In vivo somatic cell base editing and prime editing

Area of Science:

• Molecular Biology
• Genetics
• Biotechnology

Background:

• Genome editing advances offer potential for single-dose cures for genetic diseases.
• Precise DNA correction is crucial for effective genetic disorder treatment.
• Base editors (BEs) and prime editors enable precise DNA modifications.

Purpose of the Study:

• To review in vivo base editing and prime editing applications in somatic cells.
• To summarize the therapeutic potential of these genome editing tools in animal models.
• To discuss future prospects for in vivo genome editing therapies.

Main Methods:

• Development of base editors (BEs) for single-base substitutions.
• Development of prime editors for insertions and deletions.
• In vivo delivery of editors in animal models using viral and non-viral methods.

Main Results:

• Base editors and prime editors correct specific DNA changes with high precision.
• These editors show greater efficiency and fewer byproducts than DSB-dependent methods, especially in non-dividing cells.
• Successful in vivo delivery in animal models demonstrates therapeutic potential.

Conclusions:

• Base editors and prime editors are effective agents for in vivo therapeutic genome editing.
• These technologies hold significant promise for treating genetic diseases in animals.
• Further development is expected to expand their clinical applications.