CRISPR-based genome editing in disease treatment

Xu, Weihui; Yao, Kai

doi:10.1016/j.molmed.2023.05.003

Cited by 2 publications

(3 citation statements)

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“…The advent of CRISPR/Cas9 technology has revolutionized the field, offering new possibilities for treating human genetic diseases. Its precision in genome editing has made it a valuable tool in various clinical and therapeutic applications, yet its double-stranded breaks may lead to some unpredictable consequences ( Qin et al, 2023a ; Bhatia et al, 2023 ). The base editing technique enables precise modification of the human genome by achieving targeted conversion independent of HDR and double-strand DNA breaks, directly altering individual base pairs within DNA sequences.…”

Section: The Application Of Base Editing In the Treatment Of Genetic ...mentioning

confidence: 99%

Breaking genetic shackles: The advance of base editing in genetic disorder treatment

Xu,

Zheng,

et al. 2024

Front. Pharmacol.

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The rapid evolution of gene editing technology has markedly improved the outlook for treating genetic diseases. Base editing, recognized as an exceptionally precise genetic modification tool, is emerging as a focus in the realm of genetic disease therapy. We provide a comprehensive overview of the fundamental principles and delivery methods of cytosine base editors (CBE), adenine base editors (ABE), and RNA base editors, with a particular focus on their applications and recent research advances in the treatment of genetic diseases. We have also explored the potential challenges faced by base editing technology in treatment, including aspects such as targeting specificity, safety, and efficacy, and have enumerated a series of possible solutions to propel the clinical translation of base editing technology. In conclusion, this article not only underscores the present state of base editing technology but also envisions its tremendous potential in the future, providing a novel perspective on the treatment of genetic diseases. It underscores the vast potential of base editing technology in the realm of genetic medicine, providing support for the progression of gene medicine and the development of innovative approaches to genetic disease therapy.

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Section: The Application Of Base Editing In the Treatment Of Genetic ...mentioning

confidence: 99%

Breaking genetic shackles: The advance of base editing in genetic disorder treatment

Xu,

Zheng,

et al. 2024

Front. Pharmacol.

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“…Sickle cell disease and β-thalassemia have become the paradigm for CRISPR-based therapeutic genome editing in human disease, with the first regulatory approvals for Casgevy (exagamglogene autotemcel) and Lyfgenia (lovotibeglogene autotemcel) [ 19 , 20 ]. These gene editing therapies use the patient’s hematopoietic stem cells, modified ex vivo by genome editing using CRISPR-Cas9 technology, and then transplanted as a single-dose infusion [ 19 , 20 ]. Before treatment, the patient’s stem cells are collected and undergo myeloablative conditioning with high-dose chemotherapy.…”

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confidence: 99%

“…Before treatment, the patient’s stem cells are collected and undergo myeloablative conditioning with high-dose chemotherapy. The modified stem cells are then transplanted to the patient, engrafting within the bone marrow [ 19 , 20 ]. Casgevy (exagamglogene autotemcel) is used to edit CD34 + cell human hematopoietic stem and progenitor cells at the erythroid-specific enhancer region of the BCL11A gene, which prevents the production of fetal hemoglobin (HbF) [ 21 ].…”

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Editorial: First Regulatory Approvals for CRISPR-Cas9 Therapeutic Gene Editing for Sickle Cell Disease and Transfusion-Dependent β-Thalassemia

Parums

2024

Med Sci Monit

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In 2020, Emmanuelle Charpentier and Jennifer Doudna were awarded the Nobel Prize in Chemistry for their research on the endonuclease, clustered regularly interspaced short palindromic repeats (CRISPR) and the CRISPR-associated protein 9 (CRISPR-Cas9) method for DNA editing. On 16 November 2023, the UK Medicines and Healthcare Products Regulatory Agency (MHRA) was the first to approve the CRISPR-Cas9 gene editing therapy, Casgevy (exagamglogene autotemcel), for the treatment of patients with transfusion-dependent β-thalassemia and the treatment of sickle cell disease in patients aged ≥12 years with recurrent vaso-occlusive crises. On 8 December 2023, the US Food and Drug Administration (FDA) approved both Casgevy and Lyfgenia (lovotibeglogene autotemcel) for patients with sickle cell disease. On 15 December 2023, the European Medicines Agency (EMA) approved Casgevy for sickle cell disease and transfusion-dependent β-thalassemia. This Editorial aims to present an update on the landmark first regulatory approvals of CRISPR-Cas9 for patients with sickle cell disease and transfusion-dependent β-thalassemia and the potential challenges for therapeutic gene (DNA) editing.

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CRISPR-based genome editing in disease treatment

Cited by 2 publications

References 10 publications

Breaking genetic shackles: The advance of base editing in genetic disorder treatment

Breaking genetic shackles: The advance of base editing in genetic disorder treatment

Editorial: First Regulatory Approvals for CRISPR-Cas9 Therapeutic Gene Editing for Sickle Cell Disease and Transfusion-Dependent β-Thalassemia

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