint 1, Mint 2 or Mint 3 proteins. Such reagents may be useful as candidate therapeutics for Alzheimer's disease (AD), or as models for the rational design of drugs useful for the treatment of AD.
摘要翻译：本发明涉及编码具有增强的相对于野生型薄荷蛋白调节由淀粉样蛋白前体蛋白（APP）的细胞质尾部介导的转录激活的能力的Mint蛋白变体的分离的核酸。本发明还涉及具有相对于野生型薄荷蛋白调节由APP的细胞质尾巴介导的转录激活的能力的纯化的薄荷蛋白变体。本发明还包括调节转录激活的方法和鉴定调节转录激活的化合物的方法，以及载体，以及用于调节转录激活或用于鉴定可调节转录激活的化合物的转染细胞和试剂盒。本发明还包括具有很少或没有表达Mint1，Mint2或Mint3蛋白的转基因敲除小鼠。这些试剂可用作阿尔茨海默病（AD）的候选治疗剂，或作为用于治疗AD的药物的合理设计的模型。

8. 发明申请

WO2004006678A1 SPLICEOSOME MEDIATED RNA TRANS-SPLICING FOR CORRECTION OF SKIN DISORDERS 审中-公开
标题翻译： SPLICEOSOME MEDIATED RNA TRANS-SPLICING FOR Corrients of Skine Disorders
公开(公告)号：WO2004006678A1
公开(公告)日：2004-01-22
申请号：PCT/US2003/022469
申请日：2003-07-17
申请人： INTRONN, INC. , MITCHELL, Lloyd, G. , PUTTARAJU, Madaiah , DALLINGER, Guenter , KLAUSEGGER, Alfred , BAUER, Johann
发明人： MITCHELL, Lloyd, G. , PUTTARAJU, Madaiah , DALLINGER, Guenter , KLAUSEGGER, Alfred , BAUER, Johann
IPC分类号： A01N47/40
CPC分类号： C12N15/113 , A61K48/00 , C07H21/02 , C07H21/04 , C12N2510/00
摘要： The present invention provides methods and compositions for generating novel nucleic acid molecules through targeted spliceosomal mediated RNA traps-splicing. The compositions of the invention include pre-traps-splicing molecules (PTMs) designed to interact with a target precursor messenger RNA molecule (target pre-mRNA) and mediate a traps-splicing reaction resulting in the generation of a novel chimeric RNA molecule (chimeric RNA). In particular, the PTMs of the present invention can be genetically engineered to interact with a specific target premRNA expressed in cells of the skin so as to result in correction of genetic defects responsible for a variety of different skin disorders to encode a reporter molecule or protein that may have therapeutic value. The compositions of the invention further include recombinant vectors systems capable of expressing the PTMs of the invention and cells expressing said PTMs. The methods of the invention encompass contacting the PTMs of the invention with specific target pre-Mrna expressed within cells of the skin under conditions in which a portion of the PTM is traps-spliced to a portion of the target pre-mRNA to form a chimeric RNA molecule wherein the genetic defect in the specific gene has been corrected. The present invention is based on the successful trans -splicing of the collagen XVII pre-mRNA thereby establishing the usefulness of trans -splicing for correction of skin specific genetic defects. The methods and compositions of the present invention can be used in gene therapy for treatment of specific disorders of the skin, i.e ., genodermatoses, such as epidermal fragility disorders, keratinization disorders, hair disorders and pigmentation disorders as well as cancers of the skin.
摘要翻译：本发明提供了通过靶向剪接体介导的RNA捕获 - 剪接产生新的核酸分子的方法和组合物。本发明的组合物包括设计成与靶前体信使RNA分子（靶前体mRNA）相互作用的诱捕前剪接分子（PTM），并介导产生新型嵌合RNA分子的陷阱剪接反应（嵌合 RNA）。特别地，本发明的PTM可以进行遗传工程改造以与皮肤细胞中表达的特异性靶基因前体RNA相互作用，从而导致对多种不同皮肤病症负责编码报告分子或蛋白质的遗传缺陷的校正这可能有治疗价值。本发明的组合物还包括能够表达本发明的PTM和表达所述PTM的细胞的重组载体系统。本发明的方法包括使本发明的PTM与在皮肤细胞内表达的特异性靶前Mrna接触，其中一部分PTM被剪接到目标前mRNA的一部分以形成嵌合体其中特异性基因的遗传缺陷已被校正的RNA分子。本发明基于胶原XVII前体mRNA的成功的转拼，从而确定了用于修复皮肤特异性遗传缺陷的转拼的有用性。本发明的方法和组合物可用于治疗皮肤特异性病症的基因疗法，即皮肤病，如表皮脆性障碍，角质化病症，毛发病症和色素沉着病以及皮肤癌。

