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Resistance of morpholino phosphorodiamidate oligomers to enzymatic degradation

R M Hudziak1, E Barofsky, D F Barofsky

  • 1ANTIVIRALS, Inc., Corvallis, OR 97333, USA.

Insights

Morpholino phosphorodiamidate oligomers show excellent resistance to enzymatic degradation and biologic fluids. This stability suggests their potential for in vivo applications in therapeutic development.

Area of Science:

  • Biochemistry
  • Oligonucleotide Chemistry

Background:

  • Oligonucleotides are crucial in therapeutics but susceptible to enzymatic degradation.
  • Morpholino phosphorodiamidate backbones offer an alternative to traditional nucleic acid structures.

Purpose of the Study:

  • To evaluate the enzymatic and biologic fluid resistance of Morpholino phosphorodiamidate oligomers.
  • To assess the stability of these oligomers for potential in vivo applications.

Main Methods:

  • A 25-mer Morpholino phosphorodiamidate oligomer was synthesized.
  • Incubation with various hydrolases (nucleases, proteases, esterases) and biologic fluids (serum, plasma).
  • Direct analysis of reaction mixtures using MALDI-TOF mass spectrometry.

Main Results:

  • The 25-mer demonstrated complete resistance to 13 different hydrolases.
  • No degradation was observed when incubated with serum and plasma.
  • MALDI-TOF MS confirmed the integrity of the oligomer post-incubation.

Conclusions:

  • Morpholino phosphorodiamidate backbones exhibit exceptional stability against enzymatic hydrolysis.
  • This inherent resistance makes them highly suitable candidates for in vivo therapeutic applications.
  • Further development of Morpholino-based therapeutics is warranted based on their stability profile.

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