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Therapies for Inherited Retinal Dystrophies: What is Enough?

作者信息

Leroy Bart P, Daly Avril, Héon Elise, Sahel José-Alain, Dollfus Hélène

机构信息

Department of Ophthalmology, Ghent University Hospital & Department of Head & Skin, Ghent University, ERN-EYE, Ghent, Belgium; Division of Ophthalmology, Children's Hospital of Philadelphia, Philadelphia, PA, USA.

Retina International, Dublin, Ireland.

出版信息

Drug Discov Today. 2024 Sep;29(9):104095. doi: 10.1016/j.drudis.2024.104095. Epub 2024 Jul 9.

DOI:10.1016/j.drudis.2024.104095

PMID:38992419

Abstract

摘要

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Therapies for Inherited Retinal Dystrophies: What is Enough?

Drug Discov Today. 2024 Sep;29(9):104095. doi: 10.1016/j.drudis.2024.104095. Epub 2024 Jul 9.

[Gene therapy for retinal dystrophies].

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Splicing-correcting therapeutic approaches for retinal dystrophies: where endogenous gene regulation and specificity matter.

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Addressing Challenges in Developing Treatments for Inherited Retinal Diseases: Recommendations From the Third Monaciano Symposium.

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Doc Ophthalmol. 2024 Dec;149(3):139-142. doi: 10.1007/s10633-024-09997-4. Epub 2024 Nov 25.

Self-reported visual function and in-depth swept-source optical coherence tomography features of cystoid macular edema in retinitis pigmentosa.

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相似文献

Therapies for Inherited Retinal Dystrophies: What is Enough?

Drug Discov Today. 2024 Sep;29(9):104095. doi: 10.1016/j.drudis.2024.104095. Epub 2024 Jul 9.

[Gene therapy for retinal dystrophies].

Ophthalmologe. 2012 Feb;109(2):121-8. doi: 10.1007/s00347-011-2453-3.

Emerging Gene Therapy Treatments for Inherited Retinal Diseases.

Ophthalmic Surg Lasers Imaging Retina. 2018 Jul 1;49(7):472-478. doi: 10.3928/23258160-20180628-02.

Retinal dystrophies and gene therapy.

Eur J Pediatr. 2012 May;171(5):757-65. doi: 10.1007/s00431-011-1615-2. Epub 2011 Nov 15.

Voretigene neparvovec-rzyl (Luxturna) for inherited retinal dystrophy.

Med Lett Drugs Ther. 2018 Mar 26;60(1543):53-55.

[Gene Replacement Therapy for Inherited Retinal Dystrophies].

Klin Monbl Augenheilkd. 2017 Mar;234(3):320-328. doi: 10.1055/s-0043-101817. Epub 2017 Mar 29.

[Specific gene therapy for hereditary retinal dystrophies - an update].

Klin Monbl Augenheilkd. 2014 Mar;231(3):210-5. doi: 10.1055/s-0033-1351059. Epub 2013 Dec 10.

Bilateral Subretinal Voretigene Neparvovec-rzyl (Luxturna) Gene Therapy.

Ophthalmol Retina. 2019 May;3(5):450. doi: 10.1016/j.oret.2019.02.007.

Clinical Trials in Retinal Dystrophies.

Middle East Afr J Ophthalmol. 2016 Jan-Mar;23(1):49-59. doi: 10.4103/0974-9233.173135.

Splicing-correcting therapeutic approaches for retinal dystrophies: where endogenous gene regulation and specificity matter.

Invest Ophthalmol Vis Sci. 2014 May 27;55(5):3285-94. doi: 10.1167/iovs.14-14544.

引用本文的文献

Addressing Challenges in Developing Treatments for Inherited Retinal Diseases: Recommendations From the Third Monaciano Symposium.

Transl Vis Sci Technol. 2025 Aug 1;14(8):37. doi: 10.1167/tvst.14.8.37.

Valuing patients' high unmet need to drive innovative therapies for inherited retinal degenerations: insights from the RIWC 2024 workshop on value.

Front Med (Lausanne). 2025 Mar 4;12:1481670. doi: 10.3389/fmed.2025.1481670. eCollection 2025.

Documenta Ophthalmologica editorial: welcoming a broadened scope.

Doc Ophthalmol. 2024 Dec;149(3):139-142. doi: 10.1007/s10633-024-09997-4. Epub 2024 Nov 25.

Self-reported visual function and in-depth swept-source optical coherence tomography features of cystoid macular edema in retinitis pigmentosa.

Int J Retina Vitreous. 2024 Nov 22;10(1):89. doi: 10.1186/s40942-024-00608-7.

Therapies for Inherited Retinal Dystrophies: What is Enough?

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Therapies for Inherited Retinal Dystrophies: What is Enough?

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