Detalhe da pesquisa
1.
5 Year Expression and Neutrophil Defect Repair after Gene Therapy in Alpha-1 Antitrypsin Deficiency.
Mol Ther
; 25(6): 1387-1394, 2017 06 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-28408179
2.
Optimization of Retinal Gene Therapy for X-Linked Retinitis Pigmentosa Due to RPGR Mutations.
Mol Ther
; 25(8): 1866-1880, 2017 08 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-28566226
3.
REPEATABILITY AND LONGITUDINAL ASSESSMENT OF FOVEAL CONE STRUCTURE IN CNGB3-ASSOCIATED ACHROMATOPSIA.
Retina
; 37(10): 1956-1966, 2017 Oct.
Artigo
em Inglês
| MEDLINE | ID: mdl-28145975
4.
Results at 2 Years after Gene Therapy for RPE65-Deficient Leber Congenital Amaurosis and Severe Early-Childhood-Onset Retinal Dystrophy.
Ophthalmology
; 123(7): 1606-20, 2016 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-27102010
5.
Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy.
Proc Natl Acad Sci U S A
; 106(38): 16363-8, 2009 Sep 22.
Artigo
em Inglês
| MEDLINE | ID: mdl-19706466
6.
Dose Range Finding Studies with Two RPGR Transgenes in a Canine Model of X-Linked Retinitis Pigmentosa Treated with Subretinal Gene Therapy.
Hum Gene Ther
; 31(13-14): 743-755, 2020 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-32414297
7.
Toxicity and Efficacy Evaluation of an Adeno-Associated Virus Vector Expressing Codon-Optimized RPGR Delivered by Subretinal Injection in a Canine Model of X-linked Retinitis Pigmentosa.
Hum Gene Ther
; 31(3-4): 253-267, 2020 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-31910043
8.
Bridging from Intramuscular to Limb Perfusion Delivery of rAAV: Optimization in a Non-human Primate Study.
Mol Ther Methods Clin Dev
; 13: 233-242, 2019 Jun 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-30828586
9.
Results at 5 Years After Gene Therapy for RPE65-Deficient Retinal Dystrophy.
Hum Gene Ther
; 29(12): 1428-1437, 2018 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-29869534
10.
Prospective Evaluation of Patients With X-Linked Retinoschisis During 18 Months.
Invest Ophthalmol Vis Sci
; 59(15): 5941-5956, 2018 12 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-30551202
11.
Toxicology and Pharmacology of an AAV Vector Expressing Codon-Optimized RPGR in RPGR-Deficient Rd9 Mice.
Hum Gene Ther Clin Dev
; 29(4): 188-197, 2018 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-30280954
12.
Safety and Efficacy of AAV5 Vectors Expressing Human or Canine CNGB3 in CNGB3-Mutant Dogs.
Hum Gene Ther Clin Dev
; 28(4): 197-207, 2017 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-29020838
13.
Safety and Efficacy Evaluation of rAAV2tYF-PR1.7-hCNGA3 Vector Delivered by Subretinal Injection in CNGA3 Mutant Achromatopsia Sheep.
Hum Gene Ther Clin Dev
; 28(2): 96-107, 2017 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-28478700
14.
Recombinant Adeno-Associated Virus Vector Genomes Take the Form of Long-Lived, Transcriptionally Competent Episomes in Human Muscle.
Hum Gene Ther
; 27(1): 32-42, 2016 Jan.
Artigo
em Inglês
| MEDLINE | ID: mdl-26650966
15.
Cone-Specific Promoters for Gene Therapy of Achromatopsia and Other Retinal Diseases.
Hum Gene Ther
; 27(1): 72-82, 2016 Jan.
Artigo
em Inglês
| MEDLINE | ID: mdl-26603570
16.
Safety and Biodistribution Evaluation in CNGB3-Deficient Mice of rAAV2tYF-PR1.7-hCNGB3, a Recombinant AAV Vector for Treatment of Achromatopsia.
Hum Gene Ther Clin Dev
; 27(1): 27-36, 2016 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-27003752
17.
Safety and Biodistribution Evaluation in Cynomolgus Macaques of rAAV2tYF-PR1.7-hCNGB3, a Recombinant AAV Vector for Treatment of Achromatopsia.
Hum Gene Ther Clin Dev
; 27(1): 37-48, 2016 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-27003753
18.
Residual Foveal Cone Structure in CNGB3-Associated Achromatopsia.
Invest Ophthalmol Vis Sci
; 57(10): 3984-95, 2016 08 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-27479814
19.
Safety and Biodistribution Evaluation of rAAV2tYF-CB-hRS1, a Recombinant Adeno-Associated Virus Vector Expressing Retinoschisin, in RS1-Deficient Mice.
Hum Gene Ther Clin Dev
; 26(3): 177-84, 2015 Sep.
Artigo
em Inglês
| MEDLINE | ID: mdl-26390091
20.
Safety and Biodistribution Evaluation in Cynomolgus Macaques of rAAV2tYF-CB-hRS1, a Recombinant Adeno-Associated Virus Vector Expressing Retinoschisin.
Hum Gene Ther Clin Dev
; 26(3): 165-76, 2015 Sep.
Artigo
em Inglês
| MEDLINE | ID: mdl-26390090