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.
Liver-specific adiponectin gene therapy suppresses microglial NLRP3-inflammasome activation for treating Alzheimer's disease.
J Neuroinflammation
; 21(1): 77, 2024 Mar 27.
Artigo
em Inglês
| MEDLINE | ID: mdl-38539253
3.
Gene therapy for urea cycle defects: An update from historical perspectives to future prospects.
J Inherit Metab Dis
; 47(1): 50-62, 2024 Jan.
Artigo
em Inglês
| MEDLINE | ID: mdl-37026568
4.
Structural and functional characterization of capsid binding by anti-AAV9 monoclonal antibodies from infants after SMA gene therapy.
Mol Ther
; 31(7): 1979-1993, 2023 07 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-37012705
5.
Gain-of-function factor H-related 5 protein impairs glomerular complement regulation resulting in kidney damage.
Proc Natl Acad Sci U S A
; 118(13)2021 03 30.
Artigo
em Inglês
| MEDLINE | ID: mdl-33753502
6.
Liver-specific deletion of miR-181ab1 reduces liver tumour progression via upregulation of CBX7.
Cell Mol Life Sci
; 79(8): 443, 2022 Jul 22.
Artigo
em Inglês
| MEDLINE | ID: mdl-35867177
7.
Gene Therapy for Paediatric Homozygous Familial Hypercholesterolaemia.
Heart Lung Circ
; 32(7): 769-779, 2023 Jul.
Artigo
em Inglês
| MEDLINE | ID: mdl-37012174
8.
In Search of Adeno-Associated Virus Vectors With Enhanced Cardiac Tropism for Gene Therapy.
Heart Lung Circ
; 32(7): 816-824, 2023 Jul.
Artigo
em Inglês
| MEDLINE | ID: mdl-37451880
9.
Systemic AAV8-mediated delivery of a functional copy of muscle glycogen phosphorylase (Pygm) ameliorates disease in a murine model of McArdle disease.
Hum Mol Genet
; 29(1): 20-30, 2020 01 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-31511858
10.
The Balance of Stromal BMP Signaling Mediated by GREM1 and ISLR Drives Colorectal Carcinogenesis.
Gastroenterology
; 160(4): 1224-1239.e30, 2021 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-33197448
11.
The implementation of newborn screening for spinal muscular atrophy: the Australian experience.
Genet Med
; 22(3): 557-565, 2020 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-31607747
12.
The diagnostic utility of genome sequencing in a pediatric cohort with suspected mitochondrial disease.
Genet Med
; 22(7): 1254-1261, 2020 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-32313153
13.
Prevention of Cholestatic Liver Disease and Reduced Tumorigenicity in a Murine Model of PFIC Type 3 Using Hybrid AAV-piggyBac Gene Therapy.
Hepatology
; 70(6): 2047-2061, 2019 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-31099022
14.
Great expectations: virus-mediated gene therapy in neurological disorders.
J Neurol Neurosurg Psychiatry
; 91(8): 849-860, 2020 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-32503884
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.
AAV-mediated gene delivery of the calreticulin anti-angiogenic domain inhibits ocular neovascularization.
Angiogenesis
; 21(1): 95-109, 2018 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-29318471
18.
Gene therapy clinical trials worldwide to 2017: An update.
J Gene Med
; 20(5): e3015, 2018 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-29575374
19.
Partial pancreatic transdifferentiation of primary human hepatocytes in the livers of a humanised mouse model.
J Gene Med
; 20(5): e3017, 2018 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-29578255
20.
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