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CRISPR and Fanzor in sickle cell disease: current progress and future prospects
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DOI：10.3389/fgeed.2026.1774014.png)
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Abstract
En 中文
Advancements in genome editing have established a new frontier for the treatment of various genetic diseases; including sickle cell disease (SCD). SCD; the most prevalent monogenic blood disorder; causes severe pain; organ damage; and reduced life expectancy. The recent clinical approval of clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9-based gene therapies for severe sickle cell anemia marks a significant milestone in treating genetic diseases. Despite these breakthroughs; limitations in CRISPR technology persist; requiring further innovation. Alternative approaches; such as the Fanzor (Fz) system; are being developed to complement CRISPR’s capabilities. Unlike CRISPR; which is typically encoded within prokaryotic organisms; Fz is encoded in the eukaryotic genome; offering a universal RNA-guided mechanism applicable across all life kingdoms. Fz’s eukaryotic origin may facilitate more efficient delivery across diverse cell types and tissues; enhancing its therapeutic potential. Here; we will review the current successes and limitations of the CRISPR technology in editing mutation associated with SCD. Additionally; we will explore the potential role of Fz as a genome-editing tool for SCD; a field where its application has not yet been studied.
Keywords:
sickle cell disease
gene therapy
CRISPR
CRISPR-Cas9
genome editing
Fanzor
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