Detalhe da pesquisa
1.
mRNA Therapy Improves Metabolic and Behavioral Abnormalities in a Murine Model of Citrin Deficiency.
Mol Ther
; 27(7): 1242-1251, 2019 07 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-31056400
2.
A Novel Amino Lipid Series for mRNA Delivery: Improved Endosomal Escape and Sustained Pharmacology and Safety in Non-human Primates.
Mol Ther
; 26(6): 1509-1519, 2018 06 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-29653760
3.
Macrocyclic MEK1/2 inhibitor with efficacy in a mouse model of cardiomyopathy caused by lamin A/C gene mutation.
Bioorg Med Chem
; 25(3): 1004-1013, 2017 02 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-28011205
4.
Systemic messenger RNA as an etiological treatment for acute intermittent porphyria.
Nat Med
; 24(12): 1899-1909, 2018 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-30297912
5.
Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia.
Cell Rep
; 21(12): 3548-3558, 2017 Dec 19.
Artigo
em Inglês
| MEDLINE | ID: mdl-29262333
6.
Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia.
Cell Rep
; 24(9): 2520, 2018 Aug 28.
Artigo
em Inglês
| MEDLINE | ID: mdl-30157442