Advanced search
Publication Cover
Expert Opinion on Investigational Drugs Volume 29, 2020 - Issue 10
Submit an article Journal homepage
8,883
Views
40
CrossRef citations to date
0
Altmetric
Review

Antisense oligonucleotide therapeutics in clinical trials for the treatment of inherited retinal diseases

Kanmin Xuea Wellcome Trust Clinical Research Career Development Fellow, Nuffield Laboratory of Ophthalmology, Nuffield Department of Clinical Neurosciences, University of Oxford & Honorary Consultant Vitreoretinal Surgeon, Oxford Eye Hospital, Oxford University Hospitals NHS Foundation Trust, Oxford, UKCorrespondence[email protected]
View further author information
&
Robert E. MacLarenb Professor of Ophthalmology, Nuffield Laboratory of Ophthalmology, Nuffield Department of Clinical Neurosciences, University of Oxford & Honorary Consultant Vitreoretinal Surgeon, Oxford Eye Hospital, Oxford University Hospitals NHS Foundation Trust, Oxford, UKCorrespondence[email protected]
View further author information
Pages 1163-1170 | Received 11 May 2020, Accepted 30 Jul 2020, Published online: 01 Sep 2020

Citations (40)

Keep up to date with the latest research on this topic with citation updates for this article.

Subscribe to citation updates

Read on this site (3)

Laura Devlin, Praveen Dhondurao Sudhindar & John A. Sayer. (2023) Renal ciliopathies: promising drug targets and prospects for clinical trials. Expert Opinion on Therapeutic Targets 27:4-5, pages 325-346.
Read now
Karen Anthony. (2022) RNA-based therapeutics for neurological diseases. RNA Biology 19:1, pages 176-190.
Read now
Ruofan Connie Han, Lewis E. Fry, Ariel Kantor, Michelle E. McClements, Kanmin Xue & Robert E. MacLaren. (2021) Is subretinal AAV gene replacement still the only viable treatment option for choroideremia?. Expert Opinion on Orphan Drugs 9:1, pages 13-24.
Read now

Articles from other publishers (37)

