Modeling Rare Human Disorders in Mice: The Finnish Disease Heritage

Tomáš Zárybnický, Anne Heikkinen, Salla M. Kangas, Marika Karikoski, Guillermo Antonio Martinez-Nieto, Miia H. Salo, Johanna Uusimaa, Reetta Vuolteenaho, Reetta Hinttala, Petra Sipilä, Satu Kuure

Research output: Contribution to journalReview Articlepeer-review

Original languageEnglish
Article number3158
JournalCells
Volume10
Issue number11
Number of pages24
ISSN2073-4409
DOIs
Publication statusPublished - Nov 2021
MoE publication typeA2 Review article in a scientific journal

Fields of Science

  • rare diseases
  • monogenic diseases
  • mouse models
  • <p>CRISPR/Cas9</p>
  • genome engineering
  • Finnish disease heritage
  • 3-HYDROXYACYL-COA DEHYDROGENASE-DEFICIENCY
  • NEURONAL CEROID-LIPOFUSCINOSIS
  • LYSOSOMAL STORAGE DISORDER
  • CARTILAGE-HAIR HYPOPLASIA
  • MITOCHONDRIAL TRIFUNCTIONAL PROTEIN
  • STEM-CELL TRANSPLANTATION
  • DEGENERATION MND MUTANT
  • ACUTE FATTY LIVER
  • MOUSE MODEL
  • HYDROLETHALUS SYNDROME
  • 1182 Biochemistry, cell and molecular biology

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