Applications and challenges of CRISPR-Cas gene-editing to disease treatment in clinics

Liu, Wenyi; Li, Luoxi; Jiang, Jianxin; Wu, Min; Lin, Ping

doi:10.1093/pcmedi/pbab014

Cited by 58 publications

(38 citation statements)

References 151 publications

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“…This suggests that a careful combination of viral and nonviral delivery methods may be an alternative approach for cancer treatment in patients. 116 …”

Section: Challenges Of Using Crispr In Cancer Treatmentmentioning

confidence: 99%

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RETRACTED: CRISPR/Cas9: A revolutionary genome editing tool for human cancers treatment

Akram

Haq

Sahreen

et al. 2022

Technol Cancer Res Treat

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Cancer is a genetic disease stemming from genetic and epigenetic mutations and is the second most common cause of death across the globe. Clustered regularly interspaced short palindromic repeats (CRISPR) is an emerging gene-editing tool, acting as a defense system in bacteria and archaea. CRISPR/Cas9 technology holds immense potential in cancer diagnosis and treatment and has been utilized to develop cancer disease models such as medulloblastoma and glioblastoma mice models. In diagnostics, CRISPR can be used to quickly and efficiently detect genes involved in various cancer development, proliferation, metastasis, and drug resistance. CRISPR/Cas9 mediated cancer immunotherapy is a well-known treatment option after surgery, chemotherapy, and radiation therapy. It has marked a turning point in cancer treatment. However, despite its advantages and tremendous potential, there are many challenges such as off-target effects, editing efficiency of CRISPR/Cas9, efficient delivery of CRISPR/Cas9 components into the target cells and tissues, and low efficiency of HDR, which are some of the main issues and need further research and development for completely clinical application of this novel gene editing tool. Here, we present a CRISPR/Cas9 mediated cancer treatment method, its role and applications in various cancer treatments, its challenges, and possible solution to counter these challenges.

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“…This suggests that a careful combination of viral and nonviral delivery methods may be an alternative approach for cancer treatment in patients. 116 …”

Section: Challenges Of Using Crispr In Cancer Treatmentmentioning

confidence: 99%

“…Recently, in a study paired Cas9 nickase has been found to increase HDR efficiency by producing single-strand nicks. 116 …”

Section: Challenges Of Using Crispr In Cancer Treatmentmentioning

confidence: 99%

RETRACTED: CRISPR/Cas9: A revolutionary genome editing tool for human cancers treatment

Akram

Haq

Sahreen

et al. 2022

Technol Cancer Res Treat

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“…However off‐target mutations while targeting the desired genes are still a reality. Therefore, an improved genome editing system based on CRISPR can overcome certain of these limitations (W. Liu et al, 2021). A base editing technique was developed to induce precise editing, but unfortunately, it can only correct four single‐base mutations (Abdullah et al, 2020).…”

Section: Conventional Genome Editing Strategiesmentioning

confidence: 99%

Prime editing: A potential treatment option for β‐thalassemia

Arif

Farooq

Ahmad

et al. 2022

Cell Biology International

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The potential to therapeutically alter the genome is one of the remarkable scientific developments in recent years. Genome editing technologies have provided an opportunity to precisely alter genomic sequence(s) in eukaryotic cells as a treatment option for various genetic disorders. These technologies allow the correction of harmful mutations in patients by precise nucleotide editing. Genome editing technologies such as CRISPR (clustered regularly interspaced short palindromic repeat) and base editors have greatly contributed to the practical applications of gene editing. However, these technologies have certain limitations, including imperfect editing, undesirable mutations, off‐target effects, and lack of potential to simultaneously edit multiple loci. Recently, prime editing (PE) has emerged as a new gene editing technology with the potential to overcome the above‐mentioned limitations. Interestingly, PE not only has higher specificity but also does not require double‐strand breaks. In addition, a minimum possibility of potential off‐target mutant sites makes PE a preferred choice for therapeutic gene editing. Furthermore, PE has the potential to introduce insertion and deletions of all 12 single‐base mutations at target sequences. Considering its potential, PE has been applied as a treatment option for genetic diseases including hemoglobinopathies. β‐Thalassemia, for example, one of the most significant blood disorders characterized by reduced levels of functional hemoglobin, could potentially be treated using PE. Therapeutic reactivation of the γ‐globin gene in adult β‐thalassemia patients through PE technology is considered a promising therapeutic strategy. The current review aims to briefly discuss the genome editing strategies and potential applications of PE for the treatment of β‐thalassemia. In addition, the review will also focus on challenges associated with the use of PE.

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“…RNP shows improved stability, rapid action, low off-target effects, low immunogenicity, and no risk of insertional mutagenesis, despite its high cost and relatively short gene editing duration [36,41,42]. Therefore, depending on specific applications and requirements, all these three formats have been widely explored for many biomedical applications, including therapies for cancer as well as genetic, infectious, and immunological diseases [14,[43][44][45][46].…”

Section: Introductionmentioning

confidence: 99%

Stimuli-responsive nanoformulations for CRISPR-Cas9 genome editing

et al. 2022

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The CRISPR-Cas9 technology has changed the landscape of genome editing and has demonstrated extraordinary potential for treating otherwise incurable diseases. Engineering strategies to enable efficient intracellular delivery of CRISPR-Cas9 components has been a central theme for broadening the impact of the CRISPR-Cas9 technology. Various non-viral delivery systems for CRISPR-Cas9 have been investigated given their favorable safety profiles over viral systems. Many recent efforts have been focused on the development of stimuli-responsive non-viral CRISPR-Cas9 delivery systems, with the goal of achieving efficient and precise genome editing. Stimuli-responsive nanoplatforms are capable of sensing and responding to particular triggers, such as innate biological cues and external stimuli, for controlled CRISPR-Cas9 genome editing. In this Review, we overview the recent advances in stimuli-responsive nanoformulations for CRISPR-Cas9 delivery, highlight the rationale of stimuli and formulation designs, and summarize their biomedical applications.

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Applications and challenges of CRISPR-Cas gene-editing to disease treatment in clinics

Cited by 58 publications

References 151 publications

RETRACTED: CRISPR/Cas9: A revolutionary genome editing tool for human cancers treatment

RETRACTED: CRISPR/Cas9: A revolutionary genome editing tool for human cancers treatment

Prime editing: A potential treatment option for β‐thalassemia

Stimuli-responsive nanoformulations for CRISPR-Cas9 genome editing

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