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Table 1 Comparison of clinically relevant RNA delivery platforms

From: Advances in the delivery of RNA therapeutics: from concept to clinical reality

Delivery vehicle

Type of RNA in clinical trials

Advantages

Disadvantages

References

Naked RNA

siRNA, ASO, mRNA

No additional materials or synthesis required

Prone to degradation

Immunogenic

Difficulty entering cell

Poor circulation half-life

[63,64,74,75,76,77,65, 73–78, 101, 103, 114, 115]

Nanoparticle

siRNA, ASO, mRNA

Increased half life

Protection from nucleases

Aids in endocytosis and endosomal escape

Elevated risk of toxicity with introducing excipient materials

[12,13,14,15,16,17,18,19,20,21,22,23,24,25,26,27,28,29,30,31,32,33,34,35,36,59,83,84,107,111,112,157,158,37, 58–60, 82–85, 106–108, 110–113, 131, 145, 156–159]

Conjugate

siRNA, ASO

Defined chemical structure

Ability to target specific receptors

Limited toxicity due to lack of excipient materials

High doses required

Dependent on chemical modifications for RNA stability

[38,39,40,41,42,43, 62]

  1. ASO antisense oligonucleotide, siRNA short interfering RNA