Cargando…

Experimental Model Systems Used in the Preclinical Development of Nucleic Acid Therapeutics

Preclinical evaluation of nucleic acid therapeutics (NATs) in relevant experimental model systems is essential for NAT drug development. As part of COST Action “DARTER” (Delivery of Antisense RNA ThERapeutics), a network of researchers in the field of RNA therapeutics, we have conducted a survey on...

Descripción completa

Detalles Bibliográficos
Autores principales:	Zhou, Haiyan, Arechavala-Gomeza, Virginia, Garanto, Alejandro
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Mary Ann Liebert, Inc., publishers 2023
Materias:	Issues in Development
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10457615/ https://www.ncbi.nlm.nih.gov/pubmed/37145922 http://dx.doi.org/10.1089/nat.2023.0001

Ejemplares similares

Considerations in the Preclinical Assessment of the Safety of Antisense Oligonucleotides
por: Goyenvalle, Aurélie, et al.
Publicado: (2023)

OSWG Recommended Approaches to the Nonclinical Pharmacokinetic (ADME) Characterization of Therapeutic Oligonucleotides
por: Berman, Cindy L., et al.
Publicado: (2023)

Special Issue “Genetic Advances in Neuromuscular Disorders: From Gene Identification to Gene Therapy”
por: Arechavala-Gomeza, Virginia, et al.
Publicado: (2021)

Considerations of the Japanese Research Working Group for the ICH S6 & Related Issues Regarding Nonclinical Safety Assessments of Oligonucleotide Therapeutics: Comparison with Those of Biopharmaceuticals
por: Hirabayashi, Yoko, et al.
Publicado: (2021)

Researcher's Perceptions on Publishing “Negative” Results and Open Access
por: Echevarría, Lucía, et al.
Publicado: (2021)

Splicing modulation therapy in the treatment of genetic diseases
por: Arechavala-Gomeza, Virginia, et al.
Publicado: (2014)

A preclinical model of glioblastoma
Publicado: (2015)

Evaluation and Reduction of CRISPR Off-Target Cleavage Events
por: Vakulskas, Christopher A., et al.
Publicado: (2019)

On Mammalian Totipotency: What Is the Molecular Underpinning for the Totipotency of Zygote?
por: Hu, Kejin
Publicado: (2019)

FDA Approves Eteplirsen for Duchenne Muscular Dystrophy: The Next Chapter in the Eteplirsen Saga
por: Aartsma-Rus, Annemieke, et al.
Publicado: (2017)

Molecular Mechanisms of Antisense Oligonucleotides
por: Crooke, Stanley T.
Publicado: (2017)

Guidelines for Experiments Using Antisense Oligonucleotides and Double-Stranded RNAs
por: Gagnon, Keith T., et al.
Publicado: (2019)

Technical Considerations for Use of Oligonucleotide Solution API
por: Muslehiddinoglu, Jale, et al.
Publicado: (2020)

Perspectives on the Designation of Oligonucleotide Starting Materials
por: Kiesman, William F., et al.
Publicado: (2021)

Preparing n-of-1 Antisense Oligonucleotide Treatments for Rare Neurological Diseases in Europe: Genetic, Regulatory, and Ethical Perspectives
por: Synofzik, Matthis, et al.
Publicado: (2022)

Considerations for the Terminal Sterilization of Oligonucleotide Drug Products
por: DeCollibus, Daniel Paul, et al.
Publicado: (2023)

Preclinical and Clinical Advances of GalNAc-Decorated Nucleic Acid Therapeutics
por: Huang, Yuanyu
Publicado: (2017)

CATANA: an online modelling environment for proteins and nucleic acid nanostructures
por: Kuťák, David, et al.
Publicado: (2022)

Development of a group-based diabetes education model for migrants with type 2 diabetes, living in Sweden
por: Hadziabdic, Emina, et al.
Publicado: (2020)

A promising preclinical test for DMD in dog models
Publicado: (2014)

Development of an antimicrobial stewardship implementation model involving collaboration between general practitioners and pharmacists: GPPAS study in Australian primary care
por: Saha, Sajal K., et al.
Publicado: (2021)

Optimising preclinical mouse models: lesson from Batten disease
Publicado: (2015)

A five-step approach for developing and implementing a Rural Primary Health Care Model for Dementia: a community–academic partnership
por: Morgan, Debra, et al.
Publicado: (2019)

The Biomarker Potential of miRNAs in Myotonic Dystrophy Type I
por: Koehorst, Emma, et al.
Publicado: (2020)

Editorial: Nucleic Acids Research and Nucleic Acid Therapeutics
por: Stoddard, Barry L, et al.
Publicado: (2018)

PMON30 Low-dose Infigratinib, an Oral Selective Fibroblast Growth Factor Receptor Tyrosine Kinase Inhibitor, Demonstrates Activity in a Preclinical Model of Hypochondroplasia
por: Dambkowski, Carl, et al.
Publicado: (2022)

Meta-analysis on the Market Development of Electrified Vehicles
por: Kampker, Achim, et al.
Publicado: (2021)

Families First: the development of a new mentalization-based group intervention for first-time parents to promote child development and family health
por: Kalland, Mirjam, et al.
Publicado: (2015)

Development of Molecularly Targeted Therapies to Treat Pediatric Malignancies
por: Stewart, Clinton F., et al.
Publicado: (2017)

Developing modern primary care nursing in North Macedonia
por: Bryar, Rosamund, et al.
Publicado: (2023)

Digital technologies for cognitive assessment to accelerate drug development in Alzheimer's disease
por: Leurent, C, et al.
Publicado: (2015)

ProNAB: database for binding affinities of protein–nucleic acid complexes and their mutants
por: Harini, Kannan, et al.
Publicado: (2021)

DSSR-enhanced visualization of nucleic acid structures in Jmol
por: Hanson, Robert M., et al.
Publicado: (2017)

RNAcanvas: interactive drawing and exploration of nucleic acid structures
por: Johnson, Philip Z, et al.
Publicado: (2023)

Bedside to Bench: Integrating Quantitative Clinical Pharmacology and Reverse Translation to Optimize Drug Development
por: Gibbs, John P., et al.
Publicado: (2017)

Hospital avoidance: an integrated community system to reduce acute hospital demand
por: McGeoch, Graham, et al.
Publicado: (2019)

Does characterising patterns of multimorbidity in stroke matter for developing collaborative care approaches in primary care?
por: Aquino, Maria Raisa Jessica (Ryc) V, et al.
Publicado: (2019)

Short Assessment of Health Literacy (SAHL) in Portugal: development and validation of a self-administered tool
por: Pires, Carla, et al.
Publicado: (2018)

Amigos de Fibro (Fibro Friends): development of an educational program for the health promotion of fibromyalgia patients
por: Antunes, Mateus Dias, et al.
Publicado: (2022)

Strengthening health system building blocks: configuring post-COVID-19 scenario in Pakistan
por: Shaikh, Babar Tasneem
Publicado: (2021)

Cannot write session to /tmp/vufind_sessions/sess_m9oan7is86q68gq24pp3bjge1p