Rare diseases - not so rare! Scientists from @IIMCB_Poland, using the #CRISPR-Cas9 technology created zebrafish lines with mutations similar to those of the patients. The lines were used to creata models for the study of many childhood neurodegenerative diseases: NPC-2 and MPS-III.
This tweet was the Polish contribution to the DNA 2.0 campaign, which aims to promote the scientific activities of member institutions. The essence of the campaign was to identify one area of the scientific institution's activities and show the positive impact of its research on society.
The campaign's message focused on communicating that the use of animal models has contributed to overcoming barriers to understanding and treating rare diseases. The zebrafish (Danio rerio) was given as an example. The fish has also set new trends in genetics and development, pharmacodynamics, toxicology, environmental control, behavior, etc., offering new ways to find therapies for patients.
More about it in the article.