Therapeutic gene editing strategies using CRISPR-Cas9 for the β-hemoglobinopathies

Papizan James B.; Porter Shaina N.; Sharma Akshay; Pruett-Miller Shondra M.

doi:10.7555/JBR.34.20200096

Volume 35 Issue 2

Mar. 2021

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Article Navigation > The Journal of Biomedical Research > 2021 > 35(2): 115-134

Papizan James B., Porter Shaina N., Sharma Akshay, Pruett-Miller Shondra M.. Therapeutic gene editing strategies using CRISPR-Cas9 for the β-hemoglobinopathies[J]. The Journal of Biomedical Research, 2021, 35(2): 115-134. doi: 10.7555/JBR.34.20200096

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Papizan James B., Porter Shaina N., Sharma Akshay, Pruett-Miller Shondra M.. Therapeutic gene editing strategies using CRISPR-Cas9 for the β-hemoglobinopathies[J]. The Journal of Biomedical Research, 2021, 35(2): 115-134. doi: 10.7555/JBR.34.20200096

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Therapeutic gene editing strategies using CRISPR-Cas9 for the β-hemoglobinopathies

doi: 10.7555/JBR.34.20200096

1.
Department of Cellular and Molecular Biology
2.
Center for Advanced Genome Engineering
3.
Department of Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis, TN 38105, USA

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Corresponding author: Shondra M. Pruett-Miller, Department of Cellular and Molecular Biology, St. Jude Children's Research Hospital, 262 Danny Thomas Place, Memphis, TN 38105, USA. Tel/Fax: +1-901-595-7313/+1-901-525-8025, E-mail: shondra.miller@stjude.org
Received: 2020-06-18
Revised: 2020-09-02
Accepted: 2020-09-16

Published: 2020-11-09

Issue Date: 2021-03-26

Abstract

Abstract

With advancements in gene editing technologies, our ability to make precise and efficient modifications to the genome is increasing at a remarkable rate, paving the way for scientists and clinicians to uniquely treat a multitude of previously irremediable diseases. CRISPR-Cas9, short for clustered regularly interspaced short palindromic repeats and CRISPR-associated protein 9, is a gene editing platform with the ability to alter the nucleotide sequence of the genome in living cells. This technology is increasing the number and pace at which new gene editing treatments for genetic disorders are moving toward the clinic. The β-hemoglobinopathies are a group of monogenic diseases, which despite their high prevalence and chronic debilitating nature, continue to have few therapeutic options available. In this review, we will discuss our existing comprehension of the genetics and current state of treatment for β-hemoglobinopathies, consider potential genome editing therapeutic strategies, and provide an overview of the current state of clinical trials using CRISPR-Cas9 gene editing.
- sickle cell disease,
- sickle cell anemia,
- fetal hemoglobin,
- hemoglobinopathy,
- CRISPR,
- gene editing,
- genome engineering

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References(179)

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