Inhaled gene delivery: a formulation and delivery approach

Larissa Gomes dos Reis, Maree Svolos, Benedikt Hartwig, Norbert Windhab, Paul M. Young, Daniela Traini*

*Corresponding author for this work

Research output: Contribution to journalReview articlepeer-review

17 Citations (Scopus)

Abstract

Introduction: Gene therapy is a potential alternative to treat a number of diseases. Different hurdles are associated with aerosol gene delivery due to the susceptibility of plasmid DNA (pDNA) structure to be degraded during the aerosolization process. Different strategies have been investigated in order to protect and efficiently deliver pDNA to the lungs using non-viral vectors. To date, no successful therapy involving non-viral vectors has been marketed, highlighting the need for further investigation in this field. Areas covered: This review is focused on the formulation and delivery of DNA to the lungs, using non-viral vectors. Aerosol gene formulations are divided according to the current delivery systems for the lung: nebulizers, dry powder inhalers and pressurized metered dose inhalers; highlighting its benefits, challenges and potential application. Expert opinion: Successful aerosol delivery is achieved when the supercoiled DNA structure is protected during aerosolization. A formulation strategy or compounds that can protect, stabilize and efficiently transfect DNA into the cells is desired in order to produce an effective, low-cost and safe formulation. Nebulizers and dry powder inhalers are the most promising approaches to be used for aerosol delivery, due to the lower shear forces involved. In this context it is also important to highlight the importance of considering the ‘pDNA-formulation-device system’ as an integral part of the formulation development for a successful nucleic acid delivery.

Original languageEnglish
Pages (from-to)319-330
Number of pages12
JournalExpert Opinion on Drug Delivery
Volume14
Issue number3
DOIs
Publication statusPublished - 4 Mar 2017
Externally publishedYes

Keywords

  • DNA
  • dry powder inhaler (DPI)
  • nebulizer
  • Non-viral
  • pressurized metered dose inhaler (pMDI)

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