SiRNS-technológia, a jövo génterápiája?

Translated title of the contribution: SiRNA technology, the gene therapy of the future?

Zsuzsanna Rácz, P. Hamar

Research output: Contribution to journalArticle

7 Citations (Scopus)

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.

Original languageHungarian
Pages (from-to)153-159
Number of pages7
JournalOrvosi Hetilap
Volume149
Issue number4
DOIs
Publication statusPublished - Jan 27 2008

Fingerprint

RNA Interference
Genetic Therapy
Small Interfering RNA
Technology
Petunia
Double-Stranded RNA
Hydrodynamics
Eukaryota
Catheterization
Antiviral Agents
Mammals
History
Clinical Trials
Therapeutics

ASJC Scopus subject areas

  • Medicine(all)

Cite this

SiRNS-technológia, a jövo génterápiája? / Rácz, Zsuzsanna; Hamar, P.

In: Orvosi Hetilap, Vol. 149, No. 4, 27.01.2008, p. 153-159.

Research output: Contribution to journalArticle

Rácz, Zsuzsanna ; Hamar, P. / SiRNS-technológia, a jövo génterápiája?. In: Orvosi Hetilap. 2008 ; Vol. 149, No. 4. pp. 153-159.
@article{b4d554a4b5d343afa35c07760f538541,
title = "SiRNS-technol{\'o}gia, a j{\"o}vo g{\'e}nter{\'a}pi{\'a}ja?",
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.",
keywords = "Delivery, Human gene therapy, RNA interference (RNAi), Short interfering RNA (siRNA)",
author = "Zsuzsanna R{\'a}cz and P. Hamar",
year = "2008",
month = "1",
day = "27",
doi = "10.1556/OH.2008.28289",
language = "Hungarian",
volume = "149",
pages = "153--159",
journal = "Orvosi Hetilap",
issn = "0030-6002",
publisher = "Akademiai Kiado",
number = "4",

}

TY - JOUR

T1 - SiRNS-technológia, a jövo génterápiája?

AU - Rácz, Zsuzsanna

AU - Hamar, P.

PY - 2008/1/27

Y1 - 2008/1/27

N2 - 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.

AB - 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.

KW - Delivery

KW - Human gene therapy

KW - RNA interference (RNAi)

KW - Short interfering RNA (siRNA)

UR - http://www.scopus.com/inward/record.url?scp=39349086980&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=39349086980&partnerID=8YFLogxK

U2 - 10.1556/OH.2008.28289

DO - 10.1556/OH.2008.28289

M3 - Article

VL - 149

SP - 153

EP - 159

JO - Orvosi Hetilap

JF - Orvosi Hetilap

SN - 0030-6002

IS - 4

ER -