Literature DB >> 17982877

The therapeutic potential of siRNA in gene therapy of neurodegenerative disorders.

E Koutsilieri1, A Rethwilm, C Scheller.   

Abstract

RNA interference using small inhibitory RNA (siRNA) has become a powerful tool to downregulate mRNA levels by cellular nucleases that become activated when a sequence homology between the siRNA and a respective mRNA molecule is detected. Therefore siRNA can be used to silence genes involved in the pathogenesis of various diseases associated with a known genetic background. As for many neurodegenerative disorders a causative therapy is unavailable, siRNA holds a promising option for the development of novel therapeutic strategies. Here we discuss different siRNA target strategies aiming for an allele-specific degradation of disease-inducing mRNA and we review the literature in the field of siRNA and its application in animal models of neurodegenerative diseases, including Alzheimer's disease (AD), amyotrophic lateral sclerosis (ALS), Huntington's disease (HD) and spinocerebellar ataxia (SCA1).

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Year:  2007        PMID: 17982877     DOI: 10.1007/978-3-211-73574-9_7

Source DB:  PubMed          Journal:  J Neural Transm Suppl        ISSN: 0303-6995


  15 in total

Review 1.  Novel siRNA delivery strategy: a new "strand" in CNS translational medicine?

Authors:  Lisa Gherardini; Giuseppe Bardi; Mariangela Gennaro; Tommaso Pizzorusso
Journal:  Cell Mol Life Sci       Date:  2013-03-19       Impact factor: 9.261

2.  Systemic peptide mediated delivery of an siRNA targeting α-syn in the CNS ameliorates the neurodegenerative process in a transgenic model of Lewy body disease.

Authors:  Brian Spencer; Ivy Trinh; Edward Rockenstein; Michael Mante; Jazmin Florio; Anthony Adame; Omar M A El-Agnaf; Changyoun Kim; Eliezer Masliah; Robert A Rissman
Journal:  Neurobiol Dis       Date:  2019-03-05       Impact factor: 5.996

Review 3.  Role of Astrocytes in Manganese Neurotoxicity Revisited.

Authors:  Tao Ke; Marta Sidoryk-Wegrzynowicz; Edward Pajarillo; Asha Rizor; Félix Alexandre Antunes Soares; Eunsook Lee; Michael Aschner
Journal:  Neurochem Res       Date:  2019-09-30       Impact factor: 3.996

Review 4.  Spinocerebellar ataxia type 2: clinical presentation, molecular mechanisms, and therapeutic perspectives.

Authors:  J J Magaña; L Velázquez-Pérez; B Cisneros
Journal:  Mol Neurobiol       Date:  2012-09-21       Impact factor: 5.590

Review 5.  Exosome and exosomal microRNA: trafficking, sorting, and function.

Authors:  Jian Zhang; Sha Li; Lu Li; Meng Li; Chongye Guo; Jun Yao; Shuangli Mi
Journal:  Genomics Proteomics Bioinformatics       Date:  2015-02-24       Impact factor: 7.691

Review 6.  Nanocarriers as a powerful vehicle to overcome blood-brain barrier in treating neurodegenerative diseases: Focus on recent advances.

Authors:  Xiaoqian Niu; Jiejian Chen; Jianqing Gao
Journal:  Asian J Pharm Sci       Date:  2018-10-22       Impact factor: 6.598

7.  Folate-equipped nanolipoplexes mediated efficient gene transfer into human epithelial cells.

Authors:  Emmanuel Mornet; Nathalie Carmoy; Céline Lainé; Loïc Lemiègre; Tony Le Gall; Isabelle Laurent; Remi Marianowski; Claude Férec; Pierre Lehn; Thierry Benvegnu; Tristan Montier
Journal:  Int J Mol Sci       Date:  2013-01-14       Impact factor: 5.923

8.  Visualizing the attack of RNase enzymes on dendriplexes and naked RNA using atomic force microscopy.

Authors:  Hosam G Abdelhady; Yen-Ling Lin; Haiping Sun; Mohamed E H ElSayed
Journal:  PLoS One       Date:  2013-04-18       Impact factor: 3.240

9.  Lentiviral-mediated RNA interference targeting stathmin1 gene in human gastric cancer cells inhibits proliferation in vitro and tumor growth in vivo.

Authors:  Javed Akhtar; Zhou Wang; Zhi Ping Zhang; Ming Ming Bi
Journal:  J Transl Med       Date:  2013-09-16       Impact factor: 5.531

10.  Convection enhanced delivery to the Brain: preparing for gene therapy and protein delivery to the Brain for functional and restorative Neurosurgery by understanding low-flow neurocatheter infusions using the Alaris(®) system infusion pump.

Authors:  Karl Sillay; Angelica Hinchman; Erinc Akture; Shahriar Salamat; Gurwattan Miranpuri; Justin Williams; Dawn Berndt
Journal:  Ann Neurosci       Date:  2013-04
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