Key result
CRISPR-Cas9 gene editing demonstrates potential for correcting genetic mutations causing congenital heart diseases in experimental models, though significant technical and safety challenges remain.
Why the study?
Congenital heart disease is often caused by genetic mutations, and CRISPR-Cas9-based genome editing has gained attention for its therapeutic potential to prevent and treat these conditions.
This review discusses the potential therapeutic application of CRISPR-Cas9 genome editing technology for congenital heart disease.
CRISPR-Cas9 for CHD mutations warrants clinical caution; leaves open prospective trials to confirm safe mutation correction.
Clustered regularly interspaced short palindromic repeats and CRISPR-associated protein 9 (CRISPR-Cas9) is an ancient prokaryotic defense system that precisely cuts foreign genomic DNA under the control of a small number of guide RNAs. The CRISPR-Cas9 system facilitates efficient double-stranded DNA cleavage that has been recently adopted for genome editing to create or correct inherited genetic mutations causing disease. Congenital heart disease (CHD) is generally caused by genetic mutations such as base substitutions, deletions, and insertions, which result in diverse developmental defects and remains a leading cause of birth defects. Pediatric CHD patients exhibit a spectrum of cardiac abnormalities such as septal defects, valvular defects, and abnormal chamber development. CHD onset occurs during the prenatal period and often results in early lethality during childhood. Because CRISPR-Cas9-based genome editing technology has gained considerable attention for its potential to prevent and treat diseases, we will review the CRISPR-Cas9 system as a genome editing tool and focus on its therapeutic application for CHD.
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Seok et al. (2021) conducted a review in Congenital heart disease. CRISPR-Cas9 gene editing was evaluated. CRISPR-Cas9 gene editing demonstrates potential for correcting genetic mutations causing congenital heart diseases in experimental models, though significant technical and safety challenges remain.
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