Advances in miniature CRISPR-Cas proteins and their applications in gene editing

Wu, Huimin; Sun, Yixiang; Wang, Yimai; Luo, Liqiang; Song, Yizhi

doi:10.1007/s00203-024-03962-0

Arch Microbiol

2024

DOI: 10.1007/s00203-024-03962-0

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Advances in miniature CRISPR-Cas proteins and their applications in gene editing

Huimin Wu,

Yixiang Sun,

Yimai Wang

et al.

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A route to engineer a genome editor for gene therapy

Yu,

Qiao

2024

Molecular Therapy - Nucleic Acids

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A route to engineer a genome editor for gene therapy

Yu,

Qiao

2024

Molecular Therapy - Nucleic Acids

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Impact of Chromatin Organization and Epigenetics on CRISPR-Cas and TALEN Genome Editing

Jain,

Xun,

Zhao

2024

ACS Synth. Biol.

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DNA lies at the heart of the central dogma of life. Altering DNA can modify the flow of information in fundamental cellular processes such as transcription and translation. The ability to precisely manipulate DNA has led to remarkable advances in treating incurable human genetic ailments and has changed the landscape of biological research. Genome editors such as CRISPR-Cas nucleases and TALENs have become ubiquitous tools in basic and applied biological research and have been translated to the clinic to treat patients. The specificity and modularity of these genome editors have made it possible to efficiently engineer genomic DNA; however, underlying principles governing editing outcomes in eukaryotes are still being uncovered. Editing efficiency can vary from cell type to cell type for the same DNA target sequence, necessitating de novo design and validation efforts. Chromatin structure and epigenetic modifications have been shown to affect the activity of genome editors because of the role they play in hierarchical organization of the underlying DNA. Understanding the nuclear search mechanism of genome editors and their molecular interactions with higher order chromatin will lead to improved models for predicting precise genome editing outcomes. Insights from such studies will unlock the entire genome to be engineered for the creation of novel therapies to treat critical illnesses.

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Prospects for gene therapy in polycystic kidney disease

Chakraborty,

2024

Current Opinion in Nephrology &Amp; Hypertension

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Purpose of review We aim to provide an updated perspective on the recent advancements in gene therapy for polycystic kidney disease (PKD), a genetic disorder with significant morbidity. Given the rapid evolution of gene therapy technologies and their potential for treating inherited diseases, this review explores the therapeutic prospects and challenges in applying these technologies to PKD. Recent findings Significant progress has been made in understanding the genetic underpinnings of PKD, making it a prime candidate for gene therapy. Re-expression of the PKD genes, treatment with the C-terminal tail of polycystin 1 protein and antagomir therapy against miR-17 have shown promise in reducing cyst formation and preserving kidney function. The rapid development of gene-editing tools, antisense oligonucleotide-based strategies, programmable RNA, and advanced gene delivery systems has opened new possibilities for PKD treatment. However, challenges such as off-target effects, delivery efficiency, and long-term safety remain significant barriers to clinical application. Summary Current research highlights the transformative potential of gene therapy for PKD. Ongoing studies are crucial to overcoming existing challenges and translating these findings into clinical practice. We highlight the need for multidisciplinary efforts to optimize gene-editing technologies and ensure their safety and efficacy in treating PKD.

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Advances in miniature CRISPR-Cas proteins and their applications in gene editing

Cited by 3 publications

References 50 publications

A route to engineer a genome editor for gene therapy

A route to engineer a genome editor for gene therapy

Impact of Chromatin Organization and Epigenetics on CRISPR-Cas and TALEN Genome Editing

Prospects for gene therapy in polycystic kidney disease

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