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 journalArticle

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

Fingerprint

Mucopolysaccharidosis I
Clinical Trials
Genome
Injections
Gene Editing

Keywords

  • gene editing
  • gene therapy
  • lysosomal diseases

PubMed: MeSH publication types

  • Journal Article
  • Research Support, Non-U.S. Gov't

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, 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 journalArticle

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.
@article{2cd91fbca5504a678130b955ddf3da44,
title = "ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler Syndrome",
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.",
keywords = "gene editing, gene therapy, lysosomal diseases",
author = "Li Ou and DeKelver, {Russell C.} and Michelle Rohde and Susan Tom and Robert Radeke and {St. Martin}, {Susan J.} and Yolanda Santiago and Scott Sproul and Michael Przybilla and Koniar, {Brenda L.} and Podetz-Pedersen, {Kelly M.} and Kanut Laoharawee and Cooksley, {Renee D.} and Meyer, {Kathleen E.} and Holmes, {Michael C.} and {Mc Ivor}, {R S} and Thomas Wechsler and Whitley, {Chester B}",
year = "2019",
month = "1",
day = "2",
doi = "10.1016/j.ymthe.2018.10.018",
language = "English (US)",
volume = "27",
pages = "178--187",
journal = "Molecular Therapy",
issn = "1525-0016",
publisher = "Nature Publishing Group",
number = "1",

}

TY - JOUR

T1 - ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler Syndrome

AU - Ou, Li

AU - DeKelver, Russell C.

AU - Rohde, Michelle

AU - Tom, Susan

AU - Radeke, Robert

AU - St. Martin, Susan J.

AU - Santiago, Yolanda

AU - Sproul, Scott

AU - Przybilla, Michael

AU - Koniar, Brenda L.

AU - Podetz-Pedersen, Kelly M.

AU - Laoharawee, Kanut

AU - Cooksley, Renee D.

AU - Meyer, Kathleen E.

AU - Holmes, Michael C.

AU - Mc Ivor, R S

AU - Wechsler, Thomas

AU - Whitley, Chester B

PY - 2019/1/2

Y1 - 2019/1/2

N2 - 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.

AB - 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.

KW - gene editing

KW - gene therapy

KW - lysosomal diseases

UR - http://www.scopus.com/inward/record.url?scp=85057869308&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=85057869308&partnerID=8YFLogxK

U2 - 10.1016/j.ymthe.2018.10.018

DO - 10.1016/j.ymthe.2018.10.018

M3 - Article

C2 - 30528089

AN - SCOPUS:85057869308

VL - 27

SP - 178

EP - 187

JO - Molecular Therapy

JF - Molecular Therapy

SN - 1525-0016

IS - 1

ER -