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1.
CRISPR-Cas9 In Vivo Gene Editing of KLKB1 for Hereditary Angioedema.
N Engl J Med
; 390(5): 432-441, 2024 Feb 01.
Artículo
en Inglés
| MEDLINE | ID: mdl-38294975
2.
CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis.
N Engl J Med
; 385(6): 493-502, 2021 08 05.
Artículo
en Inglés
| MEDLINE | ID: mdl-34215024
3.
siRNA-mediated knockdown of P450 oxidoreductase in rats: a tool to reduce metabolism by CYPs and increase exposure of high clearance compounds.
Pharm Res
; 31(12): 3445-60, 2014 Dec.
Artículo
en Inglés
| MEDLINE | ID: mdl-24980206
4.
2023 White Paper on Recent Issues in Bioanalysis: ISR for ADA Assays, the Rise of dPCR vs qPCR, International Reference Standards for Vaccine Assays, Anti-AAV TAb Post-Dose Assessment, NanoString Validation, ELISpot as Gold Standard (Part 3 - Recommendations on Gene Therapy, Cell Therapy, Vaccines Immunogenicity & Technologies; Biotherapeutics Immunogenicity & Risk Assessment; ADA/NAb Assay/Reporting Harmonization).
Bioanalysis
; 16(7): 77-119, 2024 Apr.
Artículo
en Inglés
| MEDLINE | ID: mdl-38389403
5.
2022 White Paper on Recent Issues in Bioanalysis: FDA Draft Guidance on Immunogenicity Information in Prescription Drug Labeling, LNP & Viral Vectors Therapeutics/Vaccines Immunogenicity, Prolongation Effect, ADA Affinity, Risk-based Approaches, NGS, qPCR, ddPCR Assays (Part 3 - Recommendations on Gene Therapy, Cell Therapy, Vaccines Immunogenicity & Technologies; Immunogenicity & Risk Assessment of Biotherapeutics and Novel Modalities; NAb Assays Integrated Approach).
Bioanalysis
; 15(14): 773-814, 2023 Jul.
Artículo
en Inglés
| MEDLINE | ID: mdl-37526071
6.
Quantitative evaluation of siRNA delivery in vivo.
RNA
; 16(12): 2553-63, 2010 Dec.
Artículo
en Inglés
| MEDLINE | ID: mdl-20940339
7.
Evaluation of efficacy, biodistribution, and inflammation for a potent siRNA nanoparticle: effect of dexamethasone co-treatment.
Mol Ther
; 18(1): 171-80, 2010 Jan.
Artículo
en Inglés
| MEDLINE | ID: mdl-19738601
8.
Intratracheal Administration of siRNA Triggers mRNA Silencing in the Lung to Modulate T Cell Immune Response and Lung Inflammation.
Mol Ther Nucleic Acids
; 16: 194-205, 2019 Jun 07.
Artículo
en Inglés
| MEDLINE | ID: mdl-30901578
9.
Examination of real-time polymerase chain reaction methods for the detection and quantification of modified siRNA.
Anal Biochem
; 379(1): 96-104, 2008 Aug 01.
Artículo
en Inglés
| MEDLINE | ID: mdl-18501185
10.
A Single Administration of CRISPR/Cas9 Lipid Nanoparticles Achieves Robust and Persistent In Vivo Genome Editing.
Cell Rep
; 22(9): 2227-2235, 2018 02 27.
Artículo
en Inglés
| MEDLINE | ID: mdl-29490262
11.
Effect of biological matrix and sample preparation on qPCR quantitation of siRNA drugs in animal tissues.
J Pharmacol Toxicol Methods
; 63(2): 168-73, 2011.
Artículo
en Inglés
| MEDLINE | ID: mdl-20884364
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