Detalhe da pesquisa
1.
Development of CNS tropic AAV1-like variants with reduced liver-targeting following systemic administration in mice.
Mol Ther
; 32(3): 818-836, 2024 Mar 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-38297833
2.
Bone Marrow Transplantation for Treatment of the Col1a2+/G610C Osteogenesis Imperfecta Mouse Model.
Calcif Tissue Int
; 104(4): 426-436, 2019 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-30535573
3.
Gene therapy clinical trials worldwide to 2017: An update.
J Gene Med
; 20(5): e3015, 2018 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-29575374
4.
Thymocyte self-renewal and oncogenic risk in immunodeficient mouse models: relevance for human gene therapy clinical trials targeting haematopoietic stem cell populations?
Mamm Genome
; 29(11-12): 771-776, 2018 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-30182300
5.
Impact of next-generation sequencing error on analysis of barcoded plasmid libraries of known complexity and sequence.
Nucleic Acids Res
; 42(16): e129, 2014.
Artigo
em Inglês
| MEDLINE | ID: mdl-25013183
6.
AAV-delivered hepato-adrenal cooperativity in steroidogenesis: Implications for gene therapy for congenital adrenal hyperplasia.
Mol Ther Methods Clin Dev
; 32(2): 101232, 2024 Jun 13.
Artigo
em Inglês
| MEDLINE | ID: mdl-38558568
7.
Novel AAV variants with improved tropism for human Schwann cells.
Mol Ther Methods Clin Dev
; 32(2): 101234, 2024 Jun 13.
Artigo
em Inglês
| MEDLINE | ID: mdl-38558569
8.
Gene therapy clinical trials worldwide to 2012 - an update.
J Gene Med
; 15(2): 65-77, 2013 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-23355455
9.
Development of new adeno-associated virus capsid variants for targeted gene delivery to human cardiomyocytes.
Mol Ther Methods Clin Dev
; 30: 459-473, 2023 Sep 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-37674904
10.
Gene therapy: progress in childhood disease.
J Paediatr Child Health
; 48(6): 466-71, 2012 Jun.
Artigo
em Inglês
| MEDLINE | ID: mdl-22017270
11.
Curative Cell and Gene Therapy for Osteogenesis Imperfecta.
J Bone Miner Res
; 37(5): 826-836, 2022 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-35306687
12.
Lymphomagenesis in SCID-X1 mice following lentivirus-mediated phenotype correction independent of insertional mutagenesis and gammac overexpression.
Mol Ther
; 18(5): 965-76, 2010 May.
Artigo
em Inglês
| MEDLINE | ID: mdl-20354504
13.
Genome editing in the human liver: Progress and translational considerations.
Prog Mol Biol Transl Sci
; 182: 257-288, 2021.
Artigo
em Inglês
| MEDLINE | ID: mdl-34175044
14.
Efficient in vivo editing of OTC-deficient patient-derived primary human hepatocytes.
JHEP Rep
; 2(1): 100065, 2020 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-32039406
15.
Restoring the natural tropism of AAV2 vectors for human liver.
Sci Transl Med
; 12(560)2020 09 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-32908003
16.
Gene transfer of connexin43 mutants attenuates coupling in cardiomyocytes: novel basis for modulation of cardiac conduction by gene therapy.
Circ Res
; 100(11): 1597-604, 2007 Jun 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-17495226
17.
Myoblast sensitivity and fibroblast insensitivity to osteogenic conversion by BMP-2 correlates with the expression of Bmpr-1a.
BMC Musculoskelet Disord
; 10: 51, 2009 May 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-19442313
18.
Codon-Optimization of Wild-Type Adeno-Associated Virus Capsid Sequences Enhances DNA Family Shuffling while Conserving Functionality.
Mol Ther Methods Clin Dev
; 12: 71-84, 2019 Mar 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-30534580
19.
Limiting Thymic Precursor Supply Increases the Risk of Lymphoid Malignancy in Murine X-Linked Severe Combined Immunodeficiency.
Mol Ther Nucleic Acids
; 6: 1-14, 2017 Mar 17.
Artigo
em Inglês
| MEDLINE | ID: mdl-28325276
20.
Identification of liver-specific enhancer-promoter activity in the 3' untranslated region of the wild-type AAV2 genome.
Nat Genet
; 49(8): 1267-1273, 2017 Aug.
Artigo
em Inglês
| MEDLINE | ID: mdl-28628105