Current Opinion in Molecular Therapeutics最新文献_第7页

Expanding the catalytic repertoire of ribozymes and deoxyribozymes beyond RNA substrates. 扩大核酶和脱氧核酶的催化范围，使其超越RNA底物。

Current Opinion in Molecular Therapeutics Pub Date : 2010-04-01

Stefan Franzen

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Transcriptional gene silencing through epigenetic changes mediated by non-coding RNAs. 非编码rna介导的表观遗传变化中的转录基因沉默。

Current Opinion in Molecular Therapeutics Pub Date : 2010-04-01

Barbora Malecová, Kevin V Morris

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siRNA-based topical microbicides targeting sexually transmitted infections. 靶向性传播感染的sirna局部杀微生物剂。

Current Opinion in Molecular Therapeutics Pub Date : 2010-04-01

Joseph A Katakowski, Deborah Palliser

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Alternative antibody formats. 备选抗体格式。

Current Opinion in Molecular Therapeutics Pub Date : 2010-04-01

Roland E Kontermann

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Telomerase inhibitors for the treatment of brain tumors and the potential of intranasal delivery. 端粒酶抑制剂治疗脑肿瘤及鼻内给药的潜力。

Current Opinion in Molecular Therapeutics Pub Date : 2010-04-01

Rintaro Hashizume, Nalin Gupta

{"title":"Telomerase inhibitors for the treatment of brain tumors and the potential of intranasal delivery.","authors":"Rintaro Hashizume, Nalin Gupta","doi":"","DOIUrl":"","url":null,"abstract":"A fundamental limitation in the treatment of brain tumors is that < 1% of most therapeutic agents administered systemically are able to cross the blood-brain barrier (BBB). The development of new strategies that circumvent the BBB should increase the likelihood of tumor response to selected therapeutic agents. Intranasal delivery (IND) is a practical, noninvasive method of bypassing the BBB to deliver therapeutic agents to the brain. This technique has demonstrated promising results in the treatment of neurological disorders. Telomerase is a reverse transcriptase that is expressed in the vast majority of malignant gliomas, although not in the healthy brain. Telomerase inhibition can therefore be used as a therapeutic strategy for selectively targeting malignant gliomas. The first successful IND of a telomerase inhibitor as a therapy for brain tumors was GRN-163, an oligonucleotide N3'-->5' thiophosphoramidate telomerase inhibitor, which was successfully administered into intracerebral tumors in rats with no apparent toxicity. GRN-163 exhibited favorable tumor uptake and inhibited tumor growth, leading to prolonged lifespan in treated animals. The IND of telomerase inhibitors represents a new therapeutic approach that appears to selectively kill tumor cells, without inducing toxic effects in the surrounding healthy brain tissue.","PeriodicalId":50605,"journal":{"name":"Current Opinion in Molecular Therapeutics","volume":"12 2","pages":"168-75"},"PeriodicalIF":0.0,"publicationDate":"2010-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"28906804","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

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Sequence-selective targeting of duplex DNA by peptide nucleic acids. 肽核酸序列选择性靶向双链DNA。

Current Opinion in Molecular Therapeutics Pub Date : 2010-04-01

Peter E Nielsen

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CERE-110, an adeno-associated virus-based gene delivery vector expressing human nerve growth factor for the treatment of Alzheimer's disease. CERE-110，一种基于腺相关病毒的基因传递载体，表达用于治疗阿尔茨海默病的人神经生长因子。

Current Opinion in Molecular Therapeutics Pub Date : 2010-04-01

Ronald J Mandel

{"title":"CERE-110, an adeno-associated virus-based gene delivery vector expressing human nerve growth factor for the treatment of Alzheimer's disease.","authors":"Ronald J Mandel","doi":"","DOIUrl":"","url":null,"abstract":"To date, only five drugs have been approved for the treatment of Alzheimer's disease (AD); however, these agents impact the symptoms rather than the progression of the disease. It is well established that nerve growth factor (NGF) enhances the function and survival of basal forebrain cholinergic neurons that are vulnerable in AD. However, NGF does not cross the blood-brain barrier, and intraventricular NGF injections in animals and humans were associated with significant side effects. Adeno-associated virus (AAV)-based gene delivery is a novel technology being developed for administration of NGF to the brain to treat AD symptoms and progression. Indeed, the efficacy of ex vivo gene delivery was demonstrated in patients with AD who experienced improvements in cerebral metabolism and cognition compared with pre-operative function without adverse events. CERE-110 (AAV2-NGF), under development by Ceregene Inc, is an AAV serotype 2-based vector expressing human NGF delivered to the nucleus basalis of Meynert by stereotactic injection for the treatment of AD. Animal studies have established the preclinical efficacy of CERE-110, revealing an excellent safety profile. CERE-110 has passed phase I clinical testing and a multicenter phase II clinical trial has commenced. CERE-110 is a promising candidate for the treatment of AD.","PeriodicalId":50605,"journal":{"name":"Current Opinion in Molecular Therapeutics","volume":"12 2","pages":"240-7"},"PeriodicalIF":0.0,"publicationDate":"2010-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"28907178","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

