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Antisense RNA Design, Delivery, and Analysis / / edited by Virginia Arechavala-Gomeza, Alejandro Garanto
| Antisense RNA Design, Delivery, and Analysis / / edited by Virginia Arechavala-Gomeza, Alejandro Garanto |
| Autore | Arechavala-Gomeza Virginia |
| Edizione | [1st ed. 2022.] |
| Pubbl/distr/stampa | New York, : Springer Nature, 2022 |
| Descrizione fisica | 1 online resource (XVII, 422 p. 69 illus., 59 illus. in color.) |
| Disciplina | 615.5 |
| Collana | Methods in Molecular Biology |
| Soggetto topico |
Therapeutics
Biomaterials Nucleic acids Nucleic Acid |
| ISBN |
9781071620106
107162010X |
| Formato | Materiale a stampa  |
| Livello bibliografico | Monografia |
| Lingua di pubblicazione | eng |
| Nota di contenuto | 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. |
| Record Nr. | UNINA-9910552988503321 |
Arechavala-Gomeza Virginia
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| New York, : Springer Nature, 2022 | ||
| Materiale a stampa | ||
| Lo trovi qui: Univ. Federico II | ||
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Molecular Therapies for Inherited Retinal Diseases
| Molecular Therapies for Inherited Retinal Diseases |
| Autore | Collin Rob W.J |
| Pubbl/distr/stampa | Basel, Switzerland, : MDPI - Multidisciplinary Digital Publishing Institute, 2020 |
| Descrizione fisica | 1 online resource (262 p.) |
| Soggetto topico |
Biology, life sciences
Research & information: general |
| Soggetto non controllato |
AAV
ABCA4 adeno-associated viral adeno-associated virus (AAV) allele-specific knockdown antisense oligonucleotides AON-mediated exon skipping apical polarity apoptosis ARPE-19 cells autophagy autosomal dominant bipolar cells CEP290 chaperones chaperonins choroideremia Cilia elongation clathrin-coated vesicles clinical trials clustered regularly interspaced short palindromic repeats (CRISPR) compound therapies cones crumbs complex cyclic GMP DNA therapies DNA-wrapped gold nanoparticles drug delivery systems dual AAV endosomal trafficking Enhanced S-Cone Syndrome (ESCS) fetal retina Flanders founder c.4723A > G56R gapmer antisense oligonucleotides gene augmentation gene therapy gold nanoparticles heat shock response homology-directed repair (HDR) induced pluripotent stem cell (iPSC) inherited retinal disease inherited retinal diseases iPSC-derived photoreceptor precursor cells IRD Leber congenital amaurosis Leber congenital amaurosis and allied retinal ciliopathies microRNA Müller glia n/a necrosis nonprofit NR2E3 PAR complex patient registry photoreceptors protein degradation protein folding protein trafficking putative dominant negative effect REP1 retina retinal degeneration retinal inherited disorders retinal organoids retinal pigment epithelium retinitis pigmentosa Retinitis Pigmentosa (RP) Retinitis Pigmentosa GTPase Regulator retinogenesis RNA therapies RNA therapy rods RPE65 splicing modulation spontaneous nonsense correction Stargardt disease T nonsense mutation therapy translational translational medicine treatment unfolded protein response Usher syndrome |
| Formato | Materiale a stampa |
| Livello bibliografico | Monografia |
| Lingua di pubblicazione | eng |
| Record Nr. | UNINA-9910674051103321 |
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Collin Rob W.J
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| Basel, Switzerland, : MDPI - Multidisciplinary Digital Publishing Institute, 2020 | ||
| Materiale a stampa | ||
| Lo trovi qui: Univ. Federico II | ||
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