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34 pages, 1227 KiB  
Review
Beyond Cutting: CRISPR-Driven Synthetic Biology Toolkit for Next-Generation Microalgal Metabolic Engineering
by Limin Yang and Qian Lu
Int. J. Mol. Sci. 2025, 26(15), 7470; https://doi.org/10.3390/ijms26157470 (registering DOI) - 2 Aug 2025
Abstract
Microalgae, with their unparalleled capabilities for sunlight-driven growth, CO2 fixation, and synthesis of diverse high-value compounds, represent sustainable cell factories for a circular bioeconomy. However, industrial deployment has been hindered by biological constraints and the inadequacy of conventional genetic tools. The advent [...] Read more.
Microalgae, with their unparalleled capabilities for sunlight-driven growth, CO2 fixation, and synthesis of diverse high-value compounds, represent sustainable cell factories for a circular bioeconomy. However, industrial deployment has been hindered by biological constraints and the inadequacy of conventional genetic tools. The advent of CRISPR-Cas systems initially provided precise gene editing via targeted DNA cleavage. This review argues that the true transformative potential lies in moving decisively beyond cutting to harness CRISPR as a versatile synthetic biology “Swiss Army Knife”. We synthesize the rapid evolution of CRISPR-derived tools—including transcriptional modulators (CRISPRa/i), epigenome editors, base/prime editors, multiplexed systems, and biosensor-integrated logic gates—and their revolutionary applications in microalgal engineering. These tools enable tunable gene expression, stable epigenetic reprogramming, DSB-free nucleotide-level precision editing, coordinated rewiring of complex metabolic networks, and dynamic, autonomous control in response to environmental cues. We critically evaluate their deployment to enhance photosynthesis, boost lipid/biofuel production, engineer high-value compound pathways (carotenoids, PUFAs, proteins), improve stress resilience, and optimize carbon utilization. Persistent challenges—species-specific tool optimization, delivery efficiency, genetic stability, scalability, and biosafety—are analyzed, alongside emerging solutions and future directions integrating AI, automation, and multi-omics. The strategic integration of this CRISPR toolkit unlocks the potential to engineer robust, high-productivity microalgal cell factories, finally realizing their promise as sustainable platforms for next-generation biomanufacturing. Full article
(This article belongs to the Special Issue Developing Methods and Molecular Basis in Plant Biotechnology)
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17 pages, 814 KiB  
Review
Macrobrachium rosenbergii Genome Editing Breeding with CRISPR–Cas Nucleases, Base Editors, and Prime Editors
by Guo Li, Xinzhi Zhou, Guanglin Zhu, Yingjia Pan, Junjun Yan, Jilun Meng, Tiantian Ye, Yaxian Cheng, Cui Liu and Zhimin Gu
Animals 2025, 15(15), 2161; https://doi.org/10.3390/ani15152161 - 22 Jul 2025
Viewed by 449
Abstract
This review focuses on CRISPR genome editing technology, particularly its application in the study of Macrobrachium rosenbergii (M. rosenbergii). It first elaborates on the basic principles and mechanisms of CRISPR–Cas9 technology, base editors, and prime editors. Then, it explores the application [...] Read more.
