Antisense RNA Design, Delivery, and Analysis
This open access volume gathers a variety of models, delivery systems, and approaches that can be used to assess RNA technology for exploiting antisense as a therapeutic intervention. Beginning with a section on the design of antisense technology and their delivery, the book continues by covering mo...
| Otros Autores: | , , |
|---|---|
| Formato: | Libro electrónico |
| Idioma: | Inglés |
| Publicado: |
New York
Springer Nature
2022
New York, NY : 2022. |
| Edición: | 1st ed. 2022. |
| Colección: | Methods in Molecular Biology,
2434 |
| Materias: | |
| Ver en Biblioteca Universitat Ramon Llull: | https://discovery.url.edu/permalink/34CSUC_URL/1im36ta/alma991009654931206719 |
Tabla de Contenidos:
- Introduction and History of the Chemistry of Nucleic Acids Therapeutics
- Antisense RNA Therapeutics: A Brief Overview
- Design of Bifunctional Antisense Oligonucleotides for Exon Inclusion
- Design and Delivery of SINEUP: A New Modular Tool to Increase Protein Translation
- How to Design U1 snRNA Molecules for Splicing Rescue
- Conjugation of Nucleic Acids and Drugs to Gold Nanoparticles
- Determination of Optimum Ratio of Cationic Polymers and Small Interfering RNA with Agarose Gel Retardation Assay
- Generation of Protein-Phosphorodiamidate Morpholino Oligomer Conjugates for Efficient Cellular Delivery via Anthrax Protective Antigen
- Development and Use of Cellular Systems to Assess and Correct Splicing Defects
- Modeling Splicing Variants Amenable to Antisense Therapy by Use of CRISPR-Cas9-Based Gene Editing in HepG2 Cells
- In Vitro Models for the Evaluation of Antisense Oligonucleotides in Skin
- In Vitro Delivery of PMOs in Myoblasts by Electroporation
- Rapid Determination of MBNL1 Protein Levels by Quantitative Dot Blot for Evaluation of Antisense Oligonucleotides in Myotonic Dystrophy Myoblasts
- Evaluation of Exon Skipping and Dystrophin Restoration in In Vitro Models of Duchenne Muscular Dystrophy
- Generation of Human iPSC-Derived Myotubes to Investigate RNA-Based Therapies In Vitro
- Eye on a Dish Models to Evaluate Splicing Modulation
- Establishment of In Vitro Brain Models for AON Delivery
- Considerations for Generating Humanized Mouse Models to Test Efficacy of Antisense Oligonucleotides
- Generation of Humanized Zebrafish Models for the In Vivo Assessment of Antisense Oligonucleotide-Based Splice Modulation Therapies
- Use of Small Animal Models for Duchenne and Parameters to Assess Efficiency upon Antisense Treatment
- In Vivo Models for the Evaluation of Antisense Oligonucleotides in Skin
- Delivery of Antisense Oligonucleotides to the Mouse Retina
- Delivery of Antisense Oligonucleotides to the Mouse Brain by Intracerebroventricular Injections
- Intrathecal Delivery of Therapeutic Oligonucleotides for Potent Modulation of Gene Expression in the Central Nervous System
- Preclinical Safety Assessment of Therapeutic Oligonucleotides
- Preclinical Evaluation of the Renal Toxicity of Oligonucleotide Therapeutics in Mice
- Protocol for Isolation and Culture of Mouse Hepatocytes (HCs), Kupffer Cells (KCs), and Liver Sinusoidal Endothelial Cells (LSECs) in Analyses of Hepatic Drug Distribution
- Patent Considerations When Embarking on New Antisense Drug Programs.
