334 related articles for article (PubMed ID: 30358434)
1. Somatic Gene Editing of
McCullough KT; Boye SL; Fajardo D; Calabro K; Peterson JJ; Strang CE; Chakraborty D; Gloskowski S; Haskett S; Samuelsson S; Jiang H; Witherspoon CD; Gamlin PD; Maeder ML; Boye SE
Hum Gene Ther; 2019 May; 30(5):571-589. PubMed ID: 30358434
[TBL] [Abstract][Full Text] [Related]
2. AAV-mediated gene therapy in the guanylate cyclase (RetGC1/RetGC2) double knockout mouse model of Leber congenital amaurosis.
Boye SL; Peshenko IV; Huang WC; Min SH; McDoom I; Kay CN; Liu X; Dyka FM; Foster TC; Umino Y; Karan S; Jacobson SG; Baehr W; Dizhoor A; Hauswirth WW; Boye SE
Hum Gene Ther; 2013 Feb; 24(2):189-202. PubMed ID: 23210611
[TBL] [Abstract][Full Text] [Related]
3. Functional study of two biochemically unusual mutations in
Boye SL; Olshevskaya EV; Peshenko IV; McCullough KT; Boye SE; Dizhoor AM
Mol Vis; 2016; 22():1342-1351. PubMed ID: 27881908
[TBL] [Abstract][Full Text] [Related]
4. Development of an AAV-CRISPR-Cas9-based treatment for dominant cone-rod dystrophy 6.
Mellen RW; Calabro KR; McCullough KT; Crosson SM; Cova A; Fajardo D; Xu E; Boye SL; Boye SE
Mol Ther Methods Clin Dev; 2023 Sep; 30():48-64. PubMed ID: 37361352
[TBL] [Abstract][Full Text] [Related]
5. Gene Therapy Fully Restores Vision to the All-Cone Nrl(-/-) Gucy2e(-/-) Mouse Model of Leber Congenital Amaurosis-1.
Boye SL; Peterson JJ; Choudhury S; Min SH; Ruan Q; McCullough KT; Zhang Z; Olshevskaya EV; Peshenko IV; Hauswirth WW; Ding XQ; Dizhoor AM; Boye SE
Hum Gene Ther; 2015 Sep; 26(9):575-92. PubMed ID: 26247368
[TBL] [Abstract][Full Text] [Related]
6. Use of AAV Vectors for CRISPR-Mediated In Vivo Genome Editing in the Retina.
Yu W; Wu Z
Methods Mol Biol; 2019; 1950():123-139. PubMed ID: 30783971
[TBL] [Abstract][Full Text] [Related]
7.
Peshenko IV; Olshevskaya EV; Dizhoor AM
J Biol Chem; 2020 Dec; 295(52):18301-18315. PubMed ID: 33109612
[TBL] [Abstract][Full Text] [Related]
8. GUCY2D Cone-Rod Dystrophy-6 Is a "Phototransduction Disease" Triggered by Abnormal Calcium Feedback on Retinal Membrane Guanylyl Cyclase 1.
Sato S; Peshenko IV; Olshevskaya EV; Kefalov VJ; Dizhoor AM
J Neurosci; 2018 Mar; 38(12):2990-3000. PubMed ID: 29440533
[TBL] [Abstract][Full Text] [Related]
9. Functional and behavioral restoration of vision by gene therapy in the guanylate cyclase-1 (GC1) knockout mouse.
Boye SE; Boye SL; Pang J; Ryals R; Everhart D; Umino Y; Neeley AW; Besharse J; Barlow R; Hauswirth WW
PLoS One; 2010 Jun; 5(6):e11306. PubMed ID: 20593011
[TBL] [Abstract][Full Text] [Related]
10. Long-term preservation of cones and improvement in visual function following gene therapy in a mouse model of leber congenital amaurosis caused by guanylate cyclase-1 deficiency.
Mihelec M; Pearson RA; Robbie SJ; Buch PK; Azam SA; Bainbridge JW; Smith AJ; Ali RR
Hum Gene Ther; 2011 Oct; 22(10):1179-90. PubMed ID: 21671801
[TBL] [Abstract][Full Text] [Related]
11. Determining consequences of retinal membrane guanylyl cyclase (RetGC1) deficiency in human Leber congenital amaurosis en route to therapy: residual cone-photoreceptor vision correlates with biochemical properties of the mutants.
