BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

271 related articles for article (PubMed ID: 27653967)

  • 1. 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]  

  • 2. 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]  

  • 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. AAV2 gene therapy readministration in three adults with congenital blindness.
    Bennett J; Ashtari M; Wellman J; Marshall KA; Cyckowski LL; Chung DC; McCague S; Pierce EA; Chen Y; Bennicelli JL; Zhu X; Ying GS; Sun J; Wright JF; Auricchio A; Simonelli F; Shindler KS; Mingozzi F; High KA; Maguire AM
    Sci Transl Med; 2012 Feb; 4(120):120ra15. PubMed ID: 22323828
    [TBL] [Abstract][Full Text] [Related]  

  • 5. Long-term effect of gene therapy on Leber's congenital amaurosis.
    Bainbridge JW; Mehat MS; Sundaram V; Robbie SJ; Barker SE; Ripamonti C; Georgiadis A; Mowat FM; Beattie SG; Gardner PJ; Feathers KL; Luong VA; Yzer S; Balaggan K; Viswanathan A; de Ravel TJ; Casteels I; Holder GE; Tyler N; Fitzke FW; Weleber RG; Nardini M; Moore AT; Thompson DA; Petersen-Jones SM; Michaelides M; van den Born LI; Stockman A; Smith AJ; Rubin G; Ali RR
    N Engl J Med; 2015 May; 372(20):1887-97. PubMed ID: 25938638
    [TBL] [Abstract][Full Text] [Related]  

  • 6. Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years.
    Jacobson SG; Cideciyan AV; Ratnakaram R; Heon E; Schwartz SB; Roman AJ; Peden MC; Aleman TS; Boye SL; Sumaroka A; Conlon TJ; Calcedo R; Pang JJ; Erger KE; Olivares MB; Mullins CL; Swider M; Kaushal S; Feuer WJ; Iannaccone A; Fishman GA; Stone EM; Byrne BJ; Hauswirth WW
    Arch Ophthalmol; 2012 Jan; 130(1):9-24. PubMed ID: 21911650
    [TBL] [Abstract][Full Text] [Related]  

  • 7. Results at 2 Years after Gene Therapy for RPE65-Deficient Leber Congenital Amaurosis and Severe Early-Childhood-Onset Retinal Dystrophy.
    Weleber RG; Pennesi ME; Wilson DJ; Kaushal S; Erker LR; Jensen L; McBride MT; Flotte TR; Humphries M; Calcedo R; Hauswirth WW; Chulay JD; Stout JT
    Ophthalmology; 2016 Jul; 123(7):1606-20. PubMed ID: 27102010
    [TBL] [Abstract][Full Text] [Related]  

  • 8. 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]  

  • 9. 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]  

  • 10. Gene therapy for retinitis pigmentosa and Leber congenital amaurosis caused by defects in AIPL1: effective rescue of mouse models of partial and complete Aipl1 deficiency using AAV2/2 and AAV2/8 vectors.
    Tan MH; Smith AJ; Pawlyk B; Xu X; Liu X; Bainbridge JB; Basche M; McIntosh J; Tran HV; Nathwani A; Li T; Ali RR
    Hum Mol Genet; 2009 Jun; 18(12):2099-114. PubMed ID: 19299492
    [TBL] [Abstract][Full Text] [Related]  

  • 11. Long-term restoration of rod and cone vision by single dose rAAV-mediated gene transfer to the retina in a canine model of childhood blindness.
    Acland GM; Aguirre GD; Bennett J; Aleman TS; Cideciyan AV; Bennicelli J; Dejneka NS; Pearce-Kelling SE; Maguire AM; Palczewski K; Hauswirth WW; Jacobson SG
    Mol Ther; 2005 Dec; 12(6):1072-82. PubMed ID: 16226919
    [TBL] [Abstract][Full Text] [Related]  

  • 12. 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]  

  • 13. 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]  

  • 14. Reversal of blindness in animal models of leber congenital amaurosis using optimized AAV2-mediated gene transfer.
    Bennicelli J; Wright JF; Komaromy A; Jacobs JB; Hauck B; Zelenaia O; Mingozzi F; Hui D; Chung D; Rex TS; Wei Z; Qu G; Zhou S; Zeiss C; Arruda VR; Acland GM; Dell'Osso LF; High KA; Maguire AM; Bennett J
    Mol Ther; 2008 Mar; 16(3):458-65. PubMed ID: 18209734
    [TBL] [Abstract][Full Text] [Related]  

  • 15. Gene therapy using self-complementary Y733F capsid mutant AAV2/8 restores vision in a model of early onset Leber congenital amaurosis.
    Ku CA; Chiodo VA; Boye SL; Goldberg AF; Li T; Hauswirth WW; Ramamurthy V
    Hum Mol Genet; 2011 Dec; 20(23):4569-81. PubMed ID: 21880665
    [TBL] [Abstract][Full Text] [Related]  

  • 16. Gene therapy following subretinal AAV5 vector delivery is not affected by a previous intravitreal AAV5 vector administration in the partner eye.
    Li W; Kong F; Li X; Dai X; Liu X; Zheng Q; Wu R; Zhou X; Lü F; Chang B; Li Q; Hauswirth WW; Qu J; Pang JJ
    Mol Vis; 2009; 15():267-75. PubMed ID: 19190735
    [TBL] [Abstract][Full Text] [Related]  

  • 17. Subretinal delivery of adeno-associated virus serotype 2 results in minimal immune responses that allow repeat vector administration in immunocompetent mice.
    Barker SE; Broderick CA; Robbie SJ; Duran Y; Natkunarajah M; Buch P; Balaggan KS; MacLaren RE; Bainbridge JW; Smith AJ; Ali RR
    J Gene Med; 2009 Jun; 11(6):486-97. PubMed ID: 19340848
    [TBL] [Abstract][Full Text] [Related]  

  • 18. 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]  

  • 19. Overexpression of Type 3 Iodothyronine Deiodinase Reduces Cone Death in the Leber Congenital Amaurosis Model Mice.
    Yang F; Ma H; Boye SL; Hauswirth WW; Ding XQ
    Adv Exp Med Biol; 2018; 1074():125-131. PubMed ID: 29721936
    [TBL] [Abstract][Full Text] [Related]  

  • 20. Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration.
    Simonelli F; Maguire AM; Testa F; Pierce EA; Mingozzi F; Bennicelli JL; Rossi S; Marshall K; Banfi S; Surace EM; Sun J; Redmond TM; Zhu X; Shindler KS; Ying GS; Ziviello C; Acerra C; Wright JF; McDonnell JW; High KA; Bennett J; Auricchio A
    Mol Ther; 2010 Mar; 18(3):643-50. PubMed ID: 19953081
    [TBL] [Abstract][Full Text] [Related]  

    [Next]    [New Search]
    of 14.