Emerging Gene Therapeutics for Epidermolysis Bullosa under Development

International Journal of Molecular Sciences, Год журнала: 2024, Номер 25(4), С. 2243 - 2243

Опубликована: Фев. 13, 2024

The monogenetic disease epidermolysis bullosa (EB) is characterised by the formation of extended blisters and lesions on patient's skin upon minimal mechanical stress. Causal for this severe condition are genetic mutations in genes, leading to functional impairment, reduction, or absence encoded protein within skin's basement membrane zone connecting epidermis underlying dermis. major burden affected families justifies development long-lasting curative therapies operating at genomic level. landscape causal EB steadily expanding due recent breakthroughs gene therapy field, providing promising outcomes patients suffering from disease. Currently, two therapeutic approaches show promise EB. clinically more advanced replacement strategy was successfully applied forms, a ground-breaking vivo product named beremagene geperpavec (B-VEC) recently approved US Food Drug Administration (FDA). In addition, continuous innovations both designer nucleases editing technologies enable efficient potentially safe repair permanent manner, inspiring researchers field define reach new milestones

Язык: Английский

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CRISPR/Cas systems for genomic Editing, biochemical Sensing, Bioanalysis, and diagnostics

Microchemical Journal, Год журнала: 2025, Номер unknown, С. 112638 - 112638

Опубликована: Янв. 1, 2025

Язык: Английский

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Application of CRISPR/Cas-based gene-editing for developing better banana

Frontiers in Bioengineering and Biotechnology, Год журнала: 2024, Номер 12

Опубликована: Авг. 16, 2024

Banana ( Musa spp.), including plantain, is one of the major staple food and cash crops grown in over 140 countries subtropics tropics, with around 153 million tons annual global production, feeding about 400 people. Despite its widespread cultivation adaptability to diverse environments, banana production faces significant challenges from pathogens pests that often coexist within agricultural landscapes. Recent advancements CRISPR/Cas-based gene editing offer transformative solutions enhance resilience productivity. Researchers at IITA, Kenya, have successfully employed confer resistance diseases such as Xanthomonas wilt (BXW) by targeting susceptibility genes streak virus (BSV) disrupting viral sequences. Other breakthroughs include development semi-dwarf plants, increased β-carotene content. Additionally, non-browning been developed reduce waste, regulatory approval Philippines. The future prospects looks promising CRISPR-based activation (CRISPRa) inhibition (CRISPRi) techniques offering potential for improved disease resistance. Cas-CLOVER system provides a precise alternative CRISPR/Cas9, demonstrating success generating gene-edited mutants. Integration precision genetics traditional breeding, adopting transgene-free strategies, will be pivotal harnessing full banana. crop holds exciting producing thrives across agroecological zones offers superior nutritional value, ultimately benefiting farmers consumers. This article highlights role CRISPR/Cas technology advancing resilience, yield quality, implications security.

Язык: Английский

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Gene therapy and gene editing strategies in inherited blood disorders

Journal of genetics and genomics/Journal of Genetics and Genomics, Год журнала: 2024, Номер 51(11), С. 1162 - 1172

Опубликована: Июль 8, 2024

Язык: Английский

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Viral delivery of an RNA-guided genome editor for transgene-free plant germline editing

bioRxiv (Cold Spring Harbor Laboratory), Год журнала: 2024, Номер unknown

Опубликована: Июль 19, 2024

Abstract Genome editing is transforming plant biology by enabling precise DNA modifications. However, delivery of systems into plants remains challenging, often requiring slow, genotype-specific methods such as tissue culture or transformation. Plant viruses, which naturally infect and spread to most tissues, present a promising system for reagents. But viruses have limited cargo capacities, restricting their ability carry large CRISPR-Cas systems. Here, we engineered tobacco rattle virus (TRV) the compact RNA-guided TnpB enzyme ISYmu1 its guide RNA. This innovation allowed transgene-free Arabidopsis thaliana in single step, with edits inherited subsequent generation. By overcoming traditional reagent barriers, this approach offers novel platform genome editing, can greatly accelerate biotechnology basic research.

