4.3 Article

Nanodelivery of nucleic acids

期刊

NATURE REVIEWS METHODS PRIMERS
卷 2, 期 1, 页码 -

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SPRINGERNATURE
DOI: 10.1038/s43586-022-00104-y

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资金

  1. European Research Council (ERC) [ERC-StG-2019-848325]
  2. FundacAo para a Ciencia e a Tecnologia FCT Grant [PTDC/BTM-MAT/4738/2020]
  3. US National Institute of Health (NIH) [R01CA200900, R01HL156362, R01HL159012]
  4. US DoD PRCRP Idea Award [W81XWH1910482]
  5. American Lung Association
  6. National Key R&D Program of China [2020YFA0710700]
  7. National Natural Science Foundation of China [21991132, 52003264, 52021002, 52033010]
  8. Fundamental Research Funds for the Central Universities [WK2060000027]
  9. Innovation Discovery Grants award from the Mass General Brigham
  10. U.S. Department of Defense (DOD) [W81XWH1910482] Funding Source: U.S. Department of Defense (DOD)

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There is a growing need for a safe and effective method to deliver gene therapy materials, and nanomaterials offer an unprecedented opportunity to overcome these challenges. By utilizing their diverse physico-chemical properties, nanomaterials can be functionalized to selectively target specific biomolecules. The combination of gene therapies and nanoscale delivery systems has expanded the therapeutic and biomedical applications of nucleic acid therapeutics.
There is growing need for a safe, efficient, specific and non-pathogenic means for delivery of gene therapy materials. Nanomaterials for nucleic acid delivery offer an unprecedented opportunity to overcome these drawbacks; owing to their tunability with diverse physico-chemical properties, they can readily be functionalized with any type of biomolecules/moieties for selective targeting. Nucleic acid therapeutics such as antisense DNA, mRNA, small interfering RNA (siRNA) or microRNA (miRNA) have been widely explored to modulate DNA or RNA expression Strikingly, gene therapies combined with nanoscale delivery systems have broadened the therapeutic and biomedical applications of these molecules, such as bioanalysis, gene silencing, protein replacement and vaccines. Here, we overview how to design smart nucleic acid delivery methods, which provide functionality and efficacy in the layout of molecular diagnostics and therapeutic systems. It is crucial to outline some of the general design considerations of nucleic acid delivery nanoparticles, their extraordinary properties and the structure-function relationships of these nanomaterials with biological systems and diseased cells and tissues.

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