Genome editing in large animal models.

Area of Science:

• Biomedical research
• Translational medicine
• Gene therapy

Background:

• Genome editing holds promise for treating human diseases but faces clinical implementation challenges.
• Preclinical evaluation in large animal models is increasingly vital for assessing gene editing therapeutics.
• Small animal models have limitations in evaluating long-term safety and host immune responses.

Purpose of the Study:

• To review the progress of novel genome editing therapies in large animal models.
• To highlight the importance of large animals for evaluating immunogenicity and long-term safety.
• To focus on applications in HIV-1 infection, cancer, and genetic diseases.

Main Methods:

• Review of current research on genome editing therapies in large animal models.
• Comparison of large animal models (dogs, pigs, NHPs) with small animal models.
• Focus on specific disease applications and evaluation metrics.

Main Results:

• Large animal models offer greater anatomical, immunological, and lifespan relevance to humans.
• These models facilitate longer-term studies with clinically relevant dosing and sampling.
• Progress is being made in evaluating gene editing for HIV-1, cancer, and genetic disorders.

Conclusions:

• Large animal models are essential for rigorous preclinical evaluation of gene editing therapies.
• They enable comprehensive assessment of safety, efficacy, and immunogenicity.
• Advancements in large animal models accelerate the clinical translation of gene editing treatments.