Table of contents

Part I: Overview

 

“Introduction and history of nucleic acid therapeutics” Michael J Gait and Sudhir Agrawal. March 2022 Antisense RNA Design, Delivery, and Analysis, Methods in Molecular Biology, Springer.

 

“Antisense RNA therapeutics: A brief overview”. Virginia Arechavala and Alejandro Garanto. March 2022. Antisense RNA Design, Delivery, and Analysis, Methods in Molecular Biology, Springer.

Part II: Design and Formulation of Antisense Technology

 

“Design of Bifunctional Antisense Oligonucleotides for Exon Inclusion”. Haiyan Zhou. March 2022. Antisense RNA Design, Delivery, and Analysis, Methods in Molecular Biology, Springer.

 

Design and delivery of SINEUP: a new modular tool to increase protein translation”. Michele Arnoldi, Giulia Zarantonello, Stefano Espinoza, Stefano Gustincich, Francesca Di Leva and Marta Biagioli. March 2022. Antisense RNA Design, Delivery, and Analysis, Methods in Molecular Biology, Springer.

 

How to design U1 snRNA molecules for splicing rescue”. Liliana Matos, Juliana I. Santos, Mª Francisca Coutinho, Sandra Alves. March 2022. Antisense RNA Design, Delivery, and Analysis, Methods in Molecular Biology, Springer.

 

Conjugation of nucleic acids and drugs to gold nanoparticles”. Paula Milán-Rois, Ciro Rodriguez-Diaz, Milagros Castellanos, Álvaro Somoza. March 2022. Antisense RNA Design, Delivery, and Analysis, Methods in Molecular Biology, Springer.

 

Determining the Number of Cationic Polymers and Small Interfering RNA (siRNA) Ratio with Agarose Gel Retardation Assay”. Omer Aydin, Dilek Kanarya, Ummugulsum Yilmaz, Cansu Umran Tunc. March 2022. Antisense RNA Design, Delivery, and Analysis, Methods in Molecular Biology, Springer.

 

Generation of Protein-Phosphorodiamidate Morpholino Oligomer Conjugates for Efficient Cellular Delivery via Anthrax Protective Antigen”. Valentina Palacio-Castañeda, Roland Brock, Wouter P.R. Verdurmen. March 2022. Antisense RNA Design, Delivery, and Analysis, Methods in Molecular Biology, Springer.

Part III: Model Systems

In vitro models

 

Development and Use of Cellular Systems to Assess and Correct Splicing Defects”. Nuria Suárez-Herrera, Tomasz Z. Tomkiewicz, Alejandro Garanto, Rob W.J. Collin. March 2022. Antisense RNA Design, Delivery, and Analysis, Methods in Molecular Biology, Springer.

 

Modelling splicing variants amenable to antisense therapy by use of CRISPR-Cas9 based gene editing in HepG2 cells”. Arístides López-Márquez, Ainhoa Martínez-Pizarro, Belén Pérez, Eva Richard, Lourdes R Desviat. March 2022. Antisense RNA Design, Delivery, and Analysis, Methods in Molecular Biology, Springer.

 

In vitro models for the evaluation of antisense oligonucleotides in skin”. Jeroen Bremer, Peter C. van den Akker. March 2022. Antisense RNA Design, Delivery, and Analysis, Methods in Molecular Biology, Springer.

 

In vitro delivery of PMOs in myoblasts by electroporation”. Remko Goossens, Annemieke Aartsma-Rus. March 2022. Antisense RNA Design, Delivery, and Analysis, Methods in Molecular Biology, Springer.

 

Rapid determination of MBNL1 protein levels by quantitative dot blot for evaluation of antisense oligonucleotides in myotonic dystrophy myoblasts”. Nerea Moreno-Cervera, Irene Gonzalez-Martinez, Rubén Artero, Estefanía Cerro-Herreros. March 2022. Antisense RNA Design, Delivery, and Analysis, Methods in Molecular Biology, Springer.