This review focuses on CRISPR genome editing technology, particularly its application in the study of Macrobrachium rosenbergii (M. rosenbergii). It first elaborates on the basic principles and mechanisms of CRISPR–Cas9 technology, base editors, and prime editors. Then, it explores the application of this technology in M. rosenbergii breeding, including improving growth rate, enhancing disease resistance, and sex control. Additionally, it introduces the progress of genome editing technology in M. rosenbergii, epidemiology and pathogenesis, diagnostic techniques, analyzes the opportunities and challenges it faces, reviews the historical evolution, and looks ahead to future development directions. CRISPR technology has brought new opportunities to the research and industrial development of M. rosenbergii, but it also needs to address numerous technical and safety challenges. Full article
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14 pages, 2794 KiB  
Article
Comprehensive Analysis of Ghd7 Variations Using Pan-Genomics and Prime Editing in Rice
by Jiarui Wang, Shihang Liu, Jisong Pu, Jun Li, Changcai He, Lanjing Zhang, Xu Zhou, Dongyu Xu, Luyao Zhou, Yuting Guo, Yuxiu Zhang, Yang Wang, Bin Yang, Pingrong Wang, Xiaojian Deng and Changhui Sun
Genes 2025, 16(4), 462; https://doi.org/10.3390/genes16040462 - 17 Apr 2025
Viewed by 584
Abstract
The Ghd7 gene in rice plays a crucial role in determining heading date, plant height, and grain yield. However, the variations in Ghd7 and their functional implications across different rice accessions are not fully understood. Based on the release of a large amount [...] Read more.
The Ghd7 gene in rice plays a crucial role in determining heading date, plant height, and grain yield. However, the variations in Ghd7 and their functional implications across different rice accessions are not fully understood. Based on the release of a large amount of rice genome data in recent years, we investigated Ghd7 through pan-genome analysis of 372 diverse rice varieties and figured out the structural variations (SVs) in the Ghd7 locus. However, due to the high cost of pan-genomes, most genomes are based on next-generation sequencing (NGS) data now. Therefore, we developed a method for identifying SVs using NGS data and Polymerase Chain Reaction (PCR) based on the results of pan-genome analysis and identified 977 accessions carrying such SVs of Ghd7. Furthermore, we identified 46 single-nucleotide polymorphisms (SNPs) and one insertion-deletion (InDel) in the coding region of Ghd7. They are classified into 49 haplotypes. Notably, a splice-site mutation in haplotype H6 causes aberrant mRNA splicing. Using prime editing (PE) technology, we successfully restored the functional of Ghd7 in Yixiang 1B (YX1B), delaying the heading date by approximately 16 days. This modification synchronized the heading date between YX1B and the restorer line Yahui 2115 (YH2115R), enhancing the hybrid rice seed production efficiency. In conclusion, our findings highlight the potential of integrating pan-genomics and precision gene editing to accelerate crop improvement and enhance agronomic traits. Full article
(This article belongs to the Collection Feature Papers: 'Plant Genetics and Genomics' Section)
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15 pages, 1623 KiB  
Review
Current Advancement and Future Prospects in Simplified Transformation-Based Plant Genome Editing
by Xueying Han, Zhaolong Deng, Huiyun Liu and Xiang Ji
Plants 2025, 14(6), 889; https://doi.org/10.3390/plants14060889 - 12 Mar 2025
Cited by 2 | Viewed by 2226
Abstract
Recent years have witnessed remarkable progress in plant biology, driven largely by the rapid evolution of CRISPR/Cas-based genome editing (GE) technologies. These tools, including versatile CRISPR/Cas systems and their derivatives, such as base editors and prime editors, have significantly enhanced the universality, efficiency, [...] Read more.
Recent years have witnessed remarkable progress in plant biology, driven largely by the rapid evolution of CRISPR/Cas-based genome editing (GE) technologies. These tools, including versatile CRISPR/Cas systems and their derivatives, such as base editors and prime editors, have significantly enhanced the universality, efficiency, and convenience of plant functional genomics, genetics, and molecular breeding. However, traditional genetic transformation methods are essential for obtaining GE plants. These methods depend on tissue culture procedures, which are time-consuming, labor-intensive, genotype-dependent, and challenging to regenerate. Here, we systematically outline current advancements in simplifying plant GE, focusing on the optimization of tissue culture process through developmental regulators, the development of in planta transformation methods, and the establishment of nanomaterial- and viral vector-based delivery platforms. We also discuss critical challenges and future directions for achieving genotype-independent, tissue culture-free plant GE. Full article
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43 pages, 7604 KiB  
Review
Prime Editing: Mechanistic Insights and DNA Repair Modulation
by Astrid Mentani, Marcello Maresca and Anna Shiriaeva
Cells 2025, 14(4), 277; https://doi.org/10.3390/cells14040277 - 13 Feb 2025
Cited by 2 | Viewed by 3244
Abstract
Prime editing is a genome editing technique that allows precise modifications of cellular DNA without relying on donor DNA templates. Recently, several different prime editor proteins have been published in the literature, relying on single- or double-strand breaks. When prime editing occurs, the [...] Read more.
