Safety and Efficacy of Gene-Based Therapeutics for Inherited Disorders

1. Gene Transfer Strategies and Applications in Genetic Diseases
Nicola Brunetti-Pierri

2. Safety and Efficacy of Retroviral and Lentiviral Vectors for Gene Therapy
Daniela Cesana, Monica Volpin, Yasmin Natalia Serina Secanechia, Eugenio Montini

3. Adenovirus Vector Toxicity
Andrew W. Harmon, Andrew P. Byrnes

4. Helper-Dependent Adenoviral Vectors for Gene Therapy of Inherited Diseases
Pasquale Piccolo, Nicola Brunetti-Pierri

5. AAV Vector-Based Gene Therapy, Progress and Current Challenges
Klaudia Kuranda, Federico Mingozzi

6. Physical Methods of Gene Delivery
María José Herrero, Luis Sendra, Antonio Miguel, Salvador F. Aliño

7. Designer Effectors for Editing and Regulating Complex Genomes
Maximilian Müller, Tafadzwa Mlambo, Claudio Mussolino

8. siRNA Therapeutics to Treat Liver Disorders
Chengjung Lai, Cristina Martin-Higueras, Eduardo Salido

9. Oligonucleotide Therapy
Michela Alessandra Denti, Giuseppina Covello

Keywords: Biomedicine, Pharmacology/Toxicology, Gene Therapy, Molecular Medicine