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1.
Giroctocogene fitelparvovec gene therapy for severe hemophilia A: 104-week analysis of the phase 1/2 Alta study.
Blood
; 143(9): 796-806, 2024 Feb 29.
Artículo
en Inglés
| MEDLINE | ID: mdl-37871576
2.
Clinical enrollment assay to detect preexisting neutralizing antibodies to AAV6 with demonstrated transgene expression in gene therapy trials.
Gene Ther
; 30(1-2): 150-159, 2023 02.
Artículo
en Inglés
| MEDLINE | ID: mdl-35778500
3.
First-in-human in vivo genome editing via AAV-zinc-finger nucleases for mucopolysaccharidosis I/II and hemophilia B.
Mol Ther
; 30(12): 3587-3600, 2022 12 07.
Artículo
en Inglés
| MEDLINE | ID: mdl-36299240
4.
A Novel Allosteric Insulin Receptor-Activating Antibody Reduces Hyperglycemia without Hypoglycemia in Diabetic Cynomolgus Monkeys.
J Pharmacol Exp Ther
; 356(2): 466-73, 2016 Feb.
Artículo
en Inglés
| MEDLINE | ID: mdl-26578267
5.
A sensitive AAV transduction inhibition assay assists evaluation of critical factors for detection and concordance of pre-existing antibodies.
Mol Ther Methods Clin Dev
; 31: 101126, 2023 Dec 14.
Artículo
en Inglés
| MEDLINE | ID: mdl-37920239
6.
2022 White Paper on Recent Issues in Bioanalysis: Enzyme Assay Validation, BAV for Primary End Points, Vaccine Functional Assays, Cytometry in Tissue, LBA in Rare Matrices, Complex NAb Assays, Spectral Cytometry, Endogenous Analytes, Extracellular Vesicles Part 2 - Recommendations on Biomarkers/CDx, Flow Cytometry, Ligand-Binding Assays Development & Validation; Emerging Technologies; Critical Reagents Deep Characterization.
Bioanalysis
; 15(15): 861-903, 2023 Aug.
Artículo
en Inglés
| MEDLINE | ID: mdl-37584363
7.
Anti-drug Antibody Sample Testing and Reporting Harmonization.
AAPS J
; 24(6): 113, 2022 10 28.
Artículo
en Inglés
| MEDLINE | ID: mdl-36307592
8.
AAV2/6 Gene Therapy in a Murine Model of Fabry Disease Results in Supraphysiological Enzyme Activity and Effective Substrate Reduction.
Mol Ther Methods Clin Dev
; 18: 607-619, 2020 Sep 11.
Artículo
en Inglés
| MEDLINE | ID: mdl-32775495
9.
Multiplex mRNA assay using electrophoretic tags for high-throughput gene expression analysis.
Nucleic Acids Res
; 32(16): e126, 2004 Sep 08.
Artículo
en Inglés
| MEDLINE | ID: mdl-15356286
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