Prime editing is a genome editing technique that allows precise modifications of cellular DNA without relying on donor DNA templates. Recently, several different prime editor proteins have been published in the literature, relying on single- or double-strand breaks. When prime editing occurs, the DNA undergoes one of several DNA repair pathways, and these processes can be modulated with the use of inhibitors. Firstly, this review provides an overview of several DNA repair mechanisms and their modulation by known inhibitors. In addition, we summarize different published prime editors and provide a comprehensive overview of associated DNA repair mechanisms. Finally, we discuss the delivery and safety aspects of prime editing. Full article
(This article belongs to the Special Issue Gene Therapy for Rare Diseases)
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27 pages, 12325 KiB  
Article
Optimized Prime Editing of Human Induced Pluripotent Stem Cells to Efficiently Generate Isogenic Models of Mendelian Diseases
by Rodrigo Cerna-Chavez, Alba Ortega-Gasco, Hafiz Muhammad Azhar Baig, Nathan Ehrenreich, Thibaud Metais, Michael J. Scandura, Kinga Bujakowska, Eric A. Pierce and Marcela Garita-Hernandez
Int. J. Mol. Sci. 2025, 26(1), 114; https://doi.org/10.3390/ijms26010114 - 26 Dec 2024
Cited by 1 | Viewed by 3039
Abstract
Prime editing (PE) is a CRISPR-based tool for genome engineering that can be applied to generate human induced pluripotent stem cell (hiPSC)-based disease models. PE technology safely introduces point mutations, small insertions, and deletions (indels) into the genome. It uses a Cas9-nickase (nCas9) [...] Read more.
Prime editing (PE) is a CRISPR-based tool for genome engineering that can be applied to generate human induced pluripotent stem cell (hiPSC)-based disease models. PE technology safely introduces point mutations, small insertions, and deletions (indels) into the genome. It uses a Cas9-nickase (nCas9) fused to a reverse transcriptase (RT) as an editor and a PE guide RNA (pegRNA), which introduces the desired edit with great precision without creating double-strand breaks (DSBs). PE leads to minimal off-targets or indels when introducing single-strand breaks (SSB) in the DNA. Low efficiency can be an obstacle to its use in hiPSCs, especially when the genetic context precludes the screening of multiple pegRNAs, and other strategies must be employed to achieve the desired edit. We developed a PE platform to efficiently generate isogenic models of Mendelian disorders. We introduced the c.25G>A (p.V9M) mutation in the NMNAT1 gene with over 25% efficiency by optimizing the PE workflow. Using our optimized system, we generated other isogenic models of inherited retinal diseases (IRDs), including the c.1481C>T (p.T494M) mutation in PRPF3 and the c.6926A>C (p.H2309P) mutation in PRPF8. We modified several determinants of the hiPSC PE procedure, such as plasmid concentrations, PE component ratios, and delivery method settings, showing that our improved workflow increased the hiPSC editing efficiency. Full article
(This article belongs to the Special Issue Molecular Research in Retinal Degeneration)
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15 pages, 1830 KiB  
Article
Precise Insertion of AttB Sequences in Goat Genome Using Enhanced Prime Editor
by Aicong Li, Zhenliang Zhu, Jing Yang, Yayi Liu, Yong Zhang and Jun Liu
Int. J. Mol. Sci. 2024, 25(17), 9486; https://doi.org/10.3390/ijms25179486 - 31 Aug 2024
Viewed by 1447
Abstract
Prime editor, an editing tool based on the CRISPR/Cas9 system, allows for all 12 types of nucleotide exchanges and arbitrary indels in genomic sequences without the need for inducing DNA double-strand breaks. Despite its flexibility and precision, prime editing efficiency is still low [...] Read more.
