Genome editing has been attracting increasing attention as a new treatment for several refractory diseases since the CRISPR-Cas discovery has facilitated easy modification of target chromosomal DNA. The concept of treating refractory diseases by genome editing has been achieved in various animal models, and genome editing has been applied to human clinical trials for β-thalassemia, sickle cell disease, mucopolysaccharidosis, transthyretin amyloidosis, HIV infection, and CAR-T therapy. The genome editing technology targets the germline in industrial applications in animals and plants and is directed at the chromosomal DNA of the somatic cells in human therapeutic applications. Genome editing therapy for germline cells is currently forbidden due to ethical and safety concerns. Concerns regarding genome editing technology include safety (off-target effects) as well as technical aspects (low homologous recombination). Various technological innovations for genome editing are expected to expand its clinical application to various diseases in the future.
Download full-text PDF |
Source |
|---|---|
| http://dx.doi.org/10.11406/rinketsu.63.1558 | DOI Listing |
Publication Analysis
Top Keywords
Similar Publications
Crystal structure of the anti-CRISPR protein AcrIE7.
Biochem Biophys Res Commun
January 2025
Department of Biochemistry and Molecular Biology, School of Basic Medical Sciences, Tianjin Medical University, Tianjin, 300070, China; Department of Pharmacology, School of Basic Medical Sciences, Tianjin Medical University, Tianjin, 300070, China. Electronic address:
Bacterial adaptive immunity, driven by CRISPR-Cas systems, protects against foreign nucleic acids from mobile genetic elements (MGEs), like bacteriophages. The type I-E CRISPR-Cas system employs the Cascade (CRISPR-associated complex for antiviral defense) complex for target DNA cleavage, guided by crRNA. Anti-CRISPR (Acr) proteins, such as AcrIE7, counteract this defense by inhibiting Cascade activity.
View Article and Find Full Text PDFGalactinol synthase 4 influences plant height by affecting phenylpropanoid metabolism and the balance of soluble carbohydrates in tomato.
Plant Physiol Biochem
January 2025
Modern Protected Horticulture Engineering & Technology Center, College of Horticulture, Shenyang Agricultural University, China; National & Local Joint Engineering Research Center of Northern Horticultural Facilities Design & Application Technology (Liaoning), Shenyang, China; Key Laboratory of Protected Horticulture (Shenyang Agricultural University), Ministry of Education, Shenyang, China; Key Laboratory of Horticultural Equipment, Ministry of Agriculture and Rural Affairs, Shenyang, China. Electronic address:
Plant height is a key trait that significantly influences plant architecture, disease resistance, adaptability to mechanical cultivation, and overall economic yield. Galactinol synthase (GolS) is a crucial enzyme involved in the biosynthesis of raffinose family oligosaccharides (RFOs). It plays a significant role in carbohydrate transport and storage, combating abiotic and biotic stresses, and regulating plant growth and development.
View Article and Find Full Text PDFMolecular advances in research and applications of male sterility systems in tomato.
Plant Physiol Biochem
January 2025
Department of Vegetable Science, Institute of Agricultural Sciences, Siksha 'O' Anusandhan (Deemed to be University), Bhubaneswar, Odisha, 751029, India.
Tomato, belonging to the nightshade family, is globally considered as a model system for classical and molecular genetics, genomics, and reproductive developmental studies. In the current scenario of climate change, hybrid development is among the crucial elements in the genetic improvement of crop plants. The phenomenon of male sterility is a viable approach for ensuring hybrid seed purity and reducing the cost of hybrid seed production.
View Article and Find Full Text PDFPeptide nucleic acids: Recent advancements and future opportunities in biomedical applications.
Bioorg Chem
January 2025
Department of Chemistry, Sarojini Naidu College for Women, Kolkata 700028, India. Electronic address:
Peptide nucleic acids (PNA), synthetic molecules comprising a peptide-like backbone and natural and unnatural nucleobases, have garnered significant attention for their potential applications in gene editing and other biomedical fields. The unique properties of PNA, particularly enhanced stability/specificity/affinity towards targeted DNA and RNA sequences, achieved significant attention recently for gene silencing, gene correction, antisense therapy, drug delivery, biosensing and other various diagnostic aspects. This review explores the structure, properties, and potential of PNA in transforming genetic engineering including potent biomedical challenges.
View Article and Find Full Text PDFCRISPR/Cas9-mediated genomic insertion of functional genes into WCFS1.
Microbiol Spectr
January 2025
Faculty of Chemistry, Biotechnology and Food Science, NMBU - Norwegian University of Life Sciences, Ås, Norway.
Unlabelled: a natural inhabitant of the human body, is a promising candidate vehicle for vaccine delivery. An obstacle in developing bacterial delivery vehicles is generating a production strain that lacks antibiotic resistance genes and contains minimal foreign DNA. To deal with this obstacle, we have constructed a finetuned, inducible two-plasmid CRISPR/Cas9-system for chromosomal gene insertion in .
View Article and Find Full Text PDFWant AI Summaries of new PubMed Abstracts delivered to your In-box?
Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!