BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

297 related articles for article (PubMed ID: 31692906)

  • 1. CRISPR-Cas9-mediated therapeutic editing of
    Jo DH; Song DW; Cho CS; Kim UG; Lee KJ; Lee K; Park SW; Kim D; Kim JH; Kim JS; Kim S; Kim JH; Lee JM
    Sci Adv; 2019 Oct; 5(10):eaax1210. PubMed ID: 31692906
    [TBL] [Abstract][Full Text] [Related]  

  • 2. Pharmacological Amelioration of Cone Survival and Vision in a Mouse Model for Leber Congenital Amaurosis.
    Li S; Samardzija M; Yang Z; Grimm C; Jin M
    J Neurosci; 2016 May; 36(21):5808-19. PubMed ID: 27225770
    [TBL] [Abstract][Full Text] [Related]  

  • 3. Gene therapy rescues cone structure and function in the 3-month-old rd12 mouse: a model for midcourse RPE65 leber congenital amaurosis.
    Li X; Li W; Dai X; Kong F; Zheng Q; Zhou X; Lü F; Chang B; Rohrer B; Hauswirth WW; Qu J; Pang JJ
    Invest Ophthalmol Vis Sci; 2011 Jan; 52(1):7-15. PubMed ID: 21169527
    [TBL] [Abstract][Full Text] [Related]  

  • 4. Retinal degeneration 12 (rd12): a new, spontaneously arising mouse model for human Leber congenital amaurosis (LCA).
    Pang JJ; Chang B; Hawes NL; Hurd RE; Davisson MT; Li J; Noorwez SM; Malhotra R; McDowell JH; Kaushal S; Hauswirth WW; Nusinowitz S; Thompson DA; Heckenlively JR
    Mol Vis; 2005 Feb; 11():152-62. PubMed ID: 15765048
    [TBL] [Abstract][Full Text] [Related]  

  • 5. Visual function restoration in a mouse model of Leber congenital amaurosis via therapeutic base editing.
    Jo DH; Jang HK; Cho CS; Han JH; Ryu G; Jung Y; Bae S; Kim JH
    Mol Ther Nucleic Acids; 2023 Mar; 31():16-27. PubMed ID: 36589710
    [TBL] [Abstract][Full Text] [Related]  

  • 6. 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]  

  • 7. Successful gene therapy in older Rpe65-deficient dogs following subretinal injection of an adeno-associated vector expressing RPE65.
    Annear MJ; Mowat FM; Bartoe JT; Querubin J; Azam SA; Basche M; Curran PG; Smith AJ; Bainbridge JW; Ali RR; Petersen-Jones SM
    Hum Gene Ther; 2013 Oct; 24(10):883-93. PubMed ID: 24028205
    [TBL] [Abstract][Full Text] [Related]  

  • 8. [Gene therapy for vision restoration in patients with Leber congenital amaurosis (LCA) due to RPE65 gene mutations: beginning the phase IV trial].
    Chacón-Camacho ÓF; Zenteno JC
    Gac Med Mex; 2017; 153(2):276-278. PubMed ID: 28474714
    [TBL] [Abstract][Full Text] [Related]  

  • 9. Intervisit variability of visual parameters in Leber congenital amaurosis caused by RPE65 mutations.
    Roman AJ; Cideciyan AV; Schwartz SB; Olivares MB; Heon E; Jacobson SG
    Invest Ophthalmol Vis Sci; 2013 Feb; 54(2):1378-83. PubMed ID: 23341016
    [TBL] [Abstract][Full Text] [Related]  

  • 10. Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement.
    Cideciyan AV; Jacobson SG; Beltran WA; Sumaroka A; Swider M; Iwabe S; Roman AJ; Olivares MB; Schwartz SB; Komáromy AM; Hauswirth WW; Aguirre GD
    Proc Natl Acad Sci U S A; 2013 Feb; 110(6):E517-25. PubMed ID: 23341635
    [TBL] [Abstract][Full Text] [Related]  