Prime editor, an editing tool based on the CRISPR/Cas9 system, allows for all 12 types of nucleotide exchanges and arbitrary indels in genomic sequences without the need for inducing DNA double-strand breaks. Despite its flexibility and precision, prime editing efficiency is still low and hindered by various factors such as target sites, editing types, and the length of the primer binding site. In this study, we developed a prime editing system by incorporating an RNA motif at the 3′ terminal of the pegRNA and integrating all twin prime editor factors into a single plasmid. These two strategies enhanced prime editing efficiency at target sites by up to 3.58-fold and 2.19-fold, respectively. Subsequently, enhanced prime editor was employed in goat cells and embryos to efficiently insert a 38 bp attB sequence into the Gt(ROSA)26Sor (Rosa26) and C-C motif chemokine receptor 5 (CCR5) loci. The enhanced prime editor can mediate 11.9% and 6.8% editing efficiency in parthenogenetic activation of embryos through embryo microinjection. In summary, our study introduces a modified prime editing system with improved editing and transfection efficiency, making it more suitable for inserting foreign sequences into primary cells and embryos. These results broaden the potential applications of prime editing technologies in the production of transgenic animals. Full article
(This article belongs to the Section Molecular Genetics and Genomics)
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42 pages, 5459 KiB  
Review
Emerging Perspectives on Prime Editor Delivery to the Brain
by Eli BenDavid, Sina Ramezanian, Yaoyao Lu, Joël Rousseau, Avi Schroeder, Marc Lavertu and Jacques P. Tremblay
Pharmaceuticals 2024, 17(6), 763; https://doi.org/10.3390/ph17060763 - 11 Jun 2024
Cited by 2 | Viewed by 5047
Abstract
Prime editing shows potential as a precision genome editing technology, as well as the potential to advance the development of next-generation nanomedicine for addressing neurological disorders. However, turning in prime editors (PEs), which are macromolecular complexes composed of CRISPR/Cas9 nickase fused with a [...] Read more.
Prime editing shows potential as a precision genome editing technology, as well as the potential to advance the development of next-generation nanomedicine for addressing neurological disorders. However, turning in prime editors (PEs), which are macromolecular complexes composed of CRISPR/Cas9 nickase fused with a reverse transcriptase and a prime editing guide RNA (pegRNA), to the brain remains a considerable challenge due to physiological obstacles, including the blood–brain barrier (BBB). This review article offers an up-to-date overview and perspective on the latest technologies and strategies for the precision delivery of PEs to the brain and passage through blood barriers. Furthermore, it delves into the scientific significance and possible therapeutic applications of prime editing in conditions related to neurological diseases. It is targeted at clinicians and clinical researchers working on advancing precision nanomedicine for neuropathologies. Full article
(This article belongs to the Special Issue Pharmacotherapy of Neurodegeneration Disorders)
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11 pages, 792 KiB  
Review
The Development, Optimization and Future of Prime Editing
by Irina O. Petrova and Svetlana A. Smirnikhina
Int. J. Mol. Sci. 2023, 24(23), 17045; https://doi.org/10.3390/ijms242317045 - 1 Dec 2023
Cited by 7 | Viewed by 6822
Abstract
Prime editing is a rapidly developing method of CRISPR/Cas-based genome editing. The increasing number of novel PE applications and improved versions demands constant analysis and evaluation. The present review covers the mechanism of prime editing, the optimization of the method and the possible [...] Read more.
