Curing Genetic Diseases through Genome Reprogramming

Curing Genetic Diseases through Genome Reprogramming

1st Edition - June 23, 2021

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  • Editor: Gianluca Petris
  • Hardcover ISBN: 9780323853019
  • eBook ISBN: 9780323853026

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Curing Genetic Diseases through Genome Reprogramming, Volume 182 captures an historic moment in the field of gene therapy—the dawn of a new age in which the dream of curing genetic diseases has become realizable. The volume presents the most clinically advanced gene therapy and genome editing approaches for the treatment of genetic diseases in specific organs, including difficult therapeutic targets, futuristic ideas of genetic interventions, and large scale human genome repair. An initial chapter addresses the complex ethical aspects involved in the very idea of modifying the human genome.

Key Features

  • Provides a comprehensive view of gene therapy and genome editing technologies, including epigenetic editing
  • Describes the state-of-the-art and future directions for the treatment of genetic diseases, also considering economical aspects
  • Presents chapters that each give a thorough review of a specific disease, target organ or visionary approach, including ethical considerations


Biologists, biotechnologists and students interested in gene therapy and genome editing, but also for medical doctors, stakeholders and policy makers who are intrigued by innovative genetic therapies

Table of Contents

  • Preface
    Gianluca Petris
    1. Making sense of heritable human genome editing: scientific and ethical considerations
    Andy Greenfiel
    2. CRISPR genome engineering for retinal diseases
    Ariel Kantor, Michelle E. McClements, Caroline F. Peddle, Lewis E. Fry, Ahmed Salman, Jasmina Cehajic-Kapetanovic, Kanmin Xue and Robert E. MacLaren
    3. Advances in gene editing strategies for epidermolysis bullosa
    Thomas Kocher and Ulrich Koller
    4. Targeted genome editing for the correction or alleviation of primary immunodeficiencies
    Christopher J. Sipe, Patricia N. Claudio Vázquez, Joseph G. Skeate, R. Scott McIvor and Branden S. Moriarity
    5. Genome editing approaches to β-hemoglobinopathies
    Mégane Brusson and Annarita Miccio
    6. Rewriting CFTR to cure Cystic Fibrosis
    Giulia Maule, Marjolein Ensinck, Mattijs Bulcaen and Marianne S. Carlon
    7. Gene editing and modulation for Duchenne muscular dystrophy
    Anthony A. Stephenson and Kevin M. Flanigan
    8. Genome editing in the human liver: progress and translational considerations
    Samantha L. Ginn, Sharntie Christina and Ian E. Alexander
    9. Genome editing in lysosomal disorders
    Luisa Natalia Pimentel-Vera, Edina Poletto, Esteban Alberto Gonzalez, Fabiano de Oliveira Poswar, Roberto Giugliani and Guilherme Baldo
    10. Genome editing in mucopolysaccharidoses and mucolipidoses
    Hallana Souza Santos, Edina Poletto, Roselena Schuh, Ursula Matte and Guilherme Baldo
    11. Gene and epigenetic editing in the treatment of primary ciliopathies
    Elisa Molinari and John A. Sayer
    12. Genome editing in stem cells for genetic neurodisorders
    Dell'Amico Claudia, Tata Alice, Pellegrino Enrica, Onorati Marco and Conti Luciano
    13. Reprogramming translation for gene therapy
    Chiara Ambrosini, Francesca Garilli and Alessandro Quattrone
    14. Synthetic genomics for curing genetic diseases
    Simona Grazioli and Gianluca Petris

Product details

  • No. of pages: 558
  • Language: English
  • Copyright: © Academic Press 2021
  • Published: June 23, 2021
  • Imprint: Academic Press
  • Hardcover ISBN: 9780323853019
  • eBook ISBN: 9780323853026

About the Serial Editor

Gianluca Petris

Gianluca Petris is currently a Marie Skłodowska-Curie European Fellow and Investigator Scientist developing synthetic genomics tools at the Medical Research Council (MRC) Laboratory of Molecular Biology, Cambridge, United Kingdom. Prior to that, as a postdoctoral fellow at the Department of Cellular, Computational and Integrative Biology (CIBIO), University of Trento, Italy, he developed and applied CRISPR technologies and delivery vehicles for the correction of genetic diseases. Dr. Petris published several papers in the field of CRISPR-therapeutics and CRISPR applications, filed four patent families and co-founded a company for the development of gene therapies for genetic diseases. He was awarded a MSc Degree in Medical Biotechnology working on antibody engineering and rotavirus at the University of Trieste, Italy, and a PhD in Life Sciences (Open University, United Kingdom) in recognition of his research on protein folding and protein quality control carried out at the International Centre for Genetic Engineering and Biotechnology (ICGEB), Trieste, Italy.

Affiliations and Expertise

Marie Sklodowska-Curie European Fellow, Protein & Nucleic Acid Chemistry Division, The MRC Laboratory of Molecular Biology, Cambridge, UK

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