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Glembatumumab vedotin, a conjugate of an anti-glycoprotein non-metastatic melanoma protein B mAb and monomethyl auristatin E for the treatment of melanoma and breast cancer. Glembatumumab vedotin是一种抗糖蛋白非转移性黑色素瘤蛋白B mAb和单甲基auristatin E的结合物，用于治疗黑色素瘤和乳腺癌。

Current Opinion in Molecular Therapeutics Pub Date : 2010-04-01

Louie Naumovski, Jagath R Junutula

{"title":"Glembatumumab vedotin, a conjugate of an anti-glycoprotein non-metastatic melanoma protein B mAb and monomethyl auristatin E for the treatment of melanoma and breast cancer.","authors":"Louie Naumovski, Jagath R Junutula","doi":"","DOIUrl":"","url":null,"abstract":"Glembatumumab vedotin (CR-011-vc-MMAE) is a mAb-drug conjugate being developed by Celldex Therapeutics Inc for the treatment of glycoprotein non-metastatic melanoma protein B (GPNMB)-expressing cancers. Glembatumumab is a fully human mAb directed against an extracellular domain of GPNMB expressed in human breast cancers and melanomas. Glembatumumab is conjugated to the potent microtubule inhibitor monomethyl auristatin E using a cathepsin cleavable valine-citrulline (vc) dipeptide linker. Glembatumumab vedotin has demonstrated potent antitumor activity in preclinical studies, including in GPNMB-expressing cell lines. In human melanoma xenograft mice, intravenous glembatumumab vedotin was associated with complete tumor regression without significant toxicity. In two phase I/II clinical trials, intravenous glembatumumab vedotin demonstrated antitumor activity in patients with breast cancer or melanoma. Skin rash was the most common toxicity reported, which may have been caused by the expression of GPNMB in healthy skin. Glembatumumab vedotin had a relatively short t(1/2) , prompting the evaluation of more frequent dosing schedules. Prospective, randomized clinical trials will likely be required to determine the therapeutic potential of glembatumumab vedotin in the treatment of breast cancer and melanoma.","PeriodicalId":50605,"journal":{"name":"Current Opinion in Molecular Therapeutics","volume":"12 2","pages":"248-57"},"PeriodicalIF":0.0,"publicationDate":"2010-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"28907179","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

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Chemical modification of siRNA. siRNA的化学修饰。

Current Opinion in Molecular Therapeutics Pub Date : 2010-04-01

Elena L Chernolovskaya, Marina A Zenkova

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MEDI-528, an anti-IL-9 humanized antibody for the treatment of asthma. MEDI-528，一种抗il -9人源化抗体，用于治疗哮喘。

Current Opinion in Molecular Therapeutics Pub Date : 2010-04-01

Sabina A Antoniu

{"title":"MEDI-528, an anti-IL-9 humanized antibody for the treatment of asthma.","authors":"Sabina A Antoniu","doi":"","DOIUrl":"","url":null,"abstract":"In development by MedImmune LLC, under license from Genaera Corp, MEDI-528 is an injectable, humanized mAb against IL-9 for the potential treatment of asthma. In asthma, airway inflammation is usually adequately minimized with standard-of-care treatments, such as inhaled corticosteroids and leukotriene modifiers, but it is sometimes less responsive to such therapies and other anti-inflammatory approaches are required. Several T-helper cell type 2-derived cytokines, such as IL-4, -5, -9 and -13, play a major role in the development of disease pathogenic features in asthma, including airway eosinophilia, increased IgE production, mucus hypersecretion and airway hyperreactivity. As an IL-9 antagonist, MEDI-528 appears to inhibit a range of asthma pathogenic features in antigen-exposed mice. To date, clinical data are modest, although insufficient to judge the efficacy of the drug in humans, and larger and longer-term clinical trials are required. MEDI-528, along with other anticytokine therapies targeting different ILs, remains under investigation in early-phase trials for asthma. Time will tell if this form of therapy can be used as an add-on to less efficacious anti-inflammatory therapy or can replace the existing anti-inflammatory therapies in the treatment of asthma.","PeriodicalId":50605,"journal":{"name":"Current Opinion in Molecular Therapeutics","volume":"12 2","pages":"233-9"},"PeriodicalIF":0.0,"publicationDate":"2010-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"28907176","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

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