Detalhe da pesquisa
1.
The loop position of shRNAs and pre-miRNAs is critical for the accuracy of dicer processing in vivo.
Cell
; 151(4): 900-911, 2012 Nov 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-23141545
2.
Correlation of antigen expression with epigenetic modifications after rAAV delivery of a human factor IX variant in mice and rhesus macaques.
Mol Ther
; 2024 May 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-38715361
3.
The 3'tsRNAs are aminoacylated: Implications for their biogenesis.
PLoS Genet
; 17(7): e1009675, 2021 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-34324497
4.
Evolution of a Human-Specific Tandem Repeat Associated with ALS.
Am J Hum Genet
; 107(3): 445-460, 2020 09 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-32750315
5.
Novel NanoLuc substrates enable bright two-population bioluminescence imaging in animals.
Nat Methods
; 17(8): 852-860, 2020 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-32661427
6.
A transfer-RNA-derived small RNA regulates ribosome biogenesis.
Nature
; 552(7683): 57-62, 2017 12 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-29186115
7.
Evaluating the state of the science for adeno-associated virus integration: An integrated perspective.
Mol Ther
; 30(8): 2646-2663, 2022 08 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-35690906
8.
Promoterless, Nuclease-Free Genome Editing Confers a Growth Advantage for Corrected Hepatocytes in Mice With Methylmalonic Acidemia.
Hepatology
; 73(6): 2223-2237, 2021 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-32976669
9.
Evaluating the Genomic Parameters Governing rAAV-Mediated Homologous Recombination.
Mol Ther
; 29(3): 1028-1046, 2021 03 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-33248247
10.
Improved Genome Editing through Inhibition of FANCM and Members of the BTR Dissolvase Complex.
Mol Ther
; 29(3): 1016-1027, 2021 03 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-33678249
11.
A standardized ontology for naming tRNA-derived RNAs based on molecular origin.
Nat Methods
; 20(5): 627-628, 2023 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-36869120
12.
An orange calcium-modulated bioluminescent indicator for non-invasive activity imaging.
Nat Chem Biol
; 15(5): 433-436, 2019 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-30936501
13.
Transfer RNA-Derived Small RNAs: Another Layer of Gene Regulation and Novel Targets for Disease Therapeutics.
Mol Ther
; 28(11): 2340-2357, 2020 11 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-32956625
14.
Allele-Specific Silencing Ameliorates Restrictive Cardiomyopathy Attributable to a Human Myosin Regulatory Light Chain Mutation.
Circulation
; 140(9): 765-778, 2019 08 27.
Artigo
em Inglês
| MEDLINE | ID: mdl-31315475
15.
Selection and evaluation of clinically relevant AAV variants in a xenograft liver model.
Nature
; 506(7488): 382-6, 2014 Feb 20.
Artigo
em Inglês
| MEDLINE | ID: mdl-24390344
16.
Bioengineered AAV Capsids with Combined High Human Liver Transduction In Vivo and Unique Humoral Seroreactivity.
Mol Ther
; 26(1): 289-303, 2018 01 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-29055620
17.
Regulated complex assembly safeguards the fidelity of Sleeping Beauty transposition.
Nucleic Acids Res
; 45(1): 311-326, 2017 Jan 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-27913727
18.
Sequence-Modified Antibiotic Resistance Genes Provide Sustained Plasmid-Mediated Transgene Expression in Mammals.
Mol Ther
; 25(5): 1187-1198, 2017 05 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-28365028
19.
Survival Advantage of Both Human Hepatocyte Xenografts and Genome-Edited Hepatocytes for Treatment of α-1 Antitrypsin Deficiency.
Mol Ther
; 25(11): 2477-2489, 2017 11 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-29032169
20.
Long-term safety and efficacy of factor IX gene therapy in hemophilia B.
N Engl J Med
; 371(21): 1994-2004, 2014 Nov 20.
Artigo
em Inglês
| MEDLINE | ID: mdl-25409372