Язык: Английский

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Efforts to Downsize Base Editors for Clinical Applications

International Journal of Molecular Sciences, Год журнала: 2025, Номер 26(5), С. 2357 - 2357

Опубликована: Март 6, 2025

Since the advent of clustered regularly interspaced short palindromic repeats (CRISPR) system in gene editing field, diverse CRISPR-based tools have been developed for treating genetic diseases. Of these, base editors (BEs) are promising because they can carry out precise at single-nucleotide resolution without inducing DNA double-strand breaks (DSBs), which pose significant risks genomic instability. Despite their outstanding advantages, clinical application BEs remains challenging due to large size, limits efficient delivery, particularly adeno-associated virus (AAV)-based systems. To address this issue, various strategies explored reduce size BEs. These approaches include truncating nonessential domains and replacing bulky components with smaller substitutes compromising efficiency. In review, we highlight importance downsizing therapeutic applications introduce recent advances size-reduction strategies. Additionally, ongoing efforts overcome other limitations BEs, providing insights into potential improving vivo editing.

Язык: Английский

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Unlocking the potential of ‘Egusi’ melon (Colocynthis citrullus L.) as a crop for biotechnological improvement

Frontiers in Plant Science, Год журнала: 2025, Номер 16

Опубликована: Март 20, 2025

‘Egusi’ melon ( Colocynthis citrullus L.) plays a critical role in food security and potential biofuel production West Africa. Its seeds are valued for both their nutritional industrial applications, especially biodiesel production. However, the crop faces significant challenges, including impacts of climate change, water scarcity, declining arable land, increased pressure from pests diseases. These challenges threaten stability may hinder its ability to meet future demand. To address these issues, there is growing need complement conventional breeding methods with biotechnological approaches. Molecular approaches; genomics, transcriptomics, proteomics, metabolomics; have been utilized improvement several cucurbit species. information on molecular very limited. The current review focuses melon, biology, uses, factors affecting improvement, highlights knowledge gaps ‘Egusi’. also examines omics technologies outlines importance genetic transformation genome editing such as CRISPR that could drive development more resilient high-yielding ‘Egusi’varieties will contribute sustainability profitability farming.

Язык: Английский

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Study of tree shrew biology and models: A booming and prosperous field for biomedical research

动物学研究, Год журнала: 2024, Номер 45(4), С. 877 - 909

Опубликована: Янв. 1, 2024

The tree shrew (Tupaia belangeri) has long been proposed as a suitable alternative to non-human primates (NHPs) in biomedical and laboratory research due its close evolutionary relationship with primates. In recent years, significant advances have facilitated studies, including the determination of genome, genetic manipulation using spermatogonial stem cells, viral vector-mediated gene delivery, mapping brain atlas. However, limited availability shrews globally remains substantial challenge field. Additionally, determining key questions best answered constitutes another difficulty. Tree models historically used study hepatitis B virus (HBV) C (HCV) infection, myopia, psychosocial stress-induced depression, more studies focusing on developing animal for infectious neurodegenerative diseases. Despite these efforts, impact not yet matched that rodent or NHP research. This review summarizes prominent advancements reflects biological addressed this model. We emphasize intensive dedication robust international collaboration are essential achieving breakthroughs studies. use unique resource is expected gain considerable attention application advanced techniques development viable models, meeting increasing demands life science

Язык: Английский

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Synthetic biology for Monascus: From strain breeding to industrial production

Biotechnology Journal, Год журнала: 2024, Номер 19(7)

Опубликована: Июль 1, 2024

Traditional Chinese food therapies often motivate the development of modern medicines, and learning from them will bring bright prospects. Monascus, a conventional fungus with centuries use in industry, produces various metabolites, including natural pigments, lipid-lowering substances, other bioactive ingredients. Recent Monascus studies focused on metabolite biosynthesis mechanisms, strain modifications, fermentation process optimizations, significantly advancing lab scale. However, advanced manufacture for is lacking, restricting its scale production. Here, synthetic biology techniques their challenges engineering filamentous fungi were summarized, especially Monascus. With further in-depth discussions automatic solid-state manufacturing prospects combining intensification, industrial production succeed help improvement intelligent control, promoting applications food, cosmetic, agriculture, medicine, environmental protection industries.

Язык: Английский

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RNA-guided genome engineering: paradigm shift towards transposons

Trends in biotechnology, Год журнала: 2024, Номер 42(8), С. 970 - 985

Опубликована: Март 4, 2024

Язык: Английский

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