Nucleic Acids as Therapeutic Agents - icaam

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The major limitation of the practicalutilization of nucleic acids <strong>as</strong> therapeuticagents is delivering these compounds toinside their target cells
Fig. 11.1 Inhibition of translation ofspecific mRNAs by antisense (AS)nucleic acid molecules.Promoter and polyadenylationregions are p and pa, respectively;the intron is indicated by the letter A;and the exons are indicated bynumbers (1 and 2).(A) An antisense cDNA is clonedinto an expression vector and theconstruct is transfected into a cell,where the antisense RNA issynthesized. Antisense RNAhybridizes to target mRNA, andtranslation is blocked.(B) An antisense oligonucleotide isintroduced into a cell, and after ithybridizes with the target mRNA,translation is blocked.AB
Page 1: Nucleic Acids asTherapeutic Agents
Page 5 and 6: Fig. 11.7 Schematic representation
Page 7 and 8: Thomas Cech and Sidney Altman share
Page 9 and 10: Vascular endothelialgrowth factor
Page 11 and 12: Interfering RNAs• Addition of ds
Page 13 and 14: Fire and Mello shared the 2006 Nobe
Page 15 and 16: Nucleic Acid Delivery
Page 17 and 18: Gene Doping: non-therapeutic use of
Page 19 and 20: Fig. 11.21 Use of a nonpathogenic s
Page 21 and 22: Fig. 11.23 A single chain Fab fragm

Nucleic Acids as Therapeutic Agents - icaam

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