Detalhe da pesquisa
1.
CRISPR-Cas9 In Vivo Gene Editing of KLKB1 for Hereditary Angioedema.
N Engl J Med
; 390(5): 432-441, 2024 Feb 01.
Artigo
em Inglês
| 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.
Artigo
em Inglês
| MEDLINE | ID: mdl-34215024
3.
A convolutional neural network-based, quantitative complete blood count scattergram-mapping framework promptly screens acute promyelocytic leukemia with high sensitivity.
Cancer
; 129(19): 2986-2998, 2023 10 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-37254628
4.
The Nonclinical Disposition and Pharmacokinetic/Pharmacodynamic Properties of N-Acetylgalactosamine-Conjugated Small Interfering RNA Are Highly Predictable and Build Confidence in Translation to Human.
Drug Metab Dispos
; 50(6): 781-797, 2022 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-34154993
5.
Range-dependent geoacoustic inversion using equivalent environmental model in the presence of doppler effect.
J Acoust Soc Am
; 151(4): 2613, 2022 Apr.
Artigo
em Inglês
| MEDLINE | ID: mdl-35461480
6.
Nonclinical Pharmacokinetics and Absorption, Distribution, Metabolism, and Excretion of Givosiran, the First Approved N-Acetylgalactosamine-Conjugated RNA Interference Therapeutic.
Drug Metab Dispos
; 49(7): 572-580, 2021 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-33941543
7.
Safety evaluation of 2'-deoxy-2'-fluoro nucleotides in GalNAc-siRNA conjugates.
Nucleic Acids Res
; 47(7): 3306-3320, 2019 04 23.
Artigo
em Inglês
| MEDLINE | ID: mdl-30820542
8.
Identification of RNA Expression Profiles in Thyroid Cancer to Construct a Competing Endogenous RNA (ceRNA) Network of mRNAs, Long Noncoding RNAs (lncRNAs), and microRNAs (miRNAs).
Med Sci Monit
; 25: 1140-1154, 2019 Feb 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-30745559
9.
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.
Artigo
em Inglês
| MEDLINE | ID: mdl-38389403
10.
Clinical Pharmacology and Translational Considerations in the Development of CRISPR-Based Therapies.
Clin Pharmacol Ther
; 114(3): 591-603, 2023 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-37429825
11.
Degradation of Cyclin-Dependent Kinase 9/Cyclin T1 by Optimized Microtubule-Associated Protein 1 Light Chain 3 Beta-Recruiting Coumarin Analogs.
J Med Chem
; 66(18): 12877-12893, 2023 09 28.
Artigo
em Inglês
| MEDLINE | ID: mdl-37671907
12.
Exploration of the molecular mechanism of insulin resistance in adipose tissue of patients with type 2 diabetes mellitus through a bioinformatic analysis.
Minerva Endocrinol (Torino)
; 48(4): 440-446, 2023 Dec.
Artigo
em Inglês
| MEDLINE | ID: mdl-37534872
13.
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.
Artigo
em Inglês
| MEDLINE | ID: mdl-37526071
14.
Gather wisdom to overcome barriers: Well-designed nano-drug delivery systems for treating gliomas.
Acta Pharm Sin B
; 12(3): 1100-1125, 2022 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-35530155
15.
Tumor-derived extracellular vesicles as messengers of natural products in cancer treatment.
Theranostics
; 12(4): 1683-1714, 2022.
Artigo
em Inglês
| MEDLINE | ID: mdl-35198064
16.
Mineral medicine: from traditional drugs to multifunctional delivery systems.
Chin Med
; 17(1): 21, 2022 Feb 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-35144660
17.
Construction of a ferroptosis-related eight gene signature for predicting the prognosis and immune infiltration of thyroid cancer.
Front Endocrinol (Lausanne)
; 13: 997873, 2022.
Artigo
em Inglês
| MEDLINE | ID: mdl-36407322
18.
Assessment of the Immunogenicity Potential for Oligonucleotide-Based Drugs.
Nucleic Acid Ther
; 32(5): 369-377, 2022 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-36178478
19.
2021 White Paper on Recent Issues in Bioanalysis: TAb/NAb, Viral Vector CDx, Shedding Assays; CRISPR/Cas9 & CAR-T Immunogenicity; PCR & Vaccine Assay Performance; ADA Assay Comparability & Cut Point Appropriateness (Part 3 - Recommendations on Gene Therapy, Cell Therapy, Vaccine Assays; Immunogenicity of Biotherapeutics and Novel Modalities; Integrated Summary of Immunogenicity Harmonization).
Bioanalysis
; 14(11): 737-793, 2022 Jun.
Artigo
em Inglês
| MEDLINE | ID: mdl-35578991
20.
Normal reference ranges for urinary δ-aminolevulinic acid and porphobilinogen levels.
JIMD Rep
; 57(1): 85-93, 2021 Jan.
Artigo
em Inglês
| MEDLINE | ID: mdl-33473344