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Quoted phrase not found in phrase index: "Leber congenital amaurosis 16"
Page 1
Classification and Growth Rate of Chorioretinal Atrophy after Voretigene Neparvovec-Rzyl for RPE65-Mediated Retinal Degeneration.
Bommakanti N, Young BK, Sisk RA, Berrocal AM, Duncan JL, Bakall B, Mathias MT, Ahmed I, Chorfi S, Comander J, Nagiel A, Besirli CG. Bommakanti N, et al. Ophthalmol Retina. 2024 Jan;8(1):42-48. doi: 10.1016/j.oret.2023.08.017. Epub 2023 Sep 3. Ophthalmol Retina. 2024. PMID: 37660736
All atrophy was manually segmented. Linear mixed-effects models with random slopes and intercepts were fit using atrophy area and square root of atrophy area. ...The slopes of the mixed-effects models of atrophy area and square root of atrophy area (estimate standard error …
All atrophy was manually segmented. Linear mixed-effects models with random slopes and intercepts were fit using atrophy area and squ …
Barriers for retinal gene therapy: separating fact from fiction.
Kumar-Singh R. Kumar-Singh R. Vision Res. 2008 Jul;48(16):1671-1680. doi: 10.1016/j.visres.2008.05.005. Epub 2008 Jun 18. Vision Res. 2008. PMID: 18565565 Free PMC article. Review.
Furthermore, AAV is known to generate limiting immune responses in humans despite the absence of similar immune responses in preclinical canine and murine studies. Three clinical trials to treat Leber's congenital amaurosis using AAV are under way. A …
Furthermore, AAV is known to generate limiting immune responses in humans despite the absence of similar immune responses in preclinical can …
Gene Therapy Using a miniCEP290 Fragment Delays Photoreceptor Degeneration in a Mouse Model of Leber Congenital Amaurosis.
Zhang W, Li L, Su Q, Gao G, Khanna H. Zhang W, et al. Hum Gene Ther. 2018 Jan;29(1):42-50. doi: 10.1089/hum.2017.049. Epub 2017 Jul 5. Hum Gene Ther. 2018. PMID: 28679290 Free PMC article.
Mutations in the cilia-centrosomal protein CEP290 are frequently observed in autosomal recessive childhood blindness disorder Leber congenital amaurosis (LCA). No treatment or cure currently exists for this disorder. The Cep290(rd16) (retinal degeneration …
Mutations in the cilia-centrosomal protein CEP290 are frequently observed in autosomal recessive childhood blindness disorder Leber …
Rational Engineering and Preclinical Evaluation of Neddylation and SUMOylation Site Modified Adeno-Associated Virus Vectors in Murine Models of Hemophilia B and Leber Congenital Amaurosis.
Maurya S, Mary B, Jayandharan GR. Maurya S, et al. Hum Gene Ther. 2019 Dec;30(12):1461-1476. doi: 10.1089/hum.2019.164. Epub 2019 Nov 26. Hum Gene Ther. 2019. PMID: 31642343 Free PMC article.
Synthetic engineering of viral vectors such as adeno-associated virus (AAV) is crucial to overcome host transduction barriers observed during clinical gene therapy. We reasoned that exploring the role of cellular ubiquitin-like modifiers (UBLs) such as Neddylation or SUMOy …
Synthetic engineering of viral vectors such as adeno-associated virus (AAV) is crucial to overcome host transduction barriers observed durin …
Delivering Transgenic DNA Exceeding the Carrying Capacity of AAV Vectors.
Hirsch ML, Wolf SJ, Samulski RJ. Hirsch ML, et al. Methods Mol Biol. 2016;1382:21-39. doi: 10.1007/978-1-4939-3271-9_2. Methods Mol Biol. 2016. PMID: 26611576 Free PMC article.
Gene delivery using recombinant adeno-associated virus (rAAV) has emerged to the forefront demonstrating safe and effective phenotypic correction of diverse diseases including hemophilia B and Leber's congenital amaurosis. In addition to rAAV's high efficienc …
Gene delivery using recombinant adeno-associated virus (rAAV) has emerged to the forefront demonstrating safe and effective phenotypic corre …
Gene therapy in the second eye of RPE65-deficient dogs improves retinal function.
Annear MJ, Bartoe JT, Barker SE, Smith AJ, Curran PG, Bainbridge JW, Ali RR, Petersen-Jones SM. Annear MJ, et al. Gene Ther. 2011 Jan;18(1):53-61. doi: 10.1038/gt.2010.111. Epub 2010 Aug 12. Gene Ther. 2011. PMID: 20703309 Free PMC article.
Electroretinography (ERG) and vision testing showed rescue in 16 of 18 treated eyes, with no significant difference between first and second treated eyes. ...
Electroretinography (ERG) and vision testing showed rescue in 16 of 18 treated eyes, with no significant difference between first and …
Persistence of non-viral vector mediated RPE65 expression: case for viability as a gene transfer therapy for RPE-based diseases.
Koirala A, Conley SM, Makkia R, Liu Z, Cooper MJ, Sparrow JR, Naash MI. Koirala A, et al. J Control Release. 2013 Dec 28;172(3):745-52. doi: 10.1016/j.jconrel.2013.08.299. Epub 2013 Sep 11. J Control Release. 2013. PMID: 24035979 Free PMC article.
Mutations in the retinal pigment epithelium (RPE) gene RPE65 are associated with multiple blinding diseases including Leber's Congenital Amaurosis (LCA). Our goal has been to develop persistent, effective non-viral genetic therapies to treat this condition. . …
Mutations in the retinal pigment epithelium (RPE) gene RPE65 are associated with multiple blinding diseases including Leber's Cong …