ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler Syndrome

Li Ou, Russell C. DeKelver, Michelle Rohde, Susan Tom, Robert Radeke, Susan J. St. Martin, Yolanda Santiago, Scott Sproul, Michelle J. Przybilla, Brenda L. Koniar, Kelly M. Podetz-Pedersen, Kanut Laoharawee, Renee D. Cooksley, Kathleen E. Meyer, Michael C. Holmes, R. Scott McIvor, Thomas Wechsler, Chester B. Whitley

Research output: Contribution to journalArticlepeer-review

19 Scopus citations

Abstract

In vivo genome editing following a single injection of ZFN and IDUA donor encoding AAV8 resulted in metabolic correction and neurological benefit in a murine model of MPS I. These results enable a currently open clinical trial to evaluate targeted genome engineering for MPS I in humans.

Original languageEnglish (US)
Pages (from-to)178-187
Number of pages10
JournalMolecular Therapy
Volume27
Issue number1
DOIs
StatePublished - Jan 2 2019

Bibliographical note

Funding Information:
The authors would like to thank Carolyn Gaspar, Anne-Marie Ledeboer, Melanie Butler, and Amy Manning-Bog for assistance in necropsy and figure preparation, Seventh Wave Laboratories (Maryland Heights, MO) for performing necropsy and histopathology evaluation (Mark Martinez, DVM, DACVP), and Pacific BioLabs (Hercules, CA) for performing mass-spectrometry analysis of dermatan and heparan sulfate levels (Rick Staub and Vy Tran). This work was supported by Sangamo Therapeutics .

Funding Information:
The authors would like to thank Carolyn Gaspar, Anne-Marie Ledeboer, Melanie Butler, and Amy Manning-Bog for assistance in necropsy and figure preparation, Seventh Wave Laboratories (Maryland Heights, MO) for performing necropsy and histopathology evaluation (Mark Martinez, DVM, DACVP), and Pacific BioLabs (Hercules, CA) for performing mass-spectrometry analysis of dermatan and heparan sulfate levels (Rick Staub and Vy Tran). This work was supported by Sangamo Therapeutics.

Publisher Copyright:
© 2018 The Author(s)

Keywords

  • gene editing
  • gene therapy
  • lysosomal diseases

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