Multidrug-Resistant tuberculosis

Clinica Chimica Acta, Год журнала: 2024, Номер 559, С. 119701 - 119701

Опубликована: Май 1, 2024

Язык: Английский

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Therapeutic In Vivo Gene Editing Achieved by a Hypercompact CRISPR‐Cas12f1 System Delivered with All‐in‐One Adeno‐Associated Virus

Advanced Science, Год журнала: 2024, Номер 11(19)

Опубликована: Фев. 26, 2024

CRISPR-based gene therapies are making remarkable strides toward the clinic. But large size of most widely used Cas endonucleases including Cas9 and Cas12a restricts their efficient delivery by adeno-associated virus (AAV) for in vivo editing. Being exceptionally small, recently engineered type V-F CRISPR-Cas12f1 systems can overcome cargo packaging bottleneck present as strong candidates therapeutic applications. In this study, pairwise editing efficiencies different Cas12f1/sgRNA scaffold combinations systemically screened optimized, CasMINI_v3.1/ge4.1 system is identified being able to significantly boost activity. Moreover, packaged into single AAV vectors delivered via subretinal injection, achieves remarkably high efficiencies, over 70% transduced retinal cells. Further, efficacy Cas12f1 system-based therapy treat retinitis pigmentosa Rho

Язык: Английский

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Exploring the Role of CRISPR-Cas9 in Genetic Engineering: Advancements, Applications, and Ethical Issue

London journal of interdisciplinary sciences., Год журнала: 2025, Номер 4, С. 38 - 51

Опубликована: Фев. 9, 2025

Since its discovery in 1987, the emerging genome-modification technology CRISPR-Cas9 has augmented ever-evolving field of genetic engineering through advancements precision and accuracy to simplify efficient genome alteration. This paper introduces history explores underlying mechanisms advancements. Significant technological have enhanced efficiency engineering. Innovations like base prime editors minimize unintended off-target effects, improving gene editing. The development advanced delivery methods, such as magnetic nanoparticles, allows for faster editing components their intended destination with greater precision. complex a wide range applications fields medicine, agriculture, industrial biotechnology. recently grown popular among therapy studies disorders addition cancer research further understanding cell mechanisms. In agricultural settings, this tool been used modify crops withstand environmental constraints increase crop yield alter nutritional content. CRISPR-Cas9’s role biotechnology is also discussed modifying metabolic pathways microorganisms facilitate higher biofuel production. Ethical considerations related safety, possible human germline misuse, ecological effects GMOs catalyzed social political restraints pertinent case studies. Challenges generational consequences, unequal access are mentioned. Nevertheless, ethical questions remain without prominent responses. future hands geneticists working offer treatment options fatal disorders. review aims provide better significant use

Язык: Английский

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In vivo electroporation-based chromosomal engineering in murine uterine epithelium

bioRxiv (Cold Spring Harbor Laboratory), Год журнала: 2025, Номер unknown

Опубликована: Март 1, 2025

Abstract Clustered regularly interspaced palindromic repeat (CRISPR)/CRISPR-associated protein (Cas)-based in vivo chromosomal rearrangements are a promising approach for generating model organisms with specific abnormalities. However, conventional methods rely on viral vectors, which expensive, require specialized equipment, and pose potential safety risks, thereby limiting their widespread application. To overcome the limitations above, we developed novel, efficient, cost-effective engineering strategy using CRISPR ribonucleoprotein electroporation murine uterine epithelium. Our method successfully induced translocations at multiple loci repaired 57.8-Mb inversion. The findings of present study establish as practical alternative to traditional provide foundation its broader application genome editing technologies.

Язык: Английский

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Revolutionizing healthcare: the role of CRISPR-Cas systems in precision medicine

Pharmacogenetics and Pharmacogenomics, Год журнала: 2025, Номер 2, С. 29 - 36

Опубликована: Март 4, 2025

Introduction. CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) has been recognized as a revolutionary advancement in the biomedical field, offering unparalleled precision and versatility genome editing. This review examines transformative potential of diagnostic therapeutic tool for various diseases. Methods. A systematic was conducted following PRISMA (Preferred Reporting Items Systematic Reviews Meta-Analyses) guidelines. comprehensive search PubMed, Scopus, Google Scholar, Web Science performed to identify relevant Studies published between January 2015 2025. The inclusion criteria focused on peer-reviewed articles discussing CRISPR-based diagnostics, applications, technological advancements. were screened, assessed quality using CASP framework, categorized into thematic areas analysis. Results. platforms, such SHERLOCK DETECTR, analyzed their sensitivity rapidity detecting pathogens, cancer biomarkers, genetic mutations. Emerging innovations, including prime base editing, have explored role expanding capabilities CRISPR. Additionally, advancements delivery mechanisms use alternative Cas proteins discussed impact clinical applicability. Conclusions. Ethical, regulatory, accessibility challenges associated with technology are highlighted, emphasizing importance responsible development equitable deployment. connects cutting-edge translational underscores significant shaping future medicine global health.

Язык: Английский

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Comprehensive strategies for constructing efficient CRISPR/Cas based cancer therapy: Target gene selection, sgRNA optimization, delivery methods and evaluation

Advances in Colloid and Interface Science, Год журнала: 2025, Номер unknown, С. 103497 - 103497

Опубликована: Март 1, 2025

Язык: Английский

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Advances in Gene Therapy with Oncolytic Viruses and CAR-T Cells and Therapy-Related Groups

Current Issues in Molecular Biology, Год журнала: 2025, Номер 47(4), С. 268 - 268

Опубликована: Апрель 10, 2025

Cancer gene therapy is attracting considerable attention as a new treatment method for overcoming intractable cancers. CAR-T cell has already achieved remarkable results, particularly hematological tumors. Because cells can increase within the body, they have advantage of requiring only single administration. In addition, targeting CD19 antigen been established relapsed or refractory disease in young people with CD19-positive acute B-cell leukemia (B-acute lymphoblastic leukemia, B-ALL) and diffuse large lymphoma (DLBCL). addition to therapy, oncolytic viruses represent promising approach cancer treatment, some clinical use others being researched their potential benefits. These infect kill cells, triggering an immune response that helps body recognize fight cancer. Oncolytic virus form immunotherapy uses modified target destroy tumor while potentially stimulating antitumor responses. shown activity trials, approved specific cancers like melanoma. Research ongoing improve efficacy, expand other types, overcome logistical challenges associated delivery. Gene treat diseases caused by recessive disorders cystic fibrosis, hemophilia, muscular dystrophy, sickle anemia, well acquired genetic diseases, such viral infections immunodeficiency syndrome (AIDS).

Язык: Английский

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RNA therapeutics to control fibrinolysis: review on applications in biology and medicine

Journal of Thrombosis and Haemostasis, Год журнала: 2024, Номер 22(8), С. 2103 - 2114

Опубликована: Апрель 24, 2024

Язык: Английский

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Anticipatory gaps challenge the public governance of heritable human genome editing

Journal of Medical Ethics, Год журнала: 2024, Номер unknown, С. jme - 109801

Опубликована: Июль 2, 2024

Considering public moral attitudes is a hallmark of the anticipatory governance emerging biotechnologies, such as heritable human genome editing. However, often overlooks that future morality open to change and generations may perform different assessments on very biotechnologies we are trying govern in present. In this article, identify an ