Detalhe da pesquisa
1.
Global genome decompaction leads to stochastic activation of gene expression as a first step toward fate commitment in human hematopoietic cells.
PLoS Biol
; 20(10): e3001849, 2022 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-36288293
2.
An empowered, clinically viable hematopoietic stem cell gene therapy for the treatment of multisystemic mucopolysaccharidosis type II.
Mol Ther
; 32(3): 619-636, 2024 Mar 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-38310355
3.
Lentiviral standards to determine the sensitivity of assays that quantify lentiviral vector copy numbers and genomic insertion sites in cells.
Gene Ther
; 29(9): 536-543, 2022 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-35194185
4.
Integrated time-lapse and single-cell transcription studies highlight the variable and dynamic nature of human hematopoietic cell fate commitment.
PLoS Biol
; 15(7): e2001867, 2017 Jul.
Artigo
em Inglês
| MEDLINE | ID: mdl-28749943
5.
Genome Editing of Expanded CTG Repeats within the Human DMPK Gene Reduces Nuclear RNA Foci in the Muscle of DM1 Mice.
Mol Ther
; 27(8): 1372-1388, 2019 08 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-31253581
6.
Serotype-specific Binding Properties and Nanoparticle Characteristics Contribute to the Immunogenicity of rAAV1 Vectors.
Mol Ther
; 23(6): 1022-1033, 2015 Jun.
Artigo
em Inglês
| MEDLINE | ID: mdl-25881000
7.
Hypoxic culture conditions enhance the generation of regulatory T cells.
Immunology
; 144(3): 431-443, 2015 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-25243909
8.
Evaluation of diversity indices to estimate clonal dominance in gene therapy studies.
Mol Ther Methods Clin Dev
; 29: 418-425, 2023 Jun 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-37251980
9.
Deciphering the Molecular Mechanism of Incurable Muscle Disease by a Novel Method for the Interpretation of miRNA Dysregulation.
Noncoding RNA
; 8(4)2022 Jun 30.
Artigo
em Inglês
| MEDLINE | ID: mdl-35893231
10.
Cholesterol metabolism is a potential therapeutic target in Duchenne muscular dystrophy.
J Cachexia Sarcopenia Muscle
; 12(3): 677-693, 2021 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-34037326
11.
Author Correction: Ex vivo editing of human hematopoietic stem cells for erythroid expression of therapeutic proteins.
Nat Commun
; 11(1): 4146, 2020 08 13.
Artigo
em Inglês
| MEDLINE | ID: mdl-32792546
12.
Ex vivo editing of human hematopoietic stem cells for erythroid expression of therapeutic proteins.
Nat Commun
; 11(1): 3778, 2020 07 29.
Artigo
em Inglês
| MEDLINE | ID: mdl-32728076
13.
Constraints on Human CD34+ Cell Fate due to Lentiviral Vectors Can Be Relieved by Valproic Acid.
Hum Gene Ther
; 30(8): 1023-1034, 2019 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-30977420
14.
An AAV-SGCG Dose-Response Study in a γ-Sarcoglycanopathy Mouse Model in the Context of Mechanical Stress.
Mol Ther Methods Clin Dev
; 13: 494-502, 2019 Jun 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-31194043
15.
Temporary Reduction of Membrane CD4 with the Antioxidant MnTBAP Is Sufficient to Prevent Immune Responses Induced by Gene Transfer.
Mol Ther Methods Clin Dev
; 14: 285-299, 2019 Sep 13.
Artigo
em Inglês
| MEDLINE | ID: mdl-31497619
16.
Titin splicing regulates cardiotoxicity associated with calpain 3 gene therapy for limb-girdle muscular dystrophy type 2A.
Sci Transl Med
; 11(520)2019 11 27.
Artigo
em Inglês
| MEDLINE | ID: mdl-31776291
17.
Author Correction: miR-379 links glucocorticoid treatment with mitochondrial response in Duchenne muscular dystrophy.
Sci Rep
; 14(1): 7441, 2024 Mar 28.
Artigo
em Inglês
| MEDLINE | ID: mdl-38548795
18.
Pre-clinical Development of a Lentiviral Vector Expressing the Anti-sickling ßAS3 Globin for Gene Therapy for Sickle Cell Disease.
Mol Ther Methods Clin Dev
; 11: 167-179, 2018 Dec 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-30533448
19.
Preclinical Development of a Lentiviral Vector for Gene Therapy of X-Linked Severe Combined Immunodeficiency.
Mol Ther Methods Clin Dev
; 9: 257-269, 2018 Jun 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-29707600
20.
"RCL-Pooling Assay": A Simplified Method for the Detection of Replication-Competent Lentiviruses in Vector Batches Using Sequential Pooling.
Hum Gene Ther
; 27(2): 202-10, 2016 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-26886834