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 journal › Article

4 Citations (Scopus)

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

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Mucopolysaccharidosis I
Clinical Trials
Genome
Injections
Gene Editing

Keywords

  • gene editing
  • gene therapy
  • lysosomal diseases

Cite this

ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler Syndrome. / Ou, Li; DeKelver, Russell C.; Rohde, Michelle; Tom, Susan; Radeke, Robert; St. Martin, Susan J.; Santiago, Yolanda; Sproul, Scott; Przybilla, Michelle J.; Koniar, Brenda L.; Podetz-Pedersen, Kelly M.; Laoharawee, Kanut; Cooksley, Renee D.; Meyer, Kathleen E.; Holmes, Michael C.; McIvor, R. Scott; Wechsler, Thomas; Whitley, Chester B.

In: Molecular Therapy, Vol. 27, No. 1, 02.01.2019, p. 178-187.

Research output: Contribution to journal › Article

Ou, L, DeKelver, RC, Rohde, M, Tom, S, Radeke, R, St. Martin, SJ, Santiago, Y, Sproul, S, Przybilla, MJ, Koniar, BL, Podetz-Pedersen, KM, Laoharawee, K, Cooksley, RD, Meyer, KE, Holmes, MC, McIvor, RS, Wechsler, T & Whitley, CB 2019, 'ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler Syndrome', Molecular Therapy, vol. 27, no. 1, pp. 178-187. https://doi.org/10.1016/j.ymthe.2018.10.018
Ou L, DeKelver RC, Rohde M, Tom S, Radeke R, St. Martin SJ et al. ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler Syndrome. Molecular Therapy. 2019 Jan 2;27(1):178-187. https://doi.org/10.1016/j.ymthe.2018.10.018
Ou, Li ; DeKelver, Russell C. ; Rohde, Michelle ; Tom, Susan ; Radeke, Robert ; St. Martin, Susan J. ; Santiago, Yolanda ; Sproul, Scott ; Przybilla, Michelle J. ; Koniar, Brenda L. ; Podetz-Pedersen, Kelly M. ; Laoharawee, Kanut ; Cooksley, Renee D. ; Meyer, Kathleen E. ; Holmes, Michael C. ; McIvor, R. Scott ; Wechsler, Thomas ; Whitley, Chester B. / ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler Syndrome. In: Molecular Therapy. 2019 ; Vol. 27, No. 1. pp. 178-187.
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