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Investigating the Efficacy of the CRISPR/Cas9 Gene-Editing System for Targeting the HBB FSC 36-37 (-T) Mutation Locus in Hematopoietic Stem Cells.

Molecular biotechnology - 1 Apr 2026

Fereydani Nasim Mayeli, Galehdari Hamid, Hoveizi Elham, Alghasi Arash, Ajami Monireh, Andashti Behnaz, Malayeri Alireza

Abstract excerpt

The emergence of genome editing using the CRISPR/Cas9 system has opened up new possibilities and significantly improved the potential for long-term gene therapy of beta-thalassemia. In Iran, FSC 36/37 (-T) is one of the most common mutations among affected individuals, with the highest frequency...

Topics

Humans
CRISPR-Cas Systems
Gene Editing
HEK293 Cells
Hematopoietic Stem Cells
RNA, Guide, CRISPR-Cas Systems
beta-Thalassemia
Mutation
beta-Globins
Iran

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 40676395
DOI: 10.1007/s12033-025-01465-x

Genes

HBB

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 30 Aug 2026. Prepared record publication-40676395-2026-08-30.1. Manifest publication-graph-cohort-2026-08-30.1.

Source receipt: PubMed via Helena Literature Corpus, retrieved 30 Aug 2026.

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