Mitochondrial Neurogastrointestinal Encephalomyopathy Caused by Thymidine Phosphorylase Enzyme Deficiency: From Pathogenesis to Emerging Therapeutic Options

Rana Yadak; Peter Sillevis Smitt; Marike W. van Gisbergen; Niek P. van Til; Irenaeus F. M. de Coo

doi:10.3389/fncel.2017.00031

Mitochondrial Neurogastrointestinal Encephalomyopathy Caused by Thymidine Phosphorylase Enzyme Deficiency: From Pathogenesis to Emerging Therapeutic Options

Rana Yadak^*, Peter Sillevis Smitt, Marike W. van Gisbergen, Niek P. van Til, Irenaeus F. M. de Coo^*

^*Corresponding author for this work

GROW - Innovative Cancer Diagnostics & Therapy

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

27 Citations (Web of Science)

Original language	English
Article number	31
Number of pages	14
Journal	Frontiers in Cellular Neuroscience
Volume	11
DOIs	https://doi.org/10.3389/fncel.2017.00031
Publication status	Published - 15 Feb 2017

Keywords

mitochondrial neurogastrointestinal encephalomyopathy
MNGIE
thymidine phosphorylase
metabolic disease
HSCT
HSCGT
lentiviral vector
CELL GENE-THERAPY
CHRONIC GRANULOMATOUS-DISEASE
LONG-TERM SAFETY
LENTIVIRAL VECTOR
MNGIE SYNDROME
DNA DEPLETION
REPLACEMENT THERAPY
HEMATOPOIETIC-CELLS
MURINE MODEL
BONE-MARROW

Access to Document

10.3389/fncel.2017.00031

Cite this

@article{c4583d57f17c425db5ae5f8badf5de5d,

title = "Mitochondrial Neurogastrointestinal Encephalomyopathy Caused by Thymidine Phosphorylase Enzyme Deficiency: From Pathogenesis to Emerging Therapeutic Options",

keywords = "mitochondrial neurogastrointestinal encephalomyopathy, MNGIE, thymidine phosphorylase, metabolic disease, HSCT, HSCGT, lentiviral vector, CELL GENE-THERAPY, CHRONIC GRANULOMATOUS-DISEASE, LONG-TERM SAFETY, LENTIVIRAL VECTOR, MNGIE SYNDROME, DNA DEPLETION, REPLACEMENT THERAPY, HEMATOPOIETIC-CELLS, MURINE MODEL, BONE-MARROW",

author = "Rana Yadak and Smitt, {Peter Sillevis} and {van Gisbergen}, {Marike W.} and {van Til}, {Niek P.} and {de Coo}, {Irenaeus F. M.}",

year = "2017",

month = feb,

day = "15",

doi = "10.3389/fncel.2017.00031",

language = "English",

volume = "11",

journal = "Frontiers in Cellular Neuroscience",

issn = "1662-5102",

publisher = "Frontiers Media S.A.",

}

Mitochondrial Neurogastrointestinal Encephalomyopathy Caused by Thymidine Phosphorylase Enzyme Deficiency : From Pathogenesis to Emerging Therapeutic Options. / Yadak, Rana; Smitt, Peter Sillevis; van Gisbergen, Marike W.; van Til, Niek P.; de Coo, Irenaeus F. M.

In: Frontiers in Cellular Neuroscience, Vol. 11, 31, 15.02.2017.

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

TY - JOUR

T1 - Mitochondrial Neurogastrointestinal Encephalomyopathy Caused by Thymidine Phosphorylase Enzyme Deficiency

T2 - From Pathogenesis to Emerging Therapeutic Options

AU - Yadak, Rana

AU - Smitt, Peter Sillevis

AU - van Gisbergen, Marike W.

AU - van Til, Niek P.

AU - de Coo, Irenaeus F. M.

PY - 2017/2/15

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KW - mitochondrial neurogastrointestinal encephalomyopathy

KW - MNGIE

KW - thymidine phosphorylase

KW - metabolic disease

KW - HSCT

KW - HSCGT

KW - lentiviral vector

KW - CELL GENE-THERAPY

KW - CHRONIC GRANULOMATOUS-DISEASE

KW - LONG-TERM SAFETY

KW - LENTIVIRAL VECTOR

KW - MNGIE SYNDROME

KW - DNA DEPLETION

KW - REPLACEMENT THERAPY

KW - HEMATOPOIETIC-CELLS

KW - MURINE MODEL

KW - BONE-MARROW

U2 - 10.3389/fncel.2017.00031

DO - 10.3389/fncel.2017.00031

M3 - Article

C2 - 28261062

VL - 11

JO - Frontiers in Cellular Neuroscience

JF - Frontiers in Cellular Neuroscience

SN - 1662-5102

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