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Updated: Aug 6, 2026
Efficient PAM-Less Base Editing for Zebrafish Modeling of Human Genetic Disease with zSpRY-ABE8e
Published on: February 17, 2023
Base editing for precision therapeutics
Moksada Regmi1, Kuiying Ma2, Changhao Bi3
1State Key Laboratory of Vascular Homeostasis and Remodeling, Department of Neurosurgery, Peking University Third Hospital, Peking University, Beijing, China; Center for Precision Neurosurgery and Oncology of Peking University Health Science Center, Peking University, Beijing, China; Center for Oculocranial Pressure Instability Disorders (COPID), Zhengzhou, Henan, China.
Base editing precisely corrects single-nucleotide variants without DNA breaks, offering therapeutic potential for genetic diseases. Challenges remain in specificity, delivery, and long-term safety for precision medicine applications.
Area of Science:
- Molecular Biology
- Genetic Engineering
- Genomics
Background:
- Base editing (BE) precisely modifies DNA or RNA single nucleotides without double-strand breaks.
- Single-nucleotide variants cause over half of known pathogenic genetic variations.
- BE technology has advanced in specificity, efficiency, and delivery for clinical applications.
Purpose of the Study:
- To review the therapeutic promise and clinical applications of base editing.
- To identify key challenges and future directions for base editing technology.
Main Methods:
- Review of recent advances in base editing technology.
- Analysis of clinical successes and translational promise in various genetic diseases.
- Identification of current technical and safety challenges.
Main Results:
- BE shows early clinical success and translational promise in sickle cell disease, beta-thalassemia, leukemia, hypercholesterolemia, alpha-1-antitrypsin deficiency, and glycogen storage disease type Ia.
- Advances have improved base editor specificity, efficiency, and delivery methods.
- Key challenges include bystander editing, off-target effects, delivery constraints, immunogenicity, and long-term safety data.
Conclusions:
- Base editing holds significant therapeutic potential for genetic diseases.
- Continued technological refinement, preclinical validation, and clinical assessment are crucial.
- Realizing BE's full potential in precision medicine requires addressing current challenges.
