Detalhe da pesquisa
1.
rAAV-mediated over-expression of acid ceramidase prevents retinopathy in a mouse model of Farber lipogranulomatosis.
Gene Ther
; 30(3-4): 297-308, 2023 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-35902747
2.
Genetic ablation of acid ceramidase in Krabbe disease confirms the psychosine hypothesis and identifies a new therapeutic target.
Proc Natl Acad Sci U S A
; 116(40): 20097-20103, 2019 10 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-31527255
3.
Acid Ceramidase Deficiency in Mice Leads to Severe Ocular Pathology and Visual Impairment.
Am J Pathol
; 189(2): 320-338, 2019 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-30472209
4.
Spinal muscular atrophy-like phenotype in a mouse model of acid ceramidase deficiency.
Commun Biol
; 6(1): 560, 2023 05 25.
Artigo
em Inglês
| MEDLINE | ID: mdl-37231125
5.
Lipids and the cancer stemness regulatory system in acute myeloid leukemia.
Essays Biochem
; 66(4): 333-344, 2022 09 16.
Artigo
em Inglês
| MEDLINE | ID: mdl-35996953
6.
Autologous, lentivirus-modified, T-rapa cell "micropharmacies" for lysosomal storage disorders.
EMBO Mol Med
; 14(4): e14297, 2022 04 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-35298086
7.
An update on gene therapy for lysosomal storage disorders.
Expert Opin Biol Ther
; 19(7): 655-670, 2019 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-31056978
8.
Lentivector Iterations and Pre-Clinical Scale-Up/Toxicity Testing: Targeting Mobilized CD34+ Cells for Correction of Fabry Disease.
Mol Ther Methods Clin Dev
; 5: 241-258, 2017 Jun 16.
Artigo
em Inglês
| MEDLINE | ID: mdl-28603745
9.
Towards in vivo amplification: Overcoming hurdles in the use of hematopoietic stem cells in transplantation and gene therapy.
World J Stem Cells
; 7(11): 1233-50, 2015 Dec 26.
Artigo
em Inglês
| MEDLINE | ID: mdl-26730268