Article

Beyond HbS and Thalassemia: CRISPR-Cas9 Strategies for Targeting Rare Hemoglobin Gene Mutations

2026-08-21

Abstract excerpt

<title>Abstract</title> <p>Objective To develop an in-silico CRISPR-Cas9 workflow for identifying candidate guide RNAs (gRNAs) targeting rare HBB mutations beyond HbS and β-thalassemia, with emphasis on variants located in exon 3 and near the 3′ end of HBB. Materials and Methods Reference and variant HBB sequences were retrieved from the National Center for Biotechnology Information (NCBI). Candidate CRISPR-Cas...

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Literature Corpus work: 36ec37c8-c02f-566c-a658-8d041970e145
DOI: 10.21203/rs.3.rs-10709958/v1

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