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Small interfering RNAs: heralding a new era in gene therapy


Bujak, Maro; Ratkaj, Ivana; Baus Lončar, Mirela; Spaventi, Radan; Kraljević Pavelić, Sandra
Small interfering RNAs: heralding a new era in gene therapy // Gene Therapy - Developments and Future Perspectives / Chunsheng Kang (ur.).
Rijeka: InTech, 2011. str. 71-92


Naslov
Small interfering RNAs: heralding a new era in gene therapy

Autori
Bujak, Maro ; Ratkaj, Ivana ; Baus Lončar, Mirela ; Spaventi, Radan ; Kraljević Pavelić, Sandra

Vrsta, podvrsta i kategorija rada
Poglavlja u knjigama, stručni

Knjiga
Gene Therapy - Developments and Future Perspectives

Urednik/ci
Chunsheng Kang

Izdavač
InTech

Grad
Rijeka

Godina
2011

Raspon stranica
71-92

ISBN
978-953-307-617-1

Ključne riječi
SiRNA, gene therapy, delivery

Sažetak
Last decades have witnessed a tremendous expansion in knowledge and availability of the genome sequence, which was of great importance for advancements in the field of gene therapy. This led to improved strategies based on use of nucleic acids with sequences complementary to specific target genes in treatment of many diseases. Especially, advancements have been achieved in discovery and use of diverse RNA molecules other than messenger RNAs (mRNAs), transfer RNAs (tRNAs), or ribosomal RNAs (rRNAs). Such RNA molecules, known as non-coding RNAs (ncRNAs), serve diverse biological roles some of which are still elusive. Some biological processes known to be regulated by ncRNAs include transcriptional regulation of genes, gene silencing, messenger RNA stability and translation, development, proliferation, haematopoiesis, apoptosis, protein translocation and chromosome replication. Up to now, the use of ncRNAs as a research tool has greatly improved gene therapy approaches for various diseases (Gallaso 2010), but also substantially improved drug discovery and target validation. In this book chapter, a focus on the use of a particular approach, namely RNAi for improved gene silencing for both, therapeutic approaches and identification of new therapeutic targets is discussed.

Izvorni jezik
Engleski

Znanstvena područja
Temeljne medicinske znanosti



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