Prime editing is a rapidly developing method of CRISPR/Cas-based genome editing. The increasing number of novel PE applications and improved versions demands constant analysis and evaluation. The present review covers the mechanism of prime editing, the optimization of the method and the possible next step in the evolution of CRISPR/Cas9-associated genome editing. The basic components of a prime editing system are a prime editor fusion protein, consisting of nickase and reverse transcriptase, and prime editing guide RNA, consisting of a protospacer, scaffold, primer binding site and reverse transcription template. Some prime editing systems include other parts, such as additional RNA molecules. All of these components were optimized to achieve better efficiency for different target organisms and/or compactization for viral delivery. Insights into prime editing mechanisms allowed us to increase the efficiency by recruiting mismatch repair inhibitors. However, the next step in prime editing evolution requires the incorporation of new mechanisms. Prime editors combined with integrases allow us to combine the precision of prime editing with the target insertion of large, several-kilobase-long DNA fragments. Full article
(This article belongs to the Special Issue Gene Editing for Disease Modeling and Therapeutics)
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15 pages, 887 KiB  
Review
Evolution of CRISPR/Cas Systems for Precise Genome Editing
by Magdalena Hryhorowicz, Daniel Lipiński and Joanna Zeyland
Int. J. Mol. Sci. 2023, 24(18), 14233; https://doi.org/10.3390/ijms241814233 - 18 Sep 2023
Cited by 28 | Viewed by 8212
Abstract
The bacteria-derived CRISPR/Cas (an acronym for regularly interspaced short palindromic repeats/CRISPR-associated protein) system is currently the most widely used, versatile, and convenient tool for genome engineering. CRISPR/Cas-based technologies have been applied to disease modeling, gene therapies, transcriptional modulation, and diagnostics. Nevertheless, some challenges [...] Read more.
The bacteria-derived CRISPR/Cas (an acronym for regularly interspaced short palindromic repeats/CRISPR-associated protein) system is currently the most widely used, versatile, and convenient tool for genome engineering. CRISPR/Cas-based technologies have been applied to disease modeling, gene therapies, transcriptional modulation, and diagnostics. Nevertheless, some challenges remain, such as the risk of immunological reactions or off-target effects. To overcome these problems, many new methods and CRISPR/Cas-based tools have been developed. In this review, we describe the current classification of CRISPR systems and new precise genome-editing technologies, summarize the latest applications of this technique in several fields of research, and, finally, discuss CRISPR/Cas system limitations, ethical issues, and challenges. Full article
(This article belongs to the Special Issue CRISPR-Cas Systems and Genome Editing)
24 pages, 1163 KiB  
Review
Progress and Prospects of Gene Editing in Pluripotent Stem Cells
by Zhenwu Zhang, Xinyu Bao and Chao-Po Lin
Biomedicines 2023, 11(8), 2168; https://doi.org/10.3390/biomedicines11082168 - 1 Aug 2023
Cited by 9 | Viewed by 4921
Abstract
Applying programmable nucleases in gene editing has greatly shaped current research in basic biology and clinical translation. Gene editing in human pluripotent stem cells (PSCs), including embryonic stem cells (ESCs) and induced pluripotent stem cells (iPSCs), is highly relevant to clinical cell therapy [...] Read more.
