C57BL/6JCya-Bcl11aem1flox/Cya
Common Name:
Bcl11a-flox
Product ID:
S-CKO-02274
Background:
C57BL/6JCya
Product Type
Age
Genotype
Sex
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Basic Information
Strain Name
Bcl11a-flox
Strain ID
CKOCMP-14025-Bcl11a-B6J-VA
Gene Name
Product ID
S-CKO-02274
Gene Alias
2810047E18Rik; BCL-11A; Ctip1; D930021L15Rik; Evi9; Evi9a; Evi9b; Evi9c; mKIAA1809
Background
C57BL/6JCya
NCBI ID
Modification
Conditional knockout
Chromosome
11
Phenotype
Document
Application
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Note: When using this mouse strain in a publication, please cite “C57BL/6JCya-Bcl11aem1flox/Cya mice (Catalog S-CKO-02274) were purchased from Cyagen.”
Strain Description
Ensembl Number
ENSMUST00000109514
NCBI RefSeq
NM_001242934
Target Region
Exon 4
Size of Effective Region
~2.5 kb
Detailed Document
Overview of Gene Research
BCL11A, encoding a zinc-finger protein, is a transcriptional repressor predominantly expressed in the brain and hematopoietic tissue. It is crucial in brain and hematopoietic system development, especially in the fetal-to-adult hemoglobin switching process. Genetic variations, microRNAs, and transcription factors regulate its expression [3].
Erythroid-specific loss of BCL11A in engineered sickle cell disease (SCD) mice rescues the phenotype, suggesting that down-regulating BCL11A could benefit patients with SCD and β-thalassemia [1]. Clinical trials targeting the GATA-binding site in the BCL11A erythroid-specific enhancer using Nuclease technology gene editing have led to approval for ex vivo treatment of SCD and β-thalassemia [1]. Also, in a study, post-transcriptional genetic silencing of BCL11A in patients with sickle cell disease via infusion of autologous CD34+ cells transduced with a lentiviral vector encoding shRNA targeting BCL11A mRNA led to robust and stable HbF induction and reduced clinical manifestations [2].
In conclusion, BCL11A is essential for the fetal-to-adult hemoglobin switch. Studies using gene-edited mouse models and human clinical trials have shown that modulating BCL11A expression can be a promising therapeutic strategy for β-hemoglobinopathies like SCD and β-thalassemia.
References:
1. Zheng, Ge, Orkin, Stuart H. . Transcriptional Repressor BCL11A in Erythroid Cells. In Advances in experimental medicine and biology, 1459, 199-215. doi:10.1007/978-3-031-62731-6_9. https://pubmed.ncbi.nlm.nih.gov/39017845/
2. Esrick, Erica B, Lehmann, Leslie E, Biffi, Alessandra, Manis, John P, Williams, David A. 2020. Post-Transcriptional Genetic Silencing of BCL11A to Treat Sickle Cell Disease. In The New England journal of medicine, 384, 205-215. doi:10.1056/NEJMoa2029392. https://pubmed.ncbi.nlm.nih.gov/33283990/
3. Yin, Jiawei, Xie, Xiaoli, Ye, Yufu, Wang, Lijuan, Che, Fengyuan. . BCL11A: a potential diagnostic biomarker and therapeutic target in human diseases. In Bioscience reports, 39, . doi:10.1042/BSR20190604. https://pubmed.ncbi.nlm.nih.gov/31654056/
Quality Control Standard
Sperm Test
Pre-cryopreservation: Measurement of sperm concentration, determination of sperm viability.
Post-cryopreservation: A vial of cryopreserved sperms is selected for in-vitro fertilization from each batch.
Environmental Standards:SPF
Available Region:Global
Source:Cyagen