Efforts to Downsize Base Editors for Clinical Applications DOI Open Access
Beomjong Song

International Journal of Molecular Sciences, Journal Year: 2025, Volume and Issue: 26(5), P. 2357 - 2357

Published: March 6, 2025

Since the advent of clustered regularly interspaced short palindromic repeats (CRISPR) system in gene editing field, diverse CRISPR-based tools have been developed for treating genetic diseases. Of these, base editors (BEs) are promising because they can carry out precise at single-nucleotide resolution without inducing DNA double-strand breaks (DSBs), which pose significant risks genomic instability. Despite their outstanding advantages, clinical application BEs remains challenging due to large size, limits efficient delivery, particularly adeno-associated virus (AAV)-based systems. To address this issue, various strategies explored reduce size BEs. These approaches include truncating nonessential domains and replacing bulky components with smaller substitutes compromising efficiency. In review, we highlight importance downsizing therapeutic applications introduce recent advances size-reduction strategies. Additionally, ongoing efforts overcome other limitations BEs, providing insights into potential improving vivo editing.

Language: Английский

Efforts to Downsize Base Editors for Clinical Applications DOI Open Access
Beomjong Song

International Journal of Molecular Sciences, Journal Year: 2025, Volume and Issue: 26(5), P. 2357 - 2357

Published: March 6, 2025

Since the advent of clustered regularly interspaced short palindromic repeats (CRISPR) system in gene editing field, diverse CRISPR-based tools have been developed for treating genetic diseases. Of these, base editors (BEs) are promising because they can carry out precise at single-nucleotide resolution without inducing DNA double-strand breaks (DSBs), which pose significant risks genomic instability. Despite their outstanding advantages, clinical application BEs remains challenging due to large size, limits efficient delivery, particularly adeno-associated virus (AAV)-based systems. To address this issue, various strategies explored reduce size BEs. These approaches include truncating nonessential domains and replacing bulky components with smaller substitutes compromising efficiency. In review, we highlight importance downsizing therapeutic applications introduce recent advances size-reduction strategies. Additionally, ongoing efforts overcome other limitations BEs, providing insights into potential improving vivo editing.

Language: Английский

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