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Taking Optogenetics into the Human Brain: Opportunities and Challenges in Clinical Trial Design

Authors White M, Mackay M, Whittaker RG

Received 24 April 2020

Accepted for publication 7 July 2020

Published 25 August 2020 Volume 2020:12 Pages 33—41

DOI https://doi.org/10.2147/OAJCT.S259702

Checked for plagiarism Yes

Review by Single anonymous peer review

Peer reviewer comments 2

Editor who approved publication: Professor Arthur Frankel


Michael White,1 Michael Mackay,1 Roger G Whittaker1,2

1Translational and Clinical Research Institute, Newcastle University, Newcastle Upon Tyne NE2 4HH, UK; 2Department of Clinical Neurophysiology, Royal Victoria Hospital, Newcastle Upon Tyne NE1 4LP, UK

Correspondence: Michael White
Translational and Clinical Research Institute, Newcastle University, Framlington Place, Newcastle Upon Tyne NE2 4HH, UK
Tel +44 (0) 191 208 3281
Email Michael.white2@ncl.ac.uk

Abstract: Optogenetics, the use of light to control the activity of suitably sensitized cells, has led to major advances in the field of basic neuroscience since it first emerged in 2005. Already, the technique has entered clinical trials for conditions such as Retinitis Pigmentosa. A major focus of interest is the use of optogenetics within the brain, where the ability to precisely control the activity of specific subsets of neurons could lead to novel treatments for a wide range of disorders from epilepsy to schizophrenia. However, since any therapy would require both the use of gene therapy techniques to introduce non-human proteins, and implantable electronic devices to provide optical stimulation, applying this technique in the brain presents a unique set of obstacles and challenges. This review looks at the reasons why researchers are exploring the use of optogenetics within the brain. It then explores the challenges facing scientists, engineers and clinicians wanting to take this technology from the lab into the first human brain, discussing different possibilities for a first-in-human clinical trial from a sponsor, patient and regulatory perspective.

Keywords: gene therapy, implantable device, opsins, first-in-human, neuroscience

Creative Commons License This work is published by Dove Medical Press Limited, and licensed under a Creative Commons Attribution License. The full terms of the License are available at http://creativecommons.org/licenses/by/4.0/. The license permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.

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