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Agresti et al. J Transl Genet Genom 2018;2:9 I http://dx.doi.org/10.20517/jtgg.2018.05 Page 9 of 11

when developing a gene therapy vector. Future directions surrounding research for these diseases should
focus on developing a therapeutic vector that is capable of answering the aforementioned questions relating
to: overcoming cellular and mitochondrial barriers, establish long-term (if not permanent) correction, and
exhibiting a low (to negative) immunogenicity profile in patients.

DECLARATIONS
Acknowledgments
The authors take special acknowledgment to Ueli Gubler, Ph.D. for his scientific guidance and expertise
regarding the subject matter.

Authors’ contributions
Concept and design: Agresti CA, Tolias P
Data acquisition and analysis: Agresti CA, Halkiadakis PN
Manuscript preparation: Agresti CA, Halkiadakis PN, Tolias P
Critical revision and finalizing of the manuscript: Agresti CA, Tolias P

Availability of data and materials
Not applicable.

Financial support and sponsorship
None.

Conflicts of interest
All authors declared that there are no conflicts of interest.

Ethical approval and consent to participate
Not applicable.

Consent for publication
Not applicable.

Copyright
© The Author(s) 2018.

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