Mikroskop-integrierte Optische Kohärenztomographie: Intraoperativer Nutzen bei der Durchführung der subretinalen Gentherapie

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Abstract

In vielen laufenden Studien zu Gentherapien in der Augenheilkunde werden Adeno-assoziierte Viren oder Lentiviren als Vektoren benutzt. Allerdings können diese Vektoren die Retina aufgrund der relativ impermeablen epiretinalen Membran schlecht durchdringen, weswegen das Gen-Therapeutikum vorzugsweise subretinal injiziert wird. Ziel der vorliegenden Studie war es, die subretinale Applikation des AAV-REP1-Gentherapeutikums, bestehend aus einem genetischen Vektor, der auf das Rab-Escort-Protein-1 (REP-1) codierende Gen abzielt (AAV-REP1), mithilfe der Mikroskop-intregrierten Optischen Kohärenztomographie (MI-OCT) durchzuführen.

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Gene Therapy and Stem Cell Transplantation in Retinal Disease: The New Frontier.

Robert MacLaren, Jean Bennett, Steven D Schwartz (2016)

Gene and cell therapies have the potential to prevent, halt, or reverse diseases of the retina in patients with currently incurable blinding conditions. Over the past 2 decades, major advances in our understanding of the pathobiologic basis of retinal diseases, coupled with growth of gene transfer and cell transplantation biotechnologies, have created optimism that previously blinding retinal conditions may be treatable. It is now possible to deliver cloned genes safely and stably to specific retinal cell types in humans. Preliminary results testing gene augmentation strategies in human recessive diseases suggest promising safety and efficacy profiles, including improved visual function outcomes over extended periods. Additional gene-based strategies under development include approaches to autosomal dominant disease ("gain of function"), attempts to deliver genes encoding therapeutic proteins with proven mechanisms of action interfering with specific disease pathways, and approaches that could be used to render retinal cells other than atrophied photoreceptors light sensitive. In the programs that are the furthest along-pivotal regulatory safety and efficacy trials studying individuals with retinal degeneration resulting from RPE65 mutations-initial results reveal a robust safety profile and clinically significant improvements in visual function, thereby making this program a frontrunner for the first approved gene therapy product in the United States. Similar to gene therapy, progress in regenerative or stem cell-based transplantation strategies has been substantial. It is now possible to deliver safely stem cell-derived, terminally differentiated, biologically and genetically defined retinal pigment epithelium (RPE) to the diseased human eye. Although demonstration of clinical efficacy is still well behind the gene therapy field, multiple programs investigating regenerative strategies in RPE disease are beginning to enroll subjects, and initial results suggest possible signs of efficacy. Stem cells capable of becoming other retinal cell types, such as photoreceptors, are on the cusp of clinical trials. Stem cell-derived transplants can be delivered to precise target locations in the eye, and their ability to ameliorate, reverse, regenerate, or neuroprotect against disease processes can be assessed. Results from these studies will provide foundational knowledge that may lead to clinically significant therapies for currently untreatable retinal disease.

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Intraoperative Use of Microscope-Integrated Optical Coherence Tomography for Subretinal Gene Therapy Delivery

Ninel Gregori, Byron L Lam, Janet L Davis (2019)

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Mini-Descemet Membrane Endothelial Keratoplasty for the Early Treatment of Acute Corneal Hydrops in Keratoconus.

Björn Bachmann, Alexander Händel, Sebastian Siebelmann … (2019)

To describe a new surgical option for the treatment of acute corneal hydrops in keratoconus and to present the first results.

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Author and article information

Journal

Journal ID (publisher-id): KOP

Journal ID (doi): 10.1159/issn.2297-0118

Title: Karger Kompass Ophthalmologie

Publisher: S. Karger AG

ISSN (Print): 2297-0118

ISSN (Electronic): 2297-0045

Publication date Subscription-year: 2019

Publication date (Electronic): 11 November 2019

Volume: 5

Issue: 4

Pages: 168-169

Affiliations

Zentrum für Augenheilkunde, Uniklinik Köln, Köln, Deutschland

Author notes

*PD Dr. Sebastian Siebelmann, FEBO, Zentrum für Augenheilkunde, Uniklinik Köln, Kerpener Straße 62, 50937 Köln, Deutschland, sebastian.siebelmann@uk-koeln.de

Article

Publisher ID: 502992 Other ID: Karger Kompass Ophthalmol 2019;5:168-169

DOI: 10.1159/000502992

SO-VID: ac4239fd-e780-4b63-8b9d-643448ed2133

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Copyright: All rights reserved. No part of this publication may be translated into other languages, reproduced or utilized in any form or by any means, electronic or mechanical, including photocopying, recording, microcopying, or by any information storage and retrieval system, without permission in writing from the publisher. Drug Dosage: The authors and the publisher have exerted every effort to ensure that drug selection and dosage set forth in this text are in accord with current recommendations and practice at the time of publication. However, in view of ongoing research, changes in government regulations, and the constant flow of information relating to drug therapy and drug reactions, the reader is urged to check the package insert for each drug for any changes in indications and dosage and for added warnings and precautions. This is particularly important when the recommended agent is a new and/or infrequently employed drug. Disclaimer: The statements, opinions and data contained in this publication are solely those of the individual authors and contributors and not of the publishers and the editor(s). The appearance of advertisements or/and product references in the publication is not a warranty, endorsement, or approval of the products or services advertised or of their effectiveness, quality or safety. The publisher and the editor(s) disclaim responsibility for any injury to persons or property resulting from any ideas, methods, instructions or products referred to in the content or advertisements.

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Mikroskop-integrierte Optische Kohärenztomographie: Intraoperativer Nutzen bei der Durchführung der subretinalen Gentherapie

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Abstract

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Karger: Ophthalmology

Most cited references 3

Gene Therapy and Stem Cell Transplantation in Retinal Disease: The New Frontier.

Intraoperative Use of Microscope-Integrated Optical Coherence Tomography for Subretinal Gene Therapy Delivery

Mini-Descemet Membrane Endothelial Keratoplasty for the Early Treatment of Acute Corneal Hydrops in Keratoconus.

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