Particle-mediated delivery of frataxin plasmid to a human sensory neuronal model of Friedreich's ataxia-Reference-Cited by-同舟云学术

Particle-mediated delivery of frataxin plasmid to a human sensory neuronal model of Friedreich's ataxia

Published:2020 Issue:9 Volume:8 Page:2398-2403
ISSN:2047-4830
Container-title:Biomaterials Science
language:en
Short-container-title:Biomater. Sci.

Author:

Czuba-Wojnilowicz Ewa¹²³⁴⁵^ORCID,Viventi Serena⁶³⁴⁵^ORCID,Howden Sara E.⁷⁸³⁴⁵^ORCID,Maksour Simon⁹¹⁰¹¹¹²¹³,Hulme Amy E.⁹¹⁰¹¹¹²¹³^ORCID,Cortez-Jugo Christina¹²³⁴⁵^ORCID,Dottori Mirella⁶³⁴⁵⁹^ORCID,Caruso Frank¹²³⁴⁵^ORCID

Affiliation:

1. ARC Centre of Excellence in Convergent Bio-Nano Science and Technology

2. and the Department of Chemical Engineering

3. The University of Melbourne

4. Parkville

5. Australia

6. Department of Biomedical Engineering

7. Murdoch Children's Research Institute

8. and the Department of Paediatrics

9. Illawarra Health and Medical Research Institute

10. Molecular Horizons

11. School of Medicine

12. University of Wollongong

13. Wollongong

Abstract

Multilayered particles in gene therapy for Friedreich's ataxia induce a 27 000-fold increase in frataxin gene expression in a patient-derived cell model.

Funder

Australian Research Council

National Health and Medical Research Council

University of Melbourne

National Ataxia Foundation

Friedreich's Ataxia Research Alliance

University of Wollongong

Publisher

Royal Society of Chemistry (RSC)

Subject

General Materials Science,Biomedical Engineering

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