The first two decades of this century have seen tremendous progress in understanding human diseases through manipulation of the mouse genome, including with knockout, conditional knockout, transgenic, knock-in, point mutation, and different levels of humanization. Collectively, these efforts have contributed enormously to the understanding of gene functions as well as genetic causes of human diseases.


The advent of CRISPR technology has also greatly facilitated the genetic engineering of the mouse genome. Now we can link a multitude of genetic mutations on individual genes to thousands of monogenic human diseases, a majority of which are also rare diseases. The humanization of murine models has quickly become an indispensable tool in modern drug discovery and therapeutic development research across numerous fields.


In Cyagen's exclusive White Paper, our experts briefly introduce the background of gene therapy for rare diseases and explore the applications of humanized mouse models for genetic diseases.


Outline of Contents:

1. Gene Therapy and Rare Diseases

2. Mouse Models for Drug Development Research

  •     Humanized Mouse Models:  Development Methods & Strategies
  •     Case Example: APOE Humanization in Mice

3. Optimized Model Humanization for Improved Therapeutic Relevance



About Cyagen

With more than 15 years' experience in genetic engineering, Cyagen offers a complete range of services to support gene therapy studies and rare diseases research model development.

Contact us with your gene target(s) and desired model(s) - our custom model generation experts will be in touch with you to provide a free project strategy and quote.