NDLI: SiRNS-technológia, a jövo génterápiája?

Content Provider	World Health Organization (WHO)-Global Index Medicus
Author	Rácz, Zsuzsanna Hamar, Péter
Description	Country affiliation: Hungary Author Affiliation: Rácz Z ( Semmelweis Egyetem, Altalános Orvostudományi Kar Kórélettani Intézet, Budapest, Hungary.)
Abstract	A new era in genetics started 17 years ago, when co-suppression in petunia was discovered. Later, co-suppression was identified as RNA interference (RNAi) in many plant and lower eukaryote animals. Although an ancient antiviral host defense mechanism in plants, the physiologic role of RNAi in mammals is still not completely understood. RNAi is directed by short interfering RNAs (siRNAs), one subtype of short double stranded RNAs. In this review we summarize the history and mechanisms of RNAi. We also aim to highlight the correlation between structure and efficacy of siRNAs. Delivery is the most important obstacle for siRNA based gene therapy. Viral and nonviral deliveries are discussed. In vivo delivery is the next obstacle to clinical trials with siRNAs. Although hydrodynamic treatment is effective in animals, it cannot be used in human therapy. One possibility is organ selective catheterization. The known side effects of synthesized siRNAs are also discussed. Although there are many problems to face in this new field of gene therapy, successful in vitro and in vivo experiments raise hope for treating human disease with siRNA.
File Format	HTM / HTML
ISSN	00306002
e-ISSN	17886120
Journal	Orvosi Hetilap
Issue Number	4
Volume Number	149
Language	Hungarian
Publisher	Markusovszky Lajos
Publisher Date	2008-01-27
Publisher Place	Hungary
Access Restriction	Open
Subject Keyword	Research Support, N.i.h., Extramural Gene Transfer Techniques Genetic Therapy Gene Targeting Research Support, Non-u.s. Gov't Adenoviridae Rna, Small Interfering Gene Silencing Retroviridae Dependovirus Rna Interference Animals Discipline Medicine Genetic Vectors
Alternative Title	SiRNA technology, the gene therapy of the future?
Content Type	Text
Resource Type	Article
Subject	Medicine

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miRNA cassettes in viral vectors: Problems and solutions $^{☆}$

Lipid-based vectors for siRNA delivery.

Small silencing RNAs: an expanding universe.

DNA vector-based RNA interference to study gene function in cancer.

Adenovirus and miRNAs $^{☆}$

A genome-scale shRNA resource for transgenic RNAi in Drosophila.

Multi-armed cationic cyclodextrin:poly(ethylene glycol) polyrotaxanes as efficient gene silencing vectors.

Polymers for viral gene delivery.

Combination therapy utilizing shRNA knockdown and an optimized resistant transgene for rescue of diseases caused by misfolded proteins.

SiRNS-technológia, a jövo génterápiája?

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miRNA cassettes in viral vectors: Problems and solutions $^{☆}$

Lipid-based vectors for siRNA delivery.

Small silencing RNAs: an expanding universe.

DNA vector-based RNA interference to study gene function in cancer.

Adenovirus and miRNAs $^{☆}$

A genome-scale shRNA resource for transgenic RNAi in Drosophila.

Multi-armed cationic cyclodextrin:poly(ethylene glycol) polyrotaxanes as efficient gene silencing vectors.

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SiRNS-technológia, a jövo génterápiája?