  • 11. Available Evidence on Leber Congenital Amaurosis and Gene Therapy.
    Alkharashi M; Fulton AB
    Semin Ophthalmol; 2017; 32(1):14-21. PubMed ID: 27686653
    [TBL] [Abstract][Full Text] [Related]  

  • 12. Inverse correlation between fatty acid transport protein 4 and vision in Leber congenital amaurosis associated with RPE65 mutation.
    Li S; Gordon WC; Bazan NG; Jin M
    Proc Natl Acad Sci U S A; 2020 Dec; 117(50):32114-32123. PubMed ID: 33257550
    [TBL] [Abstract][Full Text] [Related]  

  • 13. A high prevalence of biallelic RPE65 mutations in Costa Rican children with Leber congenital amaurosis and early-onset retinal dystrophy.
    Glen WB; Peterseim MMW; Badilla R; Znoyko I; Bourg A; Wilson R; Hardiman G; Wolff D; Martinez J
    Ophthalmic Genet; 2019 Apr; 40(2):110-117. PubMed ID: 30870047
    [TBL] [Abstract][Full Text] [Related]  

  • 14. Safety and Long-Term Efficacy of AAV4 Gene Therapy in Patients with RPE65 Leber Congenital Amaurosis.
    Le Meur G; Lebranchu P; Billaud F; Adjali O; Schmitt S; Bézieau S; Péréon Y; Valabregue R; Ivan C; Darmon C; Moullier P; Rolling F; Weber M
    Mol Ther; 2018 Jan; 26(1):256-268. PubMed ID: 29033008
    [TBL] [Abstract][Full Text] [Related]  

  • 15. Novel RPE65 mutations associated with Leber congenital amaurosis in Chinese patients.
    Xu F; Dong Q; Liu L; Li H; Liang X; Jiang R; Sui R; Dong F
    Mol Vis; 2012; 18():744-50. PubMed ID: 22509104
    [TBL] [Abstract][Full Text] [Related]  

  • 16. Gene therapy for RPE65-related retinal disease.
    Miraldi Utz V; Coussa RG; Antaki F; Traboulsi EI
    Ophthalmic Genet; 2018 Dec; 39(6):671-677. PubMed ID: 30335549
    [TBL] [Abstract][Full Text] [Related]  

  • 17. Gene therapy for Leber congenital amaurosis: advances and future directions.
    Hufnagel RB; Ahmed ZM; Corrêa ZM; Sisk RA
    Graefes Arch Clin Exp Ophthalmol; 2012 Aug; 250(8):1117-28. PubMed ID: 22644094
    [TBL] [Abstract][Full Text] [Related]  

  • 18. Development of an optimized AAV2/5 gene therapy vector for Leber congenital amaurosis owing to defects in RPE65.
    Georgiadis A; Duran Y; Ribeiro J; Abelleira-Hervas L; Robbie SJ; Sünkel-Laing B; Fourali S; Gonzalez-Cordero A; Cristante E; Michaelides M; Bainbridge JW; Smith AJ; Ali RR
    Gene Ther; 2016 Dec; 23(12):857-862. PubMed ID: 27653967
    [TBL] [Abstract][Full Text] [Related]  

  • 19. Results at 5 Years After Gene Therapy for RPE65-Deficient Retinal Dystrophy.
    Pennesi ME; Weleber RG; Yang P; Whitebirch C; Thean B; Flotte TR; Humphries M; Chegarnov E; Beasley KN; Stout JT; Chulay JD
    Hum Gene Ther; 2018 Dec; 29(12):1428-1437. PubMed ID: 29869534
    [TBL] [Abstract][Full Text] [Related]  

  • 20. The Status of RPE65 Gene Therapy Trials: Safety and Efficacy.
    Pierce EA; Bennett J
    Cold Spring Harb Perspect Med; 2015 Jan; 5(9):a017285. PubMed ID: 25635059
    [TBL] [Abstract][Full Text] [Related]  

    [Next]    [New Search]
    of 15.