Applying programmable nucleases in gene editing has greatly shaped current research in basic biology and clinical translation. Gene editing in human pluripotent stem cells (PSCs), including embryonic stem cells (ESCs) and induced pluripotent stem cells (iPSCs), is highly relevant to clinical cell therapy and thus should be examined with particular caution. First, since all mutations in PSCs will be carried to all their progenies, off-target edits of editors will be amplified. Second, due to the hypersensitivity of PSCs to DNA damage, double-strand breaks (DSBs) made by gene editing could lead to low editing efficiency and the enrichment of cell populations with defective genomic safeguards. In this regard, DSB-independent gene editing tools, such as base editors and prime editors, are favored due to their nature to avoid these consequences. With more understanding of the microbial world, new systems, such as Cas-related nucleases, transposons, and recombinases, are also expanding the toolbox for gene editing. In this review, we discuss current applications of programmable nucleases in PSCs for gene editing, the efforts researchers have made to optimize these systems, as well as new tools that can be potentially employed for differentiation modeling and therapeutic applications. Full article
(This article belongs to the Special Issue Pluripotent Stem Cell: Current Understanding and Future Directions)
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39 pages, 2843 KiB  
Review
CRISPR-Cas System: The Current and Emerging Translational Landscape
by Narendranath Bhokisham, Ethan Laudermilch, Lindsay L. Traeger, Tonya D. Bonilla, Mercedes Ruiz-Estevez and Jordan R. Becker
Cells 2023, 12(8), 1103; https://doi.org/10.3390/cells12081103 - 7 Apr 2023
Cited by 19 | Viewed by 16949
Abstract
CRISPR-Cas technology has rapidly changed life science research and human medicine. The ability to add, remove, or edit human DNA sequences has transformative potential for treating congenital and acquired human diseases. The timely maturation of the cell and gene therapy ecosystem and its [...] Read more.
CRISPR-Cas technology has rapidly changed life science research and human medicine. The ability to add, remove, or edit human DNA sequences has transformative potential for treating congenital and acquired human diseases. The timely maturation of the cell and gene therapy ecosystem and its seamless integration with CRISPR-Cas technologies has enabled the development of therapies that could potentially cure not only monogenic diseases such as sickle cell anemia and muscular dystrophy, but also complex heterogenous diseases such as cancer and diabetes. Here, we review the current landscape of clinical trials involving the use of various CRISPR-Cas systems as therapeutics for human diseases, discuss challenges, and explore new CRISPR-Cas-based tools such as base editing, prime editing, CRISPR-based transcriptional regulation, CRISPR-based epigenome editing, and RNA editing, each promising new functionality and broadening therapeutic potential. Finally, we discuss how the CRISPR-Cas system is being used to understand the biology of human diseases through the generation of large animal disease models used for preclinical testing of emerging therapeutics. Full article
(This article belongs to the Special Issue CRISPR-Based Genome Editing in Translational Research)
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15 pages, 994 KiB  
Perspective
Current Bioinformatics Tools to Optimize CRISPR/Cas9 Experiments to Reduce Off-Target Effects
by Muhammad Naeem and Omer S. Alkhnbashi
Int. J. Mol. Sci. 2023, 24(7), 6261; https://doi.org/10.3390/ijms24076261 - 27 Mar 2023
Cited by 48 | Viewed by 8378
Abstract
The CRISPR-Cas system has evolved into a cutting-edge technology that has transformed the field of biological sciences through precise genetic manipulation. CRISPR/Cas9 nuclease is evolving into a revolutionizing method to edit any gene of any species with desirable outcomes. The swift advancement of [...] Read more.