9. 发明申请

WO2003104416A3 SPLICEOSOME MEDIATED RNA TRANS-SPLICING IN STEM CELLS 审中-公开
公开(公告)号：WO2003104416A3
公开(公告)日：2003-12-18
申请号：PCT/US2003/017954
申请日：2003-06-05
申请人： INTRONN, INC. , MITCHELL, Lloyd, G. , ENGLEHARDT, John , LIU, Xiao, Ming
发明人： MITCHELL, Lloyd, G. , ENGLEHARDT, John , LIU, Xiao, Ming
IPC分类号： C12P19/34
摘要： The present invention provides methods and compositions for generating novel nucleic acid molecules through targeted spliceosomal mediated trans -splicing in stem cells. The compositions of the invention include stem cells engineered to express pre- trans -splicing molecules (PTMs) designed to interact with a target precursor messenger RNA molecule (target pre-mRNA) and mediate a trans -splicing reaction resulting in the generation of novel chimeric RNA molecules (chimeric RNA). In particular, the stem cells of the present invention are genetically engineered to express a PTM that will interact with a specific target pre-mRNA expressed within a stem cell as it differentiates so as to result in correction of a genetic defect responsible for a genetic disorder. The methods of the invention encompass transferring a nucleic acid molecule capable of encoding a PTM of interest into a stem cell followed by transplantation of the PTM modified stem cell into a host. As the stem cell differentiates the target pre-mRNA is expressed thereby providing the substrate for a trans -splicing reaction. The present invention is based on the successful transfer and expression of a nucleic acid molecule encoding a PTM capable of interacting with a cystic fibrosis transmembrane conductance regulator (CFTR) pre-mRNA into primary human surface airway progenitor cells. The methods and compositions of the present invention can be used to correct genetic defects associated with a variety of different disorders such as cystic fibrosis, hemophilia, sickle cell anemia, Tay-Sachs disease, thalassemias, polycystic kidney disease and muscular dystrophy, to name a few.

10. 发明申请

WO2003104412A2 SPLICEOSOME MEDIATED RNA TRANS-SPLICING AND CORRECTION OF FACTOR VIII GENETIC DEFECTS USING SPLICEOSOME MEDIATED RNA TRANS SPLING 审中-公开
标题翻译： SPLICEOSOME介导的RNA转移分离和因子VIII的遗传缺陷的修正使用SPLICEOSOME介导的RNA转移分离
公开(公告)号：WO2003104412A2
公开(公告)日：2003-12-18
申请号：PCT/US2003/017913
申请日：2003-06-05
申请人： INTRONN, INC. , MITCHELL, Lloyd, G. , MANSFIELD, S. Gary , PUTTARAJU, Madaiah , CLARK, Rebecca , GARCIA-BLANCO, Mariano, A.
发明人： MITCHELL, Lloyd, G. , MANSFIELD, S. Gary , PUTTARAJU, Madaiah , CLARK, Rebecca , GARCIA-BLANCO, Mariano, A.
IPC分类号： C12N
CPC分类号： C07K14/755 , C12N15/10
摘要： 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 mediate a trans -splicing reaction resulting in the generation of a novel chimeric RNA molecule (chimeric RNA). In particular, the PTMs of the present invention are genetically engineered to interact with factor VIII (FVIII) target pre-mRNA so as to result in correction of clotting FVIII genetic defects responsible for hemophilia A. The compositions of the invention further include recombinant vector systems capable of expressing the PTMs of the invention and cells expressing said PTMs. The methods of the invention encompass contacting the PTMs of the invention with a FVIII target pre-mRNA under conditions in which a portion of the PTM is trans -spliced to a portion of the target pre-mRNA to form a RNA molecule wherein the genetic defect in the FVIII gene has been corrected. The methods and compositions of the present invention can be used in gene therapy for correction of FVIII disorders such as hemophilia A.
摘要翻译：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）相互作用并且介导导致产生新的嵌合RNA分子（嵌合体）的反式剪接反应的预转录分子（PTM） RNA）。特别地，本发明的PTM被遗传工程化以与因子VIII（FVIII）靶前mRNA相互作用，从而导致血友病A引起的凝血FVIII遗传缺陷的校正。本发明的组合物还包括重组载体系统能够表达本发明的PTM和表达所述PTM的细胞。本发明的方法包括将本发明的PTM与FVIII靶前体mRNA接触，其中将部分PTM转染到靶前mRNA的一部分以形成RNA分子的条件下，其中遗传缺陷在FVIII基因中已被纠正。本发明的方法和组合物可用于基因治疗中用于校正FVIII病症如血友病A.

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