摘要:
Nucleic acid sequences containing unmethylated CpG dinucleotides that modulate an immune response including stimulating a Th1 pattern of immune activation, cytokine production, NK lytic activity, and B cell proliferation are disclosed. The sequences are also useful as synthetic adjuvant.
摘要:
The present invention is directed to RNA interference (RNAi) molecules targeted against a Huntington's disease nucleic acid sequence, and methods of using these RNAi molecules to treat Huntington's disease.
摘要:
The present disclosure provides targeting peptides and vectors containing a sequence that encodes targeting peptides that deliver agents to the brain.
摘要:
The present disclosure provides methods of treating a disease or delivering a therapeutic agent to a mammal comprising administering to the mammal's cisterna magna and/or ventricle an rAAV particle containing a vector comprising a nucleic acid encoding a therapeutic protein inserted between a pair of AAV inverted terminal repeats in a manner such that cells with access to the cerebrospinal fluid (CSF) express the therapeutic agent and in certain embodiments secretes the therapeutic agent into the CSF for distribution to the brain.
摘要:
Certain embodiments of the present invention provide a method for treating or preventing juvenile neuronal ceroid lipofuscinosis (JNCL) in an animal comprising administering CBX, GRA, or GZA to the animal.
摘要:
The present invention provides, among other things, methods of treating a muscular dystrophy including administering to a subject suffering from or susceptible to a muscular dystrophy an angiotensin (1-7) peptide. The present invention is, in part, based on the unexpected discovery that administration of an angiotensin (1-7) peptide in a muscular dystrophy animal model reduces fibrosis, restores locomotor activity and restores sympathovagal balance, which are characteristic symptoms in patients suffering from muscular dystrophy. Thus, the present invention provides a new and more effective therapy for muscular dystrophy. In some embodiments, an angiotensin (1-7) peptide includes the naturally occurring angiotensin (1-7) amino acid sequence of Asp1-Arg2-Val3-Tyr4-lle5 -His6-Pro7 (SEQID NO:1). In some embodiments, the angiotensin (1-7) peptide is a functional equivalent of SEQ ID NO:1. In some embodiments, the linear peptide has an amino acid sequence of Asp1-Arg2-Val3-Ser4-lle5-H'is6-Cys7 (SEQ ID NO:2). In some embodiments, the cyclic peptide is a 4,7-cyclized angiotensin (1-7).
摘要翻译:本发明提供了治疗肌营养不良症的方法,包括给患有肌营养不良症或易患肌营养不良症的受试者服用血管紧张素(1-7)肽。 本发明部分基于意外的发现,即在肌营养不良动物模型中施用血管紧张素(1-7)肽减少纤维变性,恢复运动活性并恢复交感神经平衡,这是患有肌肉的患者的特征性症状 营养不良。 因此,本发明为肌营养不良提供了新的和更有效的疗法。 在一些实施方案中,血管紧张素(1-7)肽包括天然存在的Asp1-Arg2-Val3-Tyr4-Ile5-His6-Pro7(SEQ ID NO:1)的血管紧张素(1-7)氨基酸序列。 在一些实施方案中,血管紧张素(1-7)肽是SEQ ID NO:1的功能等同物。 在一些实施方案中,线性肽具有Asp1-Arg2-Val3-Ser4-Ile5-H'is6-Cys7(SEQ ID NO:2)的氨基酸序列。 在一些实施方案中,环肽是4,7-环化的血管紧张素(1-7)。
摘要:
The invention relates to Factor H gene polymorphisms and haplotypes associated with an elevated or a reduced risk of AMD. The invention provides methods and reagents for diagnosis and treatment of AMD.
摘要:
The invention provides isolated nucleic acid encoding one or more gene products that allow for degradation of caffeine and related structures, and for preparation of intermediates in that catabolic pathway. Further provided are methods of using one or more of those gene products.
摘要:
In one aspect, the invention relates to methods for treating muscle atrophy by providing to an animal in need thereof an effective amount of a compound. The compound can modulate the expression levels of multiple mRNA of a muscle atrophy signature. This abstract is intended as a scanning tool for purposes of searching in the particular art and is not intended to be limiting of the present invention. The disclosed compounds can treat muscle atrophy when administered in an effective amount to an animal, such as a mammal, fish or bird. For example, human. Also disclosed in a method of lowering blood glucose in an animal by administering ursolic acid or ursolic acid analogs, such as betulininc acid analogs, and narigenin analogs, such as naringenin, in an effective amount to an animal. Also disclosed in a method of lowering blood glucose in an animal by administering ungerine/hippeastrine analogs, such as hippeastrine, in an effective amount to an animal. The disclosed compounds can also promote muscle health, promote normal muscle function, and/or promote healthy aging muscles by providing to a subject in need thereof an effective amount of a disclosed compound.
摘要:
The present invention is directed to RNA interference (RNAi) molecules targeted against a Huntington's disease nucleic acid sequence, and methods of using these RNAi molecules to treat Huntington's disease.