Jacobson SG; Cideciyan AV; Peshenko IV; Sumaroka A; Olshevskaya EV; Cao L; Schwartz SB; Roman AJ; Olivares MB; Sadigh S; Yau KW; Heon E; Stone EM; Dizhoor AM
Hum Mol Genet; 2013 Jan; 22(1):168-83. PubMed ID: 23035049
[TBL] [Abstract][Full Text] [Related]
12. Light-driven cone arrestin translocation in cones of postnatal guanylate cyclase-1 knockout mouse retina treated with AAV-GC1.
Haire SE; Pang J; Boye SL; Sokal I; Craft CM; Palczewski K; Hauswirth WW; Semple-Rowland SL
Invest Ophthalmol Vis Sci; 2006 Sep; 47(9):3745-53. PubMed ID: 16936082
[TBL] [Abstract][Full Text] [Related]
13. A Mini-review: Animal Models of GUCY2D Leber Congenital Amaurosis (LCA1).
Boye SE
Adv Exp Med Biol; 2016; 854():253-8. PubMed ID: 26427419
[TBL] [Abstract][Full Text] [Related]
14. Enhanced CRISPR-Cas9 correction of Duchenne muscular dystrophy in mice by a self-complementary AAV delivery system.
Zhang Y; Li H; Min YL; Sanchez-Ortiz E; Huang J; Mireault AA; Shelton JM; Kim J; Mammen PPA; Bassel-Duby R; Olson EN
Sci Adv; 2020 Feb; 6(8):eaay6812. PubMed ID: 32128412
[TBL] [Abstract][Full Text] [Related]
15. Lentiviral expression of retinal guanylate cyclase-1 (RetGC1) restores vision in an avian model of childhood blindness.
Williams ML; Coleman JE; Haire SE; Aleman TS; Cideciyan AV; Sokal I; Palczewski K; Jacobson SG; Semple-Rowland SL
PLoS Med; 2006 Jun; 3(6):e201. PubMed ID: 16700630
[TBL] [Abstract][Full Text] [Related]
16. Postnatal Cardiac Gene Editing Using CRISPR/Cas9 With AAV9-Mediated Delivery of Short Guide RNAs Results in Mosaic Gene Disruption.
Johansen AK; Molenaar B; Versteeg D; Leitoguinho AR; Demkes C; Spanjaard B; de Ruiter H; Akbari Moqadam F; Kooijman L; Zentilin L; Giacca M; van Rooij E
Circ Res; 2017 Oct; 121(10):1168-1181. PubMed ID: 28851809
[TBL] [Abstract][Full Text] [Related]
17. In Vivo Ryr2 Editing Corrects Catecholaminergic Polymorphic Ventricular Tachycardia.
Pan X; Philippen L; Lahiri SK; Lee C; Park SH; Word TA; Li N; Jarrett KE; Gupta R; Reynolds JO; Lin J; Bao G; Lagor WR; Wehrens XHT
Circ Res; 2018 Sep; 123(8):953-963. PubMed ID: 30355031
[TBL] [Abstract][Full Text] [Related]
18. CRISPR-Based Genome Editing as a New Therapeutic Tool in Retinal Diseases.
Rasoulinejad SA; Maroufi F
Mol Biotechnol; 2021 Sep; 63(9):768-779. PubMed ID: 34057656
[TBL] [Abstract][Full Text] [Related]
19. Novel variants in GUCY2D causing retinopathy and the genotype-phenotype correlation.
Yi Z; Sun W; Xiao X; Li S; Jia X; Li X; Yu B; Wang P; Zhang Q
Exp Eye Res; 2021 Jul; 208():108637. PubMed ID: 34048777
[TBL] [Abstract][Full Text] [Related]
20. CRISPR/Cas9-Mediated Genome Editing as a Therapeutic Approach for Leber Congenital Amaurosis 10.
Ruan GX; Barry E; Yu D; Lukason M; Cheng SH; Scaria A
Mol Ther; 2017 Feb; 25(2):331-341. PubMed ID: 28109959
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]
