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Nucleotide-based therapeutic agents

Antisense oligonucleotides (AS-ODNs) present significant therapeutic potential but face key challenges. The major drawbacks include biological instability, which hinders their efficacy in clinical applications, and difficulties in cellular targeting, uptake, and endosomal efflux. Furthermore, the development of RNA interference mechanisms, such as siRNA and miRNA, offers alternative strategies for inhibiting gene expression and protein function. This overview highlights the limitations of AS-ODNs and explores innovative approaches, including small molecule inhibitors, to selectively target tumor-relevant defects.

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Nucleotide-based therapeutic agents

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  1. Nucleotide-based therapeutic agents

  2. MAJOR DRAWBACKS OF ANTISENSE OLIGONUCLEOTIDES Biologic instability

  3. MAJOR DRAWBACKS OF ANTISENSE OLIGONUCLEOTIDES Biologicinstability Cellulartargeting Uptake Effluxfromendosomes

  4. MAJOR DRAWBACKS OF ANTISENSE OLIGONUCLEOTIDES Biologicinstability Cellulartargeting Uptake Effluxfromendosomes Appropriate controls

  5. RNA INTERFERENCE: siRNA & miRNA

  6. CALAA-01 (Calando Pharmaceuticals) siRNA inhibiting tumor growth via RNA interference to reduce expression of the M2 subunit of ribonucleotide reductase (R2).

  7. HOW CAN WE SELECTIVELY TARGET TUMOR RELEVANT DEFECTS? INIBIT GENE EXPRESSION INIBIT PROTEIN FUNCTION Small molecule inhibitors AS-ODN mAbs siRNA

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