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Year Number of Results
1969 1
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1984 1
1987 1
1989 1
1990 1
1992 1
1995 1
1996 1
1997 2
1998 2
1999 1
2000 1
2001 2
2003 6
2004 3
2005 1
2006 6
2007 5
2008 7
2009 3
2010 3
2011 11
2012 8
2013 17
2014 10
2015 14
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2020 13
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2023 6
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182 results

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Page 1
Current Clinical Applications of In Vivo Gene Therapy with AAVs.
Mendell JR, Al-Zaidy SA, Rodino-Klapac LR, Goodspeed K, Gray SJ, Kay CN, Boye SL, Boye SE, George LA, Salabarria S, Corti M, Byrne BJ, Tremblay JP. Mendell JR, et al. Mol Ther. 2021 Feb 3;29(2):464-488. doi: 10.1016/j.ymthe.2020.12.007. Epub 2020 Dec 10. Mol Ther. 2021. PMID: 33309881 Free PMC article. Review.
For more than 30 years, hundreds of researchers have maintained that genetic modifications would provide effective treatments for many inherited human diseases, offering durable and possibly curative clinical benefit with a single treatment. This review is limited to gene …
For more than 30 years, hundreds of researchers have maintained that genetic modifications would provide effective treatments for many inher …
Macular dystrophies: clinical and imaging features, molecular genetics and therapeutic options.
Rahman N, Georgiou M, Khan KN, Michaelides M. Rahman N, et al. Br J Ophthalmol. 2020 Apr;104(4):451-460. doi: 10.1136/bjophthalmol-2019-315086. Epub 2019 Nov 8. Br J Ophthalmol. 2020. PMID: 31704701 Free PMC article. Review.
The developments in high-resolution multimodal retinal imaging have also transformed our ability to make accurate and more timely diagnoses and more sensitive quantitative assessment of disease progression, and allowed the design of optimised clinical trial endpoint …
The developments in high-resolution multimodal retinal imaging have also transformed our ability to make accurate and more timely diagnoses …
Of men and mice: Human X-linked retinoschisis and fidelity in mouse modeling.
Vijayasarathy C, Sardar Pasha SPB, Sieving PA. Vijayasarathy C, et al. Prog Retin Eye Res. 2022 Mar;87:100999. doi: 10.1016/j.preteyeres.2021.100999. Epub 2021 Aug 11. Prog Retin Eye Res. 2022. PMID: 34390869 Review.
X-linked Retinoschisis (XLRS) is an early-onset transretinal dystrophy, often with a prominent macular component, that affects males and generally spares heterozygous females because of X-linked recessive inheritance. ...A phase I/IIa single-center, open-label, three-dose- …
X-linked Retinoschisis (XLRS) is an early-onset transretinal dystrophy, often with a prominent macular component, that affects males …
An evidence-based review of the epidemiology of myopic traction maculopathy.
Cheong KX, Xu L, Ohno-Matsui K, Sabanayagam C, Saw SM, Hoang QV. Cheong KX, et al. Surv Ophthalmol. 2022 Nov-Dec;67(6):1603-1630. doi: 10.1016/j.survophthal.2022.03.007. Epub 2022 Mar 31. Surv Ophthalmol. 2022. PMID: 35367479 Review.
Myopic traction maculopathy (MTM), one of the complications of pathologic myopia, is a spectrum of pathological conditions that are attributed to tractional changes in the eye characterized by retinoschisis, lamellar or full thickness macular hole, and foveal retinal detac …
Myopic traction maculopathy (MTM), one of the complications of pathologic myopia, is a spectrum of pathological conditions that are attribut …
The safety and efficacy of gene therapy treatment for monogenic retinal and optic nerve diseases: A systematic review.
Britten-Jones AC, Jin R, Gocuk SA, Cichello E, O'Hare F, Hickey DG, Edwards TL, Ayton LN. Britten-Jones AC, et al. Genet Med. 2022 Mar;24(3):521-534. doi: 10.1016/j.gim.2021.10.013. Epub 2021 Nov 30. Genet Med. 2022. PMID: 34906485 Free article. Review.
A comprehensive literature search was performed in Ovid MEDLINE, Ovid Embase, Cochrane Central, and clinical trial registries (February 2021). Clinical studies describing DNA-based gene therapy treatments for monogenic posterior ocular diseases were eligible …
A comprehensive literature search was performed in Ovid MEDLINE, Ovid Embase, Cochrane Central, and clinical trial registries …
Gene therapy for inherited retinal and optic nerve degenerations.
