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; Michael Przybilla; Brenda L. Koniar; Kelly M. Podetz-Pedersen; Kanut Laoharawee; Renee D. Cooksley; Kathleen E. Meyer; Michael C. Holmes; R S Mc Ivor; Thomas Wechsler; Chester B Whitley

doi:10.1016/j.ymthe.2018.10.018

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, Michael Przybilla, Brenda L. Koniar, Kelly M. Podetz-Pedersen, Kanut Laoharawee, Renee D. Cooksley, Kathleen E. Meyer, Michael C. Holmes, R S Mc Ivor, Thomas Wechsler, Chester B Whitley

Research output: Contribution to journal › Article

2 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 language	English (US)
Pages (from-to)	178-187
Number of pages	10
Journal	Molecular Therapy
Volume	27
Issue number	1
DOIs	https://doi.org/10.1016/j.ymthe.2018.10.018
State	Published - Jan 2 2019

Fingerprint

Mucopolysaccharidosis I

Clinical Trials

Genome

Injections

Gene Editing

Keywords

gene editing
gene therapy
lysosomal diseases

Cite this

Ou, L., DeKelver, R. C., Rohde, M., Tom, S., Radeke, R., St. Martin, S. J., ... Whitley, C. B. (2019). ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler Syndrome. Molecular Therapy, 27(1), 178-187. https://doi.org/10.1016/j.ymthe.2018.10.018

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, Michael; Koniar, Brenda L.; Podetz-Pedersen, Kelly M.; Laoharawee, Kanut; Cooksley, Renee D.; Meyer, Kathleen E.; Holmes, Michael C.; Mc Ivor, R S; 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, M, Koniar, BL, Podetz-Pedersen, KM, Laoharawee, K, Cooksley, RD, Meyer, KE, Holmes, MC, Mc Ivor, 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, Michael ; Koniar, Brenda L. ; Podetz-Pedersen, Kelly M. ; Laoharawee, Kanut ; Cooksley, Renee D. ; Meyer, Kathleen E. ; Holmes, Michael C. ; Mc Ivor, R S ; 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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10.1016/j.ymthe.2018.10.018