CRISPR/Cas system and its application in the diagnosis of animal infectious diseases DOI Creative Commons
Hafiz Muhammad Hamza Rasool, Qiwei Chen, Xiaowei Gong

et al.

The FASEB Journal, Journal Year: 2024, Volume and Issue: 38(24)

Published: Dec. 13, 2024

Abstract Infectious diseases are a serious threat to the existence of animals and humans' life. In 21st century, emergence re‐emergence several zoonotic non‐zoonotic global pandemic socio‐economic importance has affected billions humans animals. The need for expensive equipment laboratories, non‐availability on‐site testing abilities, with time‐consuming low sensitivity specificity issues currently available diagnostic techniques identify these pathogenic micro‐organisms on large scale highlighted developing cheap, portable environment friendly methods. recent years, have been addressed by clustered regularly interspaced palindromic repeats (CRISPR)‐based platforms that transformed molecular field due their outstanding ultra‐sensitive nucleic acid detecting capabilities. this study, we highlight types, potential different Cas proteins, amplification systems. We also illuminate application CRISPR integrated setups diagnosis infectious diseases, majorly in food‐producing (pigs, ruminants, poultry, aquaculture), domestic pets (dogs cats), importance. conclude challenges future perspectives using systems rapidly diagnose treat other develop control strategies prevent spread organisms.

Language: Английский

What is the role of microbial biotechnology and genetic engineering in medicine? DOI Creative Commons
Fernando Santos‐Beneit

MicrobiologyOpen, Journal Year: 2024, Volume and Issue: 13(2)

Published: March 31, 2024

Abstract Microbial products are essential for developing various therapeutic agents, including antibiotics, anticancer drugs, vaccines, and enzymes. Genetic engineering techniques, functional genomics, synthetic biology unlock previously uncharacterized natural products. This review highlights major advances in microbial biotechnology, focusing on gene‐based technologies medical applications.

Language: Английский

Citations

15

Recent Therapeutic Gene Editing Applications to Genetic Disorders DOI Creative Commons
Éric Deneault

Current Issues in Molecular Biology, Journal Year: 2024, Volume and Issue: 46(5), P. 4147 - 4185

Published: April 30, 2024

Recent years have witnessed unprecedented progress in therapeutic gene editing, revolutionizing the approach to treating genetic disorders. In this comprehensive review, we discuss progression of milestones leading emergence clustered regularly interspaced short palindromic repeats (CRISPR)-based technology as a powerful tool for precise and targeted modifications human genome. CRISPR-Cas9 nuclease, base prime editing taken center stage, demonstrating remarkable precision efficacy ex vivo genomic modifications. Enhanced delivery systems, including viral vectors nanoparticles, further improved efficiency safety advancing their clinical translatability. The exploration CRISPR-Cas systems beyond commonly used Cas9, such development Cas12 Cas13 variants, has expanded repertoire tools, enabling more intricate interventions. Outstandingly, represents significant leap forward, given its unparalleled versatility minimization off-target effects. These innovations paved way multitude previously incurable disorders, ranging from monogenic diseases complex polygenic conditions. This review highlights latest innovative studies field, emphasizing breakthrough technologies preclinical trials, applications realm medicine. However, challenges effects ethical considerations remain, necessitating continued research refine profiles frameworks.

Language: Английский

Citations

13

Muscle‐specific gene editing improves molecular and phenotypic defects in a mouse model of myotonic dystrophy type 1 DOI Creative Commons
Mariapaola Izzo, Jonathan Battistini,

Elisabetta Golini

et al.

