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Precision gene editing technologies: From proof-of-concept to clinical therapies

Tongtong Cui1,2,# Send email to [email protected]

4d ago· 5 min readenInsight

science health biotechnology gene therapy

Summary

This review article examines the evolution of gene therapy from gene addition to precise genome editing, focusing on breakthrough technologies like CRISPR-Cas nucleases, base editors, prime editors, and CRISPR-associated transposases. It covers clinical applications from ex vivo therapies to in vivo treatments targeting vital organs, highlights personalized medicine approaches such as carbamoyl phosphate synthetase 1 editing for rare diseases (N-of-1 medicine), and discusses clinical trial progress, delivery challenges, accessibility issues, and the role of AI in optimizing editing tools and predicting outcomes.

Source

bskyPrecision gene editing technologies: From proof-of-concept to clinical therapiescell.com

Key quotes

· 4 pulled

Gene therapy is evolving from gene addition to precise genome editing, enabling the direct correction of disease-causing mutations.

Breakthrough technologies, such as CRISPR–Cas nucleases, base editors, prime editors, and CRISPR-associated transposases are reshaping the therapeutic landscape.

The rise of personalized medicine, highlighted by therapies, such as carbamoyl phosphate synthetase 1 editing, underscores the shift toward N-of-1 medicine for rare diseases.

These innovations are transforming genetic medicine, offering the promise of safer, more durable, and personalized cures.

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Gene therapy is evolving from gene addition to precise genome editing, enabling the direct correction of disease-causing mutations. Breakthrough technologies, such as clustered regularly interspaced short palindromic repeats-CRISPR-associated protein (CRI

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