Genome Editing

The Next Step in Gene Therapy

  • Toni Cathomen
  • Matthew Hirsch
  • Matthew Porteus

Part of the Advances in Experimental Medicine and Biology book series (AEMB)

Also part of the American Society of Gene & Cell Therapy book sub series

Table of contents

  1. Front Matter
    Pages i-xvi
  2. Maria Jasin
    Pages 1-14
  3. Alexandre Juillerat, Philippe Duchateau, Toni Cathomen, Claudio Mussolino
    Pages 29-50
  4. David G. Ousterout, Charles A. Gersbach
    Pages 51-79
  5. Michele P. Calos
    Pages 81-91
  6. Patrick Ruff, Francesca Storici
    Pages 111-124
  7. Angela M. Mitchell, Rachel Moser, Richard Jude Samulski, Matthew Louis Hirsch
    Pages 125-137
  8. John H. Wilson, Christopher Moye, David Mittelman
    Pages 139-159
  9. George Nicholas Llewellyn, Colin M. Exline, Nathalia Holt, Paula M. Cannon
    Pages 161-186
  10. Eli J. Fine, Thomas James Cradick, Gang Bao
    Pages 187-222
  11. Back Matter
    Pages 259-263

About this book


This comprehensive volume explores human genetic engineering its pre-clinical and clinical applications, current developments, and as treatment for hereditary diseases. It presents and evaluates the most recent advances in the understanding of mammalian host DNA repair mechanisms, such as double-strand break induced gene targeting and mutagenesis, the development of zinc-finger nucleases, genome editing for neuromuscular diseases, phase integrases, triplex forming oligonucleotides and peptide nucleic acids, aptamer-guided gene targeting, AAV gene editing via DSB repair, engineered nucleases and trinucleotide repeat diseases, and creation of HIV-resistant cells. The expertly authored chapters contextualize current developments within the history of genome editing while also discussing the current and potential safety concerns of this rapidly growing field. Genome Editing: The Next Step in Gene Therapy, the latest volume in the American Society of Gene and Cell Therapy series, deftly illuminates the potential of genetic engineering technology to eradicate today’s deadliest and most prolific diseases. It is ideal reading for clinicians and researchers in genetics and immunology.



DNA Repair Mechanisms gene therapy Germline Nuclease Mutagenesis

Editors and affiliations

  • Toni Cathomen
    • 1
  • Matthew Hirsch
    • 2
  • Matthew Porteus
    • 3
  1. 1.Inst for Cell & Gene TherapyUniversitaetsklinikum FreiburgFreiburgGermany
  2. 2.University of North CarolinaChapel HillUSA
  3. 3.Stanford Medical SchoolPalo AltoUSA

Bibliographic information

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