Abstract
RNA interference is a biological process that controls gene silencing in all living cells. Targeting the RNA interference system represents a novel therapeutic strategy able to intercede with multiple disease-related genes and to target many neurodegencrative diseases. Recently, the design of small interfering RNA-selective compounds has become more straightforward because of the significant progress made in predictive modeling for new therapeutic approaches. Although in vivo delivery of RNA interference remains a significant obstacle, new data show that RNAi blocks gene function in vivo, suggesting a potential therapeutic approach for humans. Some groups have demonstrated the efficacy of RNAi therapy in Alzheimer's disease. Results, based on animal models, show a down-regulation of the amyloid precursor protein and a consequent reduction of the amyloid-β peptide accumulation in the brain or the inactivation of P-secretase (BACEI). Indeed, lentiviral vectors expressing siRNAs targeting BACE1 reduce amyloid production and the neurodegencrative and behavioural deficit in APP transgenic mice. This review highlights recent advances in RNA research and focuses on strengths and weaknesses of RNAi compounds in Alzheimer's disease.
| Original language | English |
|---|---|
| Pages (from-to) | 1166-1176 |
| Number of pages | 11 |
| Journal | Mini-Reviews in Medicinal Chemistry |
| Volume | 7 |
| Issue number | 11 |
| DOIs | |
| Publication status | Published - Nov 2007 |
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ASJC Scopus subject areas
- Chemistry(all)
- Medicine (miscellaneous)
- Pharmacology
Cite this
RNA interference as a tool for Alzheimer's disease therapy. / Orlacchio, Antonio; Bernardi, Giorgio; Orlacchio, Aldo; Martino, Sabata.
In: Mini-Reviews in Medicinal Chemistry, Vol. 7, No. 11, 11.2007, p. 1166-1176.Research output: Contribution to journal › Article
}
TY - JOUR
T1 - RNA interference as a tool for Alzheimer's disease therapy
AU - Orlacchio, Antonio
AU - Bernardi, Giorgio
AU - Orlacchio, Aldo
AU - Martino, Sabata
PY - 2007/11
Y1 - 2007/11
N2 - RNA interference is a biological process that controls gene silencing in all living cells. Targeting the RNA interference system represents a novel therapeutic strategy able to intercede with multiple disease-related genes and to target many neurodegencrative diseases. Recently, the design of small interfering RNA-selective compounds has become more straightforward because of the significant progress made in predictive modeling for new therapeutic approaches. Although in vivo delivery of RNA interference remains a significant obstacle, new data show that RNAi blocks gene function in vivo, suggesting a potential therapeutic approach for humans. Some groups have demonstrated the efficacy of RNAi therapy in Alzheimer's disease. Results, based on animal models, show a down-regulation of the amyloid precursor protein and a consequent reduction of the amyloid-β peptide accumulation in the brain or the inactivation of P-secretase (BACEI). Indeed, lentiviral vectors expressing siRNAs targeting BACE1 reduce amyloid production and the neurodegencrative and behavioural deficit in APP transgenic mice. This review highlights recent advances in RNA research and focuses on strengths and weaknesses of RNAi compounds in Alzheimer's disease.
AB - RNA interference is a biological process that controls gene silencing in all living cells. Targeting the RNA interference system represents a novel therapeutic strategy able to intercede with multiple disease-related genes and to target many neurodegencrative diseases. Recently, the design of small interfering RNA-selective compounds has become more straightforward because of the significant progress made in predictive modeling for new therapeutic approaches. Although in vivo delivery of RNA interference remains a significant obstacle, new data show that RNAi blocks gene function in vivo, suggesting a potential therapeutic approach for humans. Some groups have demonstrated the efficacy of RNAi therapy in Alzheimer's disease. Results, based on animal models, show a down-regulation of the amyloid precursor protein and a consequent reduction of the amyloid-β peptide accumulation in the brain or the inactivation of P-secretase (BACEI). Indeed, lentiviral vectors expressing siRNAs targeting BACE1 reduce amyloid production and the neurodegencrative and behavioural deficit in APP transgenic mice. This review highlights recent advances in RNA research and focuses on strengths and weaknesses of RNAi compounds in Alzheimer's disease.
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U2 - 10.2174/138955707782331678
DO - 10.2174/138955707782331678
M3 - Article
C2 - 18045220
AN - SCOPUS:36749049934
VL - 7
SP - 1166
EP - 1176
JO - Mini-Reviews in Medicinal Chemistry
JF - Mini-Reviews in Medicinal Chemistry
SN - 1389-5575
IS - 11
ER -