Clinical and Translational Medicine, Journal Year: 2025, Volume and Issue: 15(2)

Published: Feb. 1, 2025

Abstract Background Myotonic dystrophy type 1 (DM1) is a genetic multisystemic disease, characterised by pleiotropic symptoms that exhibit notable variability in severity, nature and age of onset. The cause DM1 the expansion unstable CTG‐repeats 3′ untranslated region (UTR) DMPK gene, resulting accumulation toxic CUG‐transcripts sequester RNA‐binding proteins form nuclear foci affected tissues and, consequently, alter various cellular processes. Therapeutic gene editing for treatment monogenic diseases powerful technology could principle remove definitively disease‐causing defect. precision efficiency molecular mechanisms are still under investigation view possible use clinical practice. Methods Here, we describe application clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR‐associated protein 9 (Cas9) strategy to CTG‐expansion mouse model carrying human transgene from patient. To optimise vivo, identified new tools allowed improve expression levels activity CRISPR/Cas9 machinery. Newly designed guide RNA pairs were tested DM1‐patient derived cells before vivo application. Edited analysed assess occurrence off‐target accuracy on‐target genomic events. Gene editing‐dependent ‐independent leading decreased mutated transcripts also evaluated. Results Conclusion Systemic delivery components mice, through myotropic adeno‐associated viral vectors, led significant improvement alterations heart skeletal muscle. Importantly, persistent increase body weight, muscle strength composition parameters observed treated animals. Accurate evaluation CRISPR/Cas9‐mediated‐phenotypic recovery crucial preclinical step development therapy patients. Key points In therapeutic permanent deletion pathogenetic CTG‐repeat amplification causes myotonic 1. Following treatment, diseased mice show both phenotypic defects.

Language: Английский

Citations

2

Engineering Useful Microbial Species for Pharmaceutical Applications DOI Creative Commons
А.К. САДАНОВ, B.B. BAIMAKHANOVA,

Saltanat Emilievna Orasymbet

et al.

Microorganisms, Journal Year: 2025, Volume and Issue: 13(3), P. 599 - 599

Published: March 5, 2025

Microbial engineering has made a significant breakthrough in pharmaceutical biotechnology, greatly expanding the production of biologically active compounds, therapeutic proteins, and novel drug candidates. Recent advancements genetic engineering, synthetic biology, adaptive evolution have contributed to optimization microbial strains for applications, playing crucial role enhancing their productivity stability. The CRISPR-Cas system is widely utilized as precise genome modification tool, enabling enhancement metabolite biosynthesis activation biological pathways. Additionally, biology approaches allow targeted design microorganisms with improved metabolic efficiency potential, thereby accelerating development new products. integration artificial intelligence (AI) machine learning (ML) plays vital further advancing by predicting network interactions, optimizing bioprocesses, discovery process. However, challenges such efficient pathways, ensuring sustainable industrial-scale production, meeting international regulatory requirements remain critical barriers field. Furthermore, mitigate potential risks, it essential develop stringent biocontainment strategies implement appropriate oversight. This review comprehensively examines recent innovations analyzing key technological advancements, challenges, future perspectives.

Language: Английский

Citations

2

Emerging applications of gene editing technologies for the development of climate-resilient crops DOI Creative Commons
R. L. Chavhan,

Siddhant Gahininath Jaybhaye,

V. R. Hinge

et al.

Frontiers in Genome Editing, Journal Year: 2025, Volume and Issue: 7

Published: March 10, 2025

Climate change threatens global crop yield and food security due to rising temperatures, erratic rainfall, increased abiotic stresses like drought, heat, salinity. Gene editing technologies, including CRISPR/Cas9, base editors, prime offer precise tools for enhancing resilience. This review explores the mechanisms of these technologies their applications in developing climate-resilient crops address future challenges. While CRISPR/enables targeted modifications plant DNA, editors allow direct conversion without inducing double-stranded breaks, enable insertions, deletions, substitutions. By understanding manipulating key regulator genes involved stress responses, such as DREB, HSP, SOS, ERECTA, HsfA1, NHX; tolerance can be enhanced against salt stress. improve traits related root development, water use efficiency, response pathways, heat shock response, photosynthesis, membrane stability, ion homeostasis, osmotic adjustment, oxidative response. Advancements gene integration with genomics, phenomics, artificial intelligence (AI)/machine learning (ML) hold great promise. However, challenges off-target effects, delivery methods, regulatory barriers must addressed. highlights potential develop crops, contributing sustainable agriculture.