 

Evaluation of exon skipping and dystrophin restoration in in vitro models of Duchenne muscular dystrophy”. Andrea López-Martínez, Patricia Soblechero-Martín, Virginia Arechavala-Gomeza. March 2022. Antisense RNA Design, Delivery, and Analysis, Methods in Molecular Biology, Springer.

 

Generation of human iPSC-Derived Myotubes to Investigate RNA-Based Therapies In Vitro”. Pablo Herrero-Hernandez, Atze J. Bergsma, WWM Pim Pijnappel. March 2022. Antisense RNA Design, Delivery, and Analysis, Methods in Molecular Biology, Springer.

 

Eye on a dish models to evaluate splicing modulation”. Kwan-Leong Hau, Amelia Lane, Rosellina Guarascio, Michael E. Cheetham. March 2022. Antisense RNA Design, Delivery, and Analysis, Methods in Molecular Biology, Springer.

 

Establishment of in vitro brain models for AON delivery”. Elena Daoutsali, Ronald A. M. Buijsen. March 2022. Antisense RNA Design, Delivery, and Analysis, Methods in Molecular Biology, Springer.

In vivo models

 

Considerations for generating humanized mouse models to test efficacy of antisense oligonucleotides”. Irene Vázquez-Domínguez and Alejandro Garanto. March 2022. Antisense RNA Design, Delivery, and Analysis, Methods in Molecular Biology, Springer.

 

Generation of humanized zebrafish models for the in vivo assessment of antisense oligonucleotide-based splice modulation therapies”. Renske Schellens, Erik de Vrieze, Ralph Slijkerman, Hannie Kremer, Erwin van Wijk. March 2022. Antisense RNA Design, Delivery, and Analysis, Methods in Molecular Biology, Springer.

 

“Use Of Small Animal Models For Duchenne And Parameters To Assess Efficiency Upon Antisense Treatment”. Ngoc Lu-Nguyen, Alberto Malerba and Linda Popplewell. March 2022. Antisense RNA Design, Delivery, and Analysis, Methods in Molecular Biology, Springer.

 

In vivo models for the evaluation of antisense oligonucleotides in skin”. Jeroen Bremer, Peter C. van den Akker. March 2022. Antisense RNA Design, Delivery, and Analysis, Methods in Molecular Biology, Springer.

 

Delivery of antisense oligonucleotides to the mouse retina”. Alex Garanto. March 2022. Antisense RNA Design, Delivery, and Analysis, Methods in Molecular Biology, Springer.

 

Delivery of antisense oligonucleotides to the mouse brain by intracerebroventricular injections”. Tom Metz, Elsa C. Kuijper, Willeke M. C. van Roon-Mom. March 2022. Antisense RNA Design, Delivery, and Analysis, Methods in Molecular Biology, Springer.

 

Intrathecal Delivery of Therapeutic Oligonucleotides for Potent Modulation of Gene Expression in the Central Nervous System”. Zachary Kennedy, James W. Gilbert, Bruno M.D.C. Godinho. March 2022. Antisense RNA Design, Delivery, and Analysis, Methods in Molecular Biology, Springer.

Part IV: Safety and Toxicology

 

Pre-clinical evaluation of oligonucleotide therapeutics mediated toxicity“. Patrik Andersson. March 2022. Antisense RNA Design, Delivery, and Analysis, Methods in Molecular Biology, Springer.

 

Preclinical evaluation of the renal toxicity of oligonucleotide therapeutics in mice”. Lucía Echevarría and Aurelie Goyenvalle. March 2022. Antisense RNA Design, Delivery, and Analysis, Methods in Molecular Biology, Springer.

 

Protocol for isolation and culture of mouse hepatocytes (HCs), Kupffer cells (KCs) and liver sinusoidal endothelial cells (LSECs) in analyses of hepatic drug distribution”. Kjetil Elvevold, Ingelin Kyrrestad, Karen Kristine Sørensen, Bård Smedsrød. March 2022. Antisense RNA Design, Delivery, and Analysis, Methods in Molecular Biology, Springer.

Part V: Intellectual Property

 

Patent considerations when embarking on new antisense drug programs”. Laurence Gainey. March 2022. Antisense RNA Design, Delivery, and Analysis, Methods in Molecular Biology, Springer.