Safety and Efficacy of Gene-Based Therapeutics for Inherited Disorders

  • Nicola Brunetti-Pierri

Table of contents

  1. Front Matter
    Pages i-ix
  2. Daniela Cesana, Monica Volpin, Yasmin Natalia Serina Secanechia, Eugenio Montini
    Pages 9-35
  3. Andrew W. Harmon, Andrew P. Byrnes
    Pages 37-60
  4. Pasquale Piccolo, Nicola Brunetti-Pierri
    Pages 61-75
  5. Klaudia Kuranda, Federico Mingozzi
    Pages 77-112
  6. María José Herrero, Luis Sendra, Antonio Miguel, Salvador F. Aliño
    Pages 113-135
  7. Maximilian Müller, Tafadzwa Mlambo, Claudio Mussolino
    Pages 137-157
  8. Chengjung Lai, Cristina Martin-Higueras, Eduardo Salido
    Pages 159-190
  9. Michela Alessandra Denti, Giuseppina Covello
    Pages 191-217
  10. Back Matter
    Pages 219-220

About this book


In this book, leading international experts analyze state-of-the-art advances in gene transfer vectors for applications in inherited disorders and also examine the toxicity profiles of these methods. The authors discuss the strengths and weaknesses of available vectors in the clinical setting, and specifically focus on the challenges and possible solutions that researchers are testing in order to improve the safety of gene therapy for genetic diseases. This comprehensive and authoritative overview of vector development is a necessary text for researchers, toxicologists, pharmacologists, molecular biologists, physicians, and students in these fields.


Adenoviral vector Antisense oligonucleotide gene transfer immune response lentiviral vectors viral gene therapy vectors

Editors and affiliations

  • Nicola Brunetti-Pierri
    • 1
  1. 1.Department of Translational MedicineUniversity of Naples Federico IINaplesItaly

Bibliographic information