Caroline M. Kolvenbach, Shirlee Shril & Friedhelm Hildebrandt. (2023) The genetics and pathogenesis of CAKUT. Nature Reviews Nephrology 19:11, pages 709-720.
Crossref
L.L. Lozano B.L.A. Cervantes A.. (2023) Development of experimental treatments for patients with retinitis pigmentosa. Archivos de la Sociedad Española de Oftalmología (English Edition) 98:11, pages 646-655.
Crossref
Lady L. Lozano B.Luis A. Cervantes A.. (2023) Desarrollo de tratamientos experimentales para pacientes con retinosis pigmentaria. Archivos de la Sociedad Española de Oftalmología 98:11, pages 646-655.
Crossref
Xiaotong Ding, Chunfa Chen, Qian Liu, Yao Yang, Guansong Wang & Hang Qian. (2023) Reverse Engineering of DNA and RNA Hybrid Origami Structures as Targeted Nanomedicine for KRAS-Mutated Lung Cancer Therapy. ACS Applied Polymer Materials 5:8, pages 5880-5887.
Crossref
Shaoxue Zeng, Yingying Chen, Fanfan Zhou, Ting Zhang, Xiaohui Fan, Wojciech Chrzanowski, Mark C. Gillies & Ling Zhu. (2023) Recent advances and prospects for lipid-based nanoparticles as drug carriers in the treatment of human retinal diseases. Advanced Drug Delivery Reviews 199, pages 114965.
Crossref
Maria Areblom, Sten Kjellström, Sten Andréasson, Anders Öhberg, Lotta Gränse & Ulrika Kjellström. (2023) A Description of the Yield of Genetic Reinvestigation in Patients with Inherited Retinal Dystrophies and Previous Inconclusive Genetic Testing. Genes 14:7, pages 1413.
Crossref
Ziying Yang, Minhong Huang, Xiuxiu Wei, Jun Sun & Fuping Zhang. (2023) Case report: Compound heterozygous nonsense PCDH15 variant and a novel deep‐intronic variant in a Chinese child with profound hearing loss . Molecular Genetics & Genomic Medicine 11:7.
Crossref
Renske T.W. Schellens, Sanne Broekman, Theo Peters, Pam Graave, Lucija Malinar, Hanka Venselaar, Hannie Kremer, Erik De Vrieze & Erwin Van Wijk. (2023) A protein domain-oriented approach to expand the opportunities of therapeutic exon skipping for USH2A-associated retinitis pigmentosa. Molecular Therapy - Nucleic Acids 32, pages 980-994.
Crossref
Julia-Sophia Bellingrath, Michelle E. McClements, M. Dominik Fischer & Robert E. MacLaren. (2023) Programmable RNA editing with endogenous ADAR enzymes – a feasible option for the treatment of inherited retinal disease?. Frontiers in Molecular Neuroscience 16.
Crossref
Grant A. Justin, Aniz Girach & Ramiro S. Maldonado. (2023) Antisense oligonucleotide therapy for proline-23-histidine autosomal dominant retinitis pigmentosa. Current Opinion in Ophthalmology 34:3, pages 226-231.
Crossref
Jing Wang, Wei Su, Taotao Zhang, Shasha Zhang, Huiwen Lei, Fengdie Ma, Maoning Shi, Wenjing Shi, Xiaodong Xie & Cuixia Di. (2023) Aberrant Cyclin D1 splicing in cancer: from molecular mechanism to therapeutic modulation. Cell Death & Disease 14:4.
Crossref
Adrian Szczepaniak, Agnieszka Bronisz & Jakub Godlewski. (2023) Circular RNAs—New Kids on the Block in Cancer Pathophysiology and Management. Cells 12:4, pages 552.
Crossref
Malena Daich Varela, James Bellingham, Fabiana Motta, Neringa Jurkute, Jamie M Ellingford, Mathieu Quinodoz, Kathryn Oprych, Michael Niblock, Lucas Janeschitz-Kriegl, Karolina Kaminska, Francesca Cancellieri, Hendrik P N Scholl, Eva Lenassi, Elena Schiff, Hannah Knight, Graeme Black, Carlo Rivolta, Michael E Cheetham, Michel Michaelides, Omar A Mahroo, Anthony T Moore, Andrew R Webster & Gavin Arno. (2023) Multidisciplinary team directed analysis of whole genome sequencing reveals pathogenic non-coding variants in molecularly undiagnosed inherited retinal dystrophies. Human Molecular Genetics 32:4, pages 595-607.
Crossref
Satheesh Kumar, Lewis E. Fry, Jiang-Hui Wang, Keith R. Martin, Alex W. Hewitt, Fred K. Chen & Guei-Sheung Liu. (2023) RNA-targeting strategies as a platform for ocular gene therapy. Progress in Retinal and Eye Research 92, pages 101110.
Crossref
Rebecca Kohnken, Carole Harbison, Stephanie Klein & Jeffery A. Engelhardt. 2023. Haschek and Rousseaux's Handbook of Toxicologic Pathology, Volume 2 : Safety Assessment Environmental Toxicologic Pathology. Haschek and Rousseaux's Handbook of Toxicologic Pathology, Volume 2 : Safety Assessment Environmental Toxicologic Pathology 231 268 .
Xue Wu, Naihong Yan & Ming Zhang. (2022) Retinal Degeneration: Molecular Mechanisms and Therapeutic Strategies. Current Medicinal Chemistry 29:40, pages 6125-6140.
Crossref
Halit Yusuf Altay, Fatma Ozdemir, Ferdows Afghah, Zeynep Kilinc, Mehri Ahmadian, Markus Tschopp & Cavit Agca. (2022) Gene regulatory and gene editing tools and their applications for retinal diseases and neuroprotection: From proof-of-concept to clinical trial. Frontiers in Neuroscience 16.
Crossref
Marta Zuzic, Johannes Striebel, Julia S. Pawlick, Kritika Sharma, Frank G. Holz & Volker Busskamp. (2022) Gene-independent therapeutic interventions to maintain and restore light sensitivity in degenerating photoreceptors. Progress in Retinal and Eye Research 90, pages 101065.
Crossref
