Detalhe da pesquisa
1.
FoxC1 activates limbal epithelial stem cells following corneal epithelial debridement.
Exp Eye Res
; 234: 109599, 2023 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-37488009
2.
Molecular codes for cell type specification in Brn3 retinal ganglion cells.
Proc Natl Acad Sci U S A
; 114(20): E3974-E3983, 2017 05 16.
Artigo
em Inglês
| MEDLINE | ID: mdl-28465430
3.
Retinal AAV8-RS1 Gene Therapy for X-Linked Retinoschisis: Initial Findings from a Phase I/IIa Trial by Intravitreal Delivery.
Mol Ther
; 26(9): 2282-2294, 2018 09 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-30196853
4.
A long-term efficacy study of gene replacement therapy for RPGR-associated retinal degeneration.
Hum Mol Genet
; 24(14): 3956-70, 2015 Jul 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-25877300
5.
Long-term rescue of cone photoreceptor degeneration in retinitis pigmentosa 2 (RP2)-knockout mice by gene replacement therapy.
Hum Mol Genet
; 24(22): 6446-58, 2015 Nov 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-26358772
6.
In vitro modeling and rescue of ciliopathy associated with IQCB1/NPHP5 mutations using patient-derived cells.
Stem Cell Reports
; 17(10): 2172-2186, 2022 10 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-36084637
7.
Phenotype correction of hemophilia A mice by spliceosome-mediated RNA trans-splicing.
Nat Med
; 9(8): 1015-9, 2003 Aug.
Artigo
em Inglês
| MEDLINE | ID: mdl-12847523
8.
Gene Therapy of Dominant CRX-Leber Congenital Amaurosis using Patient Stem Cell-Derived Retinal Organoids.
Stem Cell Reports
; 16(2): 252-263, 2021 02 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-33513359
9.
A Common Outer Retinal Change in Retinal Degeneration by Optical Coherence Tomography Can Be Used to Assess Outcomes of Gene Therapy.
Hum Gene Ther
; 30(12): 1520-1530, 2019 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-31672061
10.
A CEP290 C-Terminal Domain Complements the Mutant CEP290 of Rd16 Mice In Trans and Rescues Retinal Degeneration.
Cell Rep
; 25(3): 611-623.e6, 2018 10 16.
Artigo
em Inglês
| MEDLINE | ID: mdl-30332642
11.
Nrl knockdown by AAV-delivered CRISPR/Cas9 prevents retinal degeneration in mice.
Nat Commun
; 8: 14716, 2017 03 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-28291770
12.
Preclinical Dose-Escalation Study of Intravitreal AAV-RS1 Gene Therapy in a Mouse Model of X-linked Retinoschisis: Dose-Dependent Expression and Improved Retinal Structure and Function.
Hum Gene Ther
; 27(5): 376-89, 2016 May.
Artigo
em Inglês
| MEDLINE | ID: mdl-27036983
13.
A visual circuit uses complementary mechanisms to support transient and sustained pupil constriction.
Elife
; 52016 09 26.
Artigo
em Inglês
| MEDLINE | ID: mdl-27669145
14.
Retinal Structure and Gene Therapy Outcome in Retinoschisin-Deficient Mice Assessed by Spectral-Domain Optical Coherence Tomography.
Invest Ophthalmol Vis Sci
; 57(9): OCT277-87, 2016 07 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-27409484
15.
Ocular and systemic safety of a recombinant AAV8 vector for X-linked retinoschisis gene therapy: GLP studies in rabbits and Rs1-KO mice.
Mol Ther Methods Clin Dev
; 5: 16011, 2016.
Artigo
em Inglês
| MEDLINE | ID: mdl-27626041
16.
Microarray analysis of gene expression in the aging human retina.
Invest Ophthalmol Vis Sci
; 43(8): 2554-60, 2002 Aug.
Artigo
em Inglês
| MEDLINE | ID: mdl-12147584
17.
Clinical studies of X-linked retinitis pigmentosa in three Swedish families with newly identified mutations in the RP2 and RPGR-ORF15 genes.
Ophthalmic Genet
; 24(4): 215-23, 2003 Dec.
Artigo
em Inglês
| MEDLINE | ID: mdl-14566651
18.
Preclinical safety evaluation of a recombinant AAV8 vector for X-linked retinoschisis after intravitreal administration in rabbits.
Hum Gene Ther Clin Dev
; 25(4): 202-11, 2014 Dec.
Artigo
em Inglês
| MEDLINE | ID: mdl-25211193
19.
Rd9 is a naturally occurring mouse model of a common form of retinitis pigmentosa caused by mutations in RPGR-ORF15.
PLoS One
; 7(5): e35865, 2012.
Artigo
em Inglês
| MEDLINE | ID: mdl-22563472
20.
Potent immune responses and in vitro pro-inflammatory cytokine suppression by a novel adenovirus vaccine vector based on rare human serotype 28.
Vaccine
; 28(35): 5691-702, 2010 Aug 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-20600496