Preclinical Efficacy and Safety Evaluation of Hematopoietic Stem Cell Gene Therapy in a Mouse Model of MNGIE

Rana Yadak; R Cabrera-Perez; J Torres-Torronteras; M Bugiani; Joost Haeck; MW Huston; Elly Taal; S Goffart; Ed Jacobs; M Stok; L Leonardelli; L Biasco; Rob Verdijk; Monique Bernsen; GJG Ruijter; R Marti; G Wagemaker; NP Til; IFM Coo

doi:10.1016/j.omtm.2018.01.001

Preclinical Efficacy and Safety Evaluation of Hematopoietic Stem Cell Gene Therapy in a Mouse Model of MNGIE

Rana Yadak, R Cabrera-Perez, J Torres-Torronteras, M Bugiani, Joost Haeck, MW Huston, Elly Taal, S Goffart, Ed Jacobs, M Stok, L Leonardelli, L Biasco, Rob Verdijk, Monique Bernsen, GJG Ruijter, R Marti, G Wagemaker, NP Til, IFM Coo

Research output: Contribution to journal › Article › Academic › peer-review

18 Citations (Scopus)

35 Downloads (Pure)

Original language	Undefined/Unknown
Pages (from-to)	152-165
Number of pages	14
Journal	Molecular Therapy-Methods & Clinical Development
Volume	8
DOIs	https://doi.org/10.1016/j.omtm.2018.01.001
Publication status	Published - 2018

Research programs

EMC NIHES-03-30-02
EMC NIHES-03-30-03

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10.1016/j.omtm.2018.01.001

REPUB_105259-OA.pdfFinal published version, 2.33 MB

http://hdl.handle.net/1765/105259

Cite this

Yadak, R., Cabrera-Perez, R., Torres-Torronteras, J., Bugiani, M., Haeck, J., Huston, MW., Taal, E., Goffart, S., Jacobs, E., Stok, M., Leonardelli, L., Biasco, L., Verdijk, R., Bernsen, M., Ruijter, GJG., Marti, R., Wagemaker, G., Til, NP., & Coo, IFM. (2018). Preclinical Efficacy and Safety Evaluation of Hematopoietic Stem Cell Gene Therapy in a Mouse Model of MNGIE. Molecular Therapy-Methods & Clinical Development, 8, 152-165. https://doi.org/10.1016/j.omtm.2018.01.001

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author = "Rana Yadak and R Cabrera-Perez and J Torres-Torronteras and M Bugiani and Joost Haeck and MW Huston and Elly Taal and S Goffart and Ed Jacobs and M Stok and L Leonardelli and L Biasco and Rob Verdijk and Monique Bernsen and GJG Ruijter and R Marti and G Wagemaker and NP Til and IFM Coo",

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Yadak, R, Cabrera-Perez, R, Torres-Torronteras, J, Bugiani, M, Haeck, J, Huston, MW, Taal, E, Goffart, S, Jacobs, E, Stok, M, Leonardelli, L, Biasco, L, Verdijk, R , Bernsen, M, Ruijter, GJG, Marti, R, Wagemaker, G, Til, NP & Coo, IFM 2018, 'Preclinical Efficacy and Safety Evaluation of Hematopoietic Stem Cell Gene Therapy in a Mouse Model of MNGIE', Molecular Therapy-Methods & Clinical Development, vol. 8, pp. 152-165. https://doi.org/10.1016/j.omtm.2018.01.001

TY - JOUR

T1 - Preclinical Efficacy and Safety Evaluation of Hematopoietic Stem Cell Gene Therapy in a Mouse Model of MNGIE

AU - Yadak, Rana

AU - Cabrera-Perez, R

AU - Torres-Torronteras, J

AU - Bugiani, M

AU - Haeck, Joost

AU - Huston, MW

AU - Taal, Elly

AU - Goffart, S

AU - Jacobs, Ed

AU - Stok, M

AU - Leonardelli, L

AU - Biasco, L

AU - Verdijk, Rob

AU - Bernsen, Monique

AU - Ruijter, GJG

AU - Marti, R

AU - Wagemaker, G

AU - Til, NP

AU - Coo, IFM

PY - 2018

Y1 - 2018

U2 - 10.1016/j.omtm.2018.01.001

DO - 10.1016/j.omtm.2018.01.001

M3 - Article

C2 - 29687034

SN - 2329-0501

VL - 8

SP - 152

EP - 165

JO - Molecular Therapy-Methods & Clinical Development

JF - Molecular Therapy-Methods & Clinical Development

ER -