The use of genetically humanized animal models for personalized medicine approaches

• Main Authors: Annemieke Aartsma-Rus (Author), Maaike van Putten (Author), James Dowling (Author)
• Formato: Livro
• Publicado em: The Company of Biologists, 2020-02-01T00:00:00Z.
• Assuntos: article
• Acesso em linha: Connect to this object online.

For many genetic diseases, researchers are developing personalized medicine approaches. These sometimes employ custom genetic interventions such as antisense-mediated exon skipping or genome editing, aiming to restore protein function in a mutation-specific manner. Animal models can facilitate the d...