Evaluating the feasibility of gene replacement strategies to treat MTRFR deficiency
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| Main Authors: | , , , , , , , , , , |
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| Format: | Article |
| Language: | English |
| Published: |
The Company of Biologists
2025-05-01
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| Series: | Disease Models & Mechanisms |
| Subjects: | |
| Online Access: | http://dmm.biologists.org/content/18/5/dmm052120 |
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| _version_ | 1849334690782117888 |
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| author | Samia L. Pratt Mariana Zarate-Mendez Lidiia Koludarova Sonja Jansson Mikko Airavaara Irena Hlushchuk David Coleman Caleb Heffner Rita Horvath Brendan J. Battersby Robert W. Burgess |
| author_facet | Samia L. Pratt Mariana Zarate-Mendez Lidiia Koludarova Sonja Jansson Mikko Airavaara Irena Hlushchuk David Coleman Caleb Heffner Rita Horvath Brendan J. Battersby Robert W. Burgess |
| author_sort | Samia L. Pratt |
| collection | DOAJ |
| format | Article |
| id | doaj-art-584dbfb3aeb142ab886f040fd06ca038 |
| institution | Kabale University |
| issn | 1754-8403 1754-8411 |
| language | English |
| publishDate | 2025-05-01 |
| publisher | The Company of Biologists |
| record_format | Article |
| series | Disease Models & Mechanisms |
| spelling | doaj-art-584dbfb3aeb142ab886f040fd06ca0382025-08-20T03:45:30ZengThe Company of BiologistsDisease Models & Mechanisms1754-84031754-84112025-05-0118510.1242/dmm.052120052120Evaluating the feasibility of gene replacement strategies to treat MTRFR deficiencySamia L. Pratt0Mariana Zarate-Mendez1Lidiia Koludarova2Sonja Jansson3Mikko Airavaara4Irena Hlushchuk5David Coleman6Caleb Heffner7Rita Horvath8Brendan J. Battersby9Robert W. Burgess10 The Jackson Laboratory, Bar Harbor, ME 04609, USA Department of Clinical Neurosciences, John Van Geest Centre for Brain Repair, University of Cambridge, CB2 0PY Cambridge, UK Institute of Biotechnology, HiLIFE, University of Helsinki, 00014 Helsinki, Finland Institute of Biotechnology, HiLIFE, University of Helsinki, 00014 Helsinki, Finland Drug Research Program, Faculty of Pharmacy, University of Helsinki, 00014 Helsinki, Finland Drug Research Program, Faculty of Pharmacy, University of Helsinki, 00014 Helsinki, Finland The Jackson Laboratory, Bar Harbor, ME 04609, USA The Jackson Laboratory, Bar Harbor, ME 04609, USA Department of Clinical Neurosciences, John Van Geest Centre for Brain Repair, University of Cambridge, CB2 0PY Cambridge, UK Institute of Biotechnology, HiLIFE, University of Helsinki, 00014 Helsinki, Finland The Jackson Laboratory, Bar Harbor, ME 04609, USA http://dmm.biologists.org/content/18/5/dmm052120c12orf65leigh syndromecmt6mitochondrial translationmrpl58behr's syndrome |
| spellingShingle | Samia L. Pratt Mariana Zarate-Mendez Lidiia Koludarova Sonja Jansson Mikko Airavaara Irena Hlushchuk David Coleman Caleb Heffner Rita Horvath Brendan J. Battersby Robert W. Burgess Evaluating the feasibility of gene replacement strategies to treat MTRFR deficiency Disease Models & Mechanisms c12orf65 leigh syndrome cmt6 mitochondrial translation mrpl58 behr's syndrome |
| title | Evaluating the feasibility of gene replacement strategies to treat MTRFR deficiency |
| title_full | Evaluating the feasibility of gene replacement strategies to treat MTRFR deficiency |
| title_fullStr | Evaluating the feasibility of gene replacement strategies to treat MTRFR deficiency |
| title_full_unstemmed | Evaluating the feasibility of gene replacement strategies to treat MTRFR deficiency |
| title_short | Evaluating the feasibility of gene replacement strategies to treat MTRFR deficiency |
| title_sort | evaluating the feasibility of gene replacement strategies to treat mtrfr deficiency |
| topic | c12orf65 leigh syndrome cmt6 mitochondrial translation mrpl58 behr's syndrome |
| url | http://dmm.biologists.org/content/18/5/dmm052120 |
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