Detalhe da pesquisa
1.
Systemic mRNA Therapy for the Treatment of Fabry Disease: Preclinical Studies in Wild-Type Mice, Fabry Mouse Model, and Wild-Type Non-human Primates.
Am J Hum Genet
; 104(4): 625-637, 2019 04 04.
Artigo
Inglês
| MEDLINE | ID: mdl-30879639
2.
mRNA Therapy Improves Metabolic and Behavioral Abnormalities in a Murine Model of Citrin Deficiency.
Mol Ther
; 27(7): 1242-1251, 2019 07 03.
Artigo
Inglês
| MEDLINE | ID: mdl-31056400
3.
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
Inglês
| MEDLINE | ID: mdl-29653760
4.
Boosting Intracellular Delivery of Lipid Nanoparticle-Encapsulated mRNA.
Nano Lett
; 17(9): 5711-5718, 2017 09 13.
Artigo
Inglês
| MEDLINE | ID: mdl-28836442
5.
Pharmacodynamic Profiling of a Siderophore-Conjugated Monocarbam in Pseudomonas aeruginosa: Assessing the Risk for Resistance and Attenuated Efficacy.
Antimicrob Agents Chemother
; 59(12): 7743-52, 2015 Dec.
Artigo
Inglês
| MEDLINE | ID: mdl-26438502
6.
Negishi cross-coupling enabled synthesis of novel NAD(+)-dependent DNA ligase inhibitors and SAR development.
Bioorg Med Chem Lett
; 25(22): 5172-7, 2015 Nov 15.
Artigo
Inglês
| MEDLINE | ID: mdl-26463129
7.
Dual mRNA therapy restores metabolic function in long-term studies in mice with propionic acidemia.
Nat Commun
; 11(1): 5339, 2020 10 21.
Artigo
Inglês
| MEDLINE | ID: mdl-33087718
8.
Accelerated Blood Clearance of Lipid Nanoparticles Entails a Biphasic Humoral Response of B-1 Followed by B-2 Lymphocytes to Distinct Antigenic Moieties.
Immunohorizons
; 3(7): 282-293, 2019 07 11.
Artigo
Inglês
| MEDLINE | ID: mdl-31356158
9.
Optimization of Lipid Nanoparticles for Intramuscular Administration of mRNA Vaccines.
Mol Ther Nucleic Acids
; 15: 1-11, 2019 Apr 15.
Artigo
Inglês
| MEDLINE | ID: mdl-30785039
10.
Systemic messenger RNA as an etiological treatment for acute intermittent porphyria.
Nat Med
; 24(12): 1899-1909, 2018 12.
Artigo
Inglês
| MEDLINE | ID: mdl-30297912
11.
Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia.
Cell Rep
; 21(12): 3548-3558, 2017 Dec 19.
Artigo
Inglês
| MEDLINE | ID: mdl-29262333
12.
SAR and Structural Analysis of Siderophore-Conjugated Monocarbam Inhibitors of Pseudomonas aeruginosa PBP3.
ACS Med Chem Lett
; 6(5): 537-42, 2015 May 14.
Artigo
Inglês
| MEDLINE | ID: mdl-26005529
13.
Discovery of efficacious Pseudomonas aeruginosa-targeted siderophore-conjugated monocarbams by application of a semi-mechanistic pharmacokinetic/pharmacodynamic model.
J Med Chem
; 58(5): 2195-205, 2015 Mar 12.
Artigo
Inglês
| MEDLINE | ID: mdl-25658376
14.
Synthesis, Structure, and SAR of Tetrahydropyran-Based LpxC Inhibitors.
ACS Med Chem Lett
; 5(11): 1213-8, 2014 Nov 13.
Artigo
Inglês
| MEDLINE | ID: mdl-25408833
15.
Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia.
Cell Rep
; 24(9): 2520, 2018 Aug 28.
Artigo
Inglês
| MEDLINE | ID: mdl-30157442