Imran H. Yusuf, Andrew M. Garrett, Robert E. MacLaren & Peter Charbel Issa. (2022) Retinal cadherins and the retinal cadherinopathies: Current concepts and future directions. Progress in Retinal and Eye Research 90, pages 101038.
Crossref
Jenny Lange, Haiyan Zhou & Amy McTague. (2022) Cerebral Organoids and Antisense Oligonucleotide Therapeutics: Challenges and Opportunities. Frontiers in Molecular Neuroscience 15.
Crossref
Yongdong Su, Prithi Raguraman, Rakesh N. Veedu & Vyacheslav V. Filichev. (2022) Phosphorothioate modification improves exon-skipping of antisense oligonucleotides based on sulfonyl phosphoramidates in mdx mouse myotubes . Organic & Biomolecular Chemistry 20:18, pages 3790-3797.
Crossref
Michael Telias, Kevin K. Sit, Daniel FrozenfarBenjamin Smith, Arjit Misra, Michael J. Goard & Richard H. Kramer. (2022) Retinoic acid inhibitors mitigate vision loss in a mouse model of retinal degeneration. Science Advances 8:11.
Crossref
Beau J. Fenner, Tien-En Tan, Amutha Veluchamy Barathi, Sai Bo Bo Tun, Sia Wey Yeo, Andrew S. H. Tsai, Shu Yen Lee, Chui Ming Gemmy Cheung, Choi Mun Chan, Jodhbir S. Mehta & Kelvin Y. C. Teo. (2022) Gene-Based Therapeutics for Inherited Retinal Diseases. Frontiers in Genetics 12.
Crossref
Aniz Girach, Isabelle Audo, David G. Birch, Rachel M. Huckfeldt, Byron L. Lam, Bart P. Leroy, Michel Michaelides, Stephen R. Russell, Juliana M.F. Sallum, Katarina Stingl, Stephen H. Tsang & Paul Yang. (2022) RNA-based therapies in inherited retinal diseases. Therapeutic Advances in Ophthalmology 14, pages 251584142211346.
Crossref
Kate Ariadna Rossano Gutiérrez, Nancy Martínez Montiel, Maricruz Anaya Ruiz, Mayra Patricia Gaspariano Cholula & Rebeca Débora Martínez Contreras. (2021) Reprogramación del splicing alternativo de genes asociados a cáncer de mama. EPISTEMUS 15:30.
Crossref
Kevin J. Cao, Elijah F. Lyons, Benjamin E. Smith, Bristol L. Denlinger, Hong Ma, Jonathan D. Shirian & Richard H. Kramer. (2021) Cyclodextrin‐Assisted Delivery of Azobenzene Photoswitches for Uniform and Long‐Term Restoration of Light Responses in Degenerated Retinas of Blind Mice. Advanced Therapeutics 4:9.
Crossref
Belén García Bohórquez, Elena Aller, Ana Rodríguez Muñoz, Teresa Jaijo, Gema García García & José M. Millán. (2021) Updating the Genetic Landscape of Inherited Retinal Dystrophies. Frontiers in Cell and Developmental Biology 9.
Crossref
Francesco Testa, Andrea Sodi, Sabrina Signorini, Valentina Di Iorio, Vittoria Murro, Raffaella Brunetti-Pierri, Enza Maria Valente, Marianthi Karali, Paolo Melillo, Sandro Banfi & Francesca Simonelli. (2021) Spectrum of Disease Severity in Nonsyndromic Patients With Mutations in the CEP290 Gene: A Multicentric Longitudinal Study . Investigative Opthalmology & Visual Science 62:9, pages 1.
Crossref
Emilio Iturriaga-Goyon, Beatriz Buentello-Volante, Fátima Sofía Magaña-Guerrero & Yonathan Garfias. (2021) Future Perspectives of Therapeutic, Diagnostic and Prognostic Aptamers in Eye Pathological Angiogenesis. Cells 10:6, pages 1455.
Crossref
Aanchal Gupta, Konstantinos N. Kafetzis, Aristides D. Tagalakis & Cynthia Yu-Wai-Man. (2021) RNA therapeutics in ophthalmology - translation to clinical trials. Experimental Eye Research 205, pages 108482.
Crossref
Joel QuinnAyesha MusaAriel KantorMichelle E. McClementsJasmina Cehajic-KapetanovicRobert E. MacLarenKanmin Xue. (2021) Genome-Editing Strategies for Treating Human Retinal Degenerations. Human Gene Therapy 32:5-6, pages 247-259.
Crossref
Lewis E. Fry, Michelle E. McClements & Robert E. MacLaren. (2021) Analysis of Pathogenic Variants Correctable With CRISPR Base Editing Among Patients With Recessive Inherited Retinal Degeneration. JAMA Ophthalmology 139:3, pages 319.
Crossref
Rong Dong, Bo Zhang, Biqin Tan & Nengming Lin. (2021) Long non-coding RNAs as the regulators and targets of macrophage M2 polarization. Life Sciences 266, pages 118895.
Crossref
Maya Ross & Ron Ofri. (2021) The future of retinal gene therapy: evolving from subretinal to intravitreal vector delivery. Neural Regeneration Research 16:9, pages 1751.
Crossref
Astra Dinculescu, Brian A. Link & David A. Saperstein. (2021) Retinal Gene Therapy for Usher Syndrome: Current Developments, Challenges, and Perspectives. International Ophthalmology Clinics 61:4, pages 109-124.
Crossref
Aumer Shughoury, Thomas A. Ciulla, Benjamin Bakall, Mark E. Pennesi, Szilárd Kiss & Emmett T. Cunningham. (2021) Genes and Gene Therapy in Inherited Retinal Disease. International Ophthalmology Clinics 61:4, pages 3-45.
Crossref
Ariel Kantor, Michelle E. McClements, Caroline F. Peddle, Lewis E. Fry, Ahmed Salman, Jasmina Cehajic-Kapetanovic, Kanmin Xue & Robert E. MacLaren. 2021. Curing Genetic Diseases Through Genome Reprogramming. Curing Genetic Diseases Through Genome Reprogramming 29 79 .

Related research

People also read lists articles that other readers of this article have read.

Recommended articles lists articles that we recommend and is powered by our AI driven recommendation engine.

Cited by lists all citing articles based on Crossref citations.
Articles with the Crossref icon will open in a new tab.