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Year Number of Results
1997 1
1999 2
2005 1
2006 2
2009 1
2010 2
2011 1
2013 1
2014 2
2015 1
2016 1
2018 1
2020 3
2021 2
2022 1
2023 3
2024 2

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23 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.
We review gene therapy for neuromuscular disorders (spinal muscular atrophy [SMA]; Duchenne muscular dystrophy [DMD]; X-linked myotubular myopathy [XLMTM]; and diseases of the central nervous system, including Alzheimer's disease, Parkinson's disease, Canavan disease, aromatic l- …
We review gene therapy for neuromuscular disorders (spinal muscular atrophy [SMA]; Duchenne muscular dystrophy [DMD]; X-linked myotubular my …
A Global Assessment of Eye Health and Quality of Life: A Systematic Review of Systematic Reviews.
Assi L, Chamseddine F, Ibrahim P, Sabbagh H, Rosman L, Congdon N, Evans J, Ramke J, Kuper H, Burton MJ, Ehrlich JR, Swenor BK. Assi L, et al. JAMA Ophthalmol. 2021 May 1;139(5):526-541. doi: 10.1001/jamaophthalmol.2021.0146. JAMA Ophthalmol. 2021. PMID: 33576772 Free PMC article. Review.
EVIDENCE REVIEW: The electronic databases MEDLINE, Ovid, Embase, Cochrane Database of Systematic Reviews, Proquest Dissertations, and Theses Global were searched from inception through June 29, 2020, using a comprehensive search strategy. Systematic reviews addressing visi …
EVIDENCE REVIEW: The electronic databases MEDLINE, Ovid, Embase, Cochrane Database of Systematic Reviews, Proquest Dissertations, and Theses …
Opsins for vision restoration.
Simon CJ, Sahel JA, Duebel J, Herlitze S, Dalkara D. Simon CJ, et al. Biochem Biophys Res Commun. 2020 Jun 25;527(2):325-330. doi: 10.1016/j.bbrc.2019.12.117. Epub 2020 Jan 23. Biochem Biophys Res Commun. 2020. PMID: 31982136 Review.
The former category has been extensively employed for vision restoration in the past decade with two ongoing clinical trials employing microbial opsins to restore light sensation in retinitis pigmentosa patients. The latter subtype of animal opsins is emerging more …
The former category has been extensively employed for vision restoration in the past decade with two ongoing clinical trials employing micro …
Stem cell therapy for inherited retinal diseases: a systematic review and meta-analysis.
Chen X, Xu N, Li J, Zhao M, Huang L. Chen X, et al. Stem Cell Res Ther. 2023 Oct 5;14(1):286. doi: 10.1186/s13287-023-03526-x. Stem Cell Res Ther. 2023. PMID: 37798796 Free PMC article. Review.
This study aims to quantitatively examine the effectiveness and safety of stem cell therapy for patients with IRDs, including retinitis pigmentosa and Stargardt disease (STGD). METHODS: We searched PubMed, EMBASE, Web of Science, Cochrane Library databases, and the …
This study aims to quantitatively examine the effectiveness and safety of stem cell therapy for patients with IRDs, including retinitis …
Systemic diseases associated with retinal dystrophies.
Werdich XQ, Place EM, Pierce EA. Werdich XQ, et al. Semin Ophthalmol. 2014 Sep-Nov;29(5-6):319-28. doi: 10.3109/08820538.2014.959202. Semin Ophthalmol. 2014. PMID: 25325857 Review.
Ocular manifestations may be the presenting symptom of a syndromic disease and can include retinitis pigmentosa, cone-rod dystrophy, or maculopathy. Alternatively, patients affected with syndromic disease may already have other systemic manifestations at the time re …
Ocular manifestations may be the presenting symptom of a syndromic disease and can include retinitis pigmentosa, cone-rod dyst …
Clinical and Therapeutic Aspects of Sideroblastic Anaemia with B-Cell Immunodeficiency, Periodic Fever and Developmental Delay (SIFD) Syndrome: a Systematic Review.
Maccora I, Ramanan AV, Wiseman D, Marrani E, Mastrolia MV, Simonini G. Maccora I, et al. J Clin Immunol. 2023 Jan;43(1):1-30. doi: 10.1007/s10875-022-01343-0. Epub 2022 Aug 19. J Clin Immunol. 2023. PMID: 35984545 Free PMC article. Review.
METHODS: A systematic review according to PRISMA approach, including all articles published before the 30(th) of July 2021 in Pubmed and EMBASE database, was performed. RESULTS: The search identified 29 publications describing 58 unique patients. To date, 41 unique mutatio …
METHODS: A systematic review according to PRISMA approach, including all articles published before the 30(th) of July 2021 in Pubmed and EMB …
The dynamic nature of Bruch's membrane.
Booij JC, Baas DC, Beisekeeva J, Gorgels TG, Bergen AA. Booij JC, et al. Prog Retin Eye Res. 2010 Jan;29(1):1-18. doi: 10.1016/j.preteyeres.2009.08.003. Epub 2009 Sep 10. Prog Retin Eye Res. 2010. PMID: 19747980 Review.
These age-related changes may not only influence the normal age-related health of photoreceptor cells, but also the onset and progression of diseases like retinitis pigmentosa (RP) and age-related macular degeneration (AMD). ...
These age-related changes may not only influence the normal age-related health of photoreceptor cells, but also the onset and progression of …
The molecular basis of human retinal and vitreoretinal diseases.
Berger W, Kloeckener-Gruissem B, Neidhardt J. Berger W, et al. Prog Retin Eye Res. 2010 Sep;29(5):335-75. doi: 10.1016/j.preteyeres.2010.03.004. Epub 2010 Mar 31. Prog Retin Eye Res. 2010. PMID: 20362068 Review.
Knowledge of the many different eye diseases affecting retinal structure and function (night and colour blindness, retinitis pigmentosa, cone and cone rod dystrophies, photoreceptor dysfunctions, as well as vitreoretinal traits) is critical for future therapeutic de …
Knowledge of the many different eye diseases affecting retinal structure and function (night and colour blindness, retinitis pigme …
23 results