Moore NA, Morral N, Ciulla TA, Bracha P. Moore NA, et al. Expert Opin Biol Ther. 2018 Jan;18(1):37-49. doi: 10.1080/14712598.2018.1389886. Epub 2017 Oct 23. Expert Opin Biol Ther. 2018. PMID: 29057663 Free article. Review.
INTRODUCTION: The eye is a target for investigational gene therapy due to the monogenic nature of many inherited retinal and optic nerve degenerations (IRD), its accessibility, tight blood-ocular barrier, the ability to non-invasively monitor for functional and anatomic outcomes, …
INTRODUCTION: The eye is a target for investigational gene therapy due to the monogenic nature of many inherited retinal and optic nerve deg …
AAV2-Mediated Gene Therapy for Choroideremia: 5-Year Results and Alternate Anti-sense Oligonucleotide Therapy.
Zhai Y, Xu M, Radziwon A, Dimopoulos IS, Crichton P, Mah R, MacLaren RE, Somani R, Tennant MT, MacDonald IM. Zhai Y, et al. Am J Ophthalmol. 2023 Apr;248:145-156. doi: 10.1016/j.ajo.2022.12.022. Epub 2022 Dec 26. Am J Ophthalmol. 2023. PMID: 36581191
PURPOSE: To assess the long-term safety and efficacy of AAV2-REP1 in choroideremia (CHM) patients, and to test a potential antisense oligonucleotide therapy for CHM. DESIGN: Extended, prospective phase 1/2 clinical trial and laboratory investigation. METHODS: Five p …
PURPOSE: To assess the long-term safety and efficacy of AAV2-REP1 in choroideremia (CHM) patients, and to test a potential antisense oligonu …
Microperimetry in Three Inherited Retinal Disorders.
Bagdonaite-Bejarano L, Hansen RM, Fulton AB. Bagdonaite-Bejarano L, et al. Semin Ophthalmol. 2019;34(4):334-339. doi: 10.1080/08820538.2019.1622025. Epub 2019 May 30. Semin Ophthalmol. 2019. PMID: 31146612 Review.
Herein, we review MP results in three inherited retinal disorders for which gene therapy trials have been initiated-choroideremia, Stargardt disease, and X-linked juvenile retinoschisis. Each of these disorders typically presents in childhood and each has distinct effects …
Herein, we review MP results in three inherited retinal disorders for which gene therapy trials have been initiated-choroideremia, Stargardt …
Safety and Efficacy of Adeno-Associated Viral Gene Therapy in Patients With Retinal Degeneration: A Systematic Review and Meta-Analysis.
Sobh M, Lagali PS, Ghiasi M, Montroy J, Dollin M, Hurley B, Leonard BC, Dimopoulos I, Lafreniere M, Fergusson DA, Lalu MM, Tsilfidis C. Sobh M, et al. Transl Vis Sci Technol. 2023 Nov 1;12(11):24. doi: 10.1167/tvst.12.11.24. Transl Vis Sci Technol. 2023. PMID: 37982768 Free PMC article.
Of these, 80 publications met eligibility criteria, representing 28 registered clinical trials and 5 postmarket surveillance studies involving AAV gene therapy for Leber congenital amaurosis (LCA), choroideremia, Leber hereditary optic neuropathy (LHON), age-related macula …
Of these, 80 publications met eligibility criteria, representing 28 registered clinical trials and 5 postmarket surveillance studies …
Macular cysts in retinal dystrophy.
Ganesh A, Stroh E, Manayath GJ, Al-Zuhaibi S, Levin AV. Ganesh A, et al. Curr Opin Ophthalmol. 2011 Sep;22(5):332-9. doi: 10.1097/ICU.0b013e328349229e. Curr Opin Ophthalmol. 2011. PMID: 21730849 Review.
RECENT FINDINGS: Macular cysts in retinal dystrophy are seen in retinopathies caused by mutations in the NR2E3 gene, juvenile X-linked retinoschisis (XLRS), and some other retinal dystrophies. These must be distinguished from CME. ...SUMMARY: Non-CME macular cysts in retin …
RECENT FINDINGS: Macular cysts in retinal dystrophy are seen in retinopathies caused by mutations in the NR2E3 gene, juvenile X-linked re …
182 results