Language: Английский

Citations

1

Mesenchymal Stromal Cells for Aging Cartilage Regeneration: A Review DOI Open Access
Kun‐Chi Wu,

Yu-Hsun Chang,

Dah‐Ching Ding

et al.

International Journal of Molecular Sciences, Journal Year: 2024, Volume and Issue: 25(23), P. 12911 - 12911

Published: Nov. 30, 2024

Cartilage degeneration is a key feature of aging and osteoarthritis, characterized by the progressive deterioration joint function, pain, limited mobility. Current treatments focus on symptom relief, not cartilage regeneration. Mesenchymal stromal cells (MSCs) offer promising therapeutic option due to their capability differentiate into chondrocytes, modulate inflammation, promote tissue This review explores potential MSCs for regeneration, examining biological properties, action mechanisms, applications in preclinical clinical settings. derived from bone marrow, adipose tissue, other sources can self-renew multiple cell types. In cartilage, they aid regeneration secreting growth factors cytokines that enhance repair immune responses. Recent studies show restore integrity, reduce improve although translation remains challenging limitations such as viability, scalability, regulatory concerns. Advancements MSC delivery, including scaffold-based approaches engineered exosomes, may effectiveness. Potential risks, tumorigenicity rejection, are also discussed, emphasizing need optimized treatment protocols large-scale trials develop effective, minimally invasive therapies

Language: Английский

Citations

5

Advancing diabetes management: Exploring pancreatic beta-cell restoration’s potential and challenges DOI
Mona Mohamed Ibrahim Abdalla

World Journal of Gastroenterology, Journal Year: 2024, Volume and Issue: 30(40), P. 4339 - 4353

Published: Oct. 16, 2024

Diabetes mellitus, characterized by chronic hyperglycemia due to insulin deficiency or resistance, poses a significant global health burden. Central its pathogenesis is the dysfunction loss of pancreatic beta cells, which are res-ponsible for production. Recent advances in beta-cell regeneration research offer promising strategies diabetes treatment, aiming restore endogenous production and achieve glycemic control. This review explores physiological basis function, recent scientific advan-cements, challenges translating these findings into clinical applications. It highlights key developments stem cell therapy, gene editing technologies, identification novel regenerative molecules. Despite potential, field faces hurdles such as ensuring safety long-term efficacy regen-erative therapies, ethical concerns around use, complexity differentiation integration. The importance interdisciplinary collaboration, increased funding, need patient-centered approaches integration new treatments comprehensive care overcome challenges. Through continued holds potential revolutionize care, turning condition manageable even curable disease.

Language: Английский

Citations

4

Protocol Development for CRISPR/Cas9 Knockout of the Anti-inflammatory Protein TNIP1 in HaCaT Keratinocytes DOI
Liam E. Carman, Michael L. Samulevich, Brian J. Aneskievich

et al.

Methods in molecular biology, Journal Year: 2025, Volume and Issue: unknown

Published: Jan. 1, 2025

Language: Английский

Citations

0

The Strategy and Application of Gene Attenuation in Metabolic Engineering DOI Creative Commons
Yahui Zhang, Zhaoxia Jin, Linxia Liu

et al.

Microorganisms, Journal Year: 2025, Volume and Issue: 13(4), P. 927 - 927

Published: April 17, 2025

Metabolic engineering has a wide range of applications, spanning key sectors such as energy, pharmaceuticals, agriculture, chemicals, and environmental sustainability. Its core focus is on precisely modulating metabolic pathways to achieve efficient, sustainable, environmentally friendly biomanufacturing processes, offering new possibilities for societal sustainable development. Gene attenuation critical technique within engineering, pivotal in optimizing fluxes improving target metabolite yields. This review article discusses gene mechanisms, the applications across various biological systems, implementation strategies. Additionally, we address potential future challenges explore its drive further advancements field.

Language: Английский

Citations

0

Gasdermin E as a potential target and biomarker for CRISPR-Cas9-based cancer therapy DOI
Mojtaba Tarin, Amir Sh. Saljooghi

Biochemical Pharmacology, Journal Year: 2025, Volume and Issue: unknown, P. 116961 - 116961

Published: April 1, 2025

Language: Английский

Citations

0