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Article

The Cas9 Hammer-and Sickle: A Challenge for Genome Editors.

The CRISPR journal - 1 Feb 2021

Urnov Fyodor D

Abstract excerpt

Genome editing using CRISPR-Cas9 has produced a functional cure for a small number of patients with sickle cell disease and beta-thalassemia. Rather than repairing the causative mutation, this striking outcome was attained by the knockout of a lineage-specific regulatory element for a gene, BCL11A, that controls fetal hemoglobin levels: a first example of clinical success in targeting a locus initially identified...

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Share to Anemia, Sickle Cell
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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Source

Identifiers

PMID: 33616446
DOI: 10.1089/crispr.2021.29120.fur

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 6 Sept 2026. Manifest publication-graph-cohort-2026-09-06.1.

Source receipt: pubmed, retrieved 6 Sept 2026.

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