The CRISPR-Cas system has evolved into a cutting-edge technology that has transformed the field of biological sciences through precise genetic manipulation. CRISPR/Cas9 nuclease is evolving into a revolutionizing method to edit any gene of any species with desirable outcomes. The swift advancement of CRISPR-Cas technology is reflected in an ever-expanding ecosystem of bioinformatics tools designed to make CRISPR/Cas9 experiments easier. To assist researchers with efficient guide RNA designs with fewer off-target effects, nuclease target site selection, and experimental validation, bioinformaticians have built and developed a comprehensive set of tools. In this article, we will review the various computational tools available for the assessment of off-target effects, as well as the quantification of nuclease activity and specificity, including web-based search tools and experimental methods, and we will describe how these tools can be optimized for gene knock-out (KO) and gene knock-in (KI) for model organisms. We also discuss future directions in precision genome editing and its applications, as well as challenges in target selection, particularly in predicting off-target effects. Full article
(This article belongs to the Special Issue RNA-Targeting CRISPR Systems)
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13 pages, 1476 KiB  
Article
Counteracting the Common Shwachman–Diamond Syndrome-Causing SBDS c.258+2T>C Mutation by RNA Therapeutics and Base/Prime Editing
by Laura Peretto, Elena Tonetto, Iva Maestri, Valentino Bezzerri, Roberto Valli, Marco Cipolli, Mirko Pinotti and Dario Balestra
Int. J. Mol. Sci. 2023, 24(4), 4024; https://doi.org/10.3390/ijms24044024 - 16 Feb 2023
Cited by 7 | Viewed by 3581
Abstract
Shwachman–Diamond syndrome (SDS) represents one of the most common inherited bone marrow failure syndromes and is mainly caused by SBDS gene mutations. Only supportive treatments are available, with hematopoietic cell transplantation required when marrow failure occurs. Among all causative mutations, the SBDS c.258+2T>C [...] Read more.
Shwachman–Diamond syndrome (SDS) represents one of the most common inherited bone marrow failure syndromes and is mainly caused by SBDS gene mutations. Only supportive treatments are available, with hematopoietic cell transplantation required when marrow failure occurs. Among all causative mutations, the SBDS c.258+2T>C variant at the 5′ splice site (ss) of exon 2 is one of the most frequent. Here, we investigated the molecular mechanisms underlying aberrant SBDS splicing and showed that SBDS exon 2 is dense in splicing regulatory elements and cryptic splice sites, complicating proper 5′ss selection. Studies ex vivo and in vitro demonstrated that the mutation alters splicing, but it is also compatible with tiny amounts of correct transcripts, which would explain the survival of SDS patients. Moreover, for the first time for SDS, we explored a panel of correction approaches at the RNA and DNA levels and provided experimental evidence that the mutation effect can be partially counteracted by engineered U1snRNA, trans-splicing, and base/prime editors, ultimately leading to correctly spliced transcripts (from barely detectable to 2.5–5.5%). Among them, we propose DNA editors that, by stably reverting the mutation and potentially conferring positive selection to bone-marrow cells, could lead to the development of an innovative SDS therapy. Full article
(This article belongs to the Special Issue Advances in Gene and Cell Therapy)
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24 pages, 1837 KiB  
Review
Future Perspectives of Prime Editing for the Treatment of Inherited Retinal Diseases
by Silja Hansen, Michelle E. McClements, Thomas J. Corydon and Robert E. MacLaren
Cells 2023, 12(3), 440; https://doi.org/10.3390/cells12030440 - 29 Jan 2023
Cited by 17 | Viewed by 7342
Abstract
Inherited retinal diseases (IRD) are a clinically and genetically heterogenous group of diseases and a leading cause of blindness in the working-age population. Even though gene augmentation therapies have shown promising results, they are only feasible to treat a small number of autosomal [...] Read more.
Inherited retinal diseases (IRD) are a clinically and genetically heterogenous group of diseases and a leading cause of blindness in the working-age population. Even though gene augmentation therapies have shown promising results, they are only feasible to treat a small number of autosomal recessive IRDs, because the size of the gene is limited by the vector used. DNA editing however could potentially correct errors regardless of the overall size of the gene and might also be used to correct dominant mutations. Prime editing is a novel CRISPR/Cas9 based gene editing tool that enables precise correction of point mutations, insertions, and deletions without causing double strand DNA breaks. Due to its versatility and precision this technology may be a potential treatment option for virtually all genetic causes of IRD. Since its initial description, the prime editing technology has been further improved, resulting in higher efficacy and a larger target scope. Additionally, progress has been achieved concerning the size-related delivery issue of the prime editor components. This review aims to give an overview of these recent advancements and discusses prime editing as a potential treatment for IRDs. Full article
(This article belongs to the Special Issue CRISPR-Based Genome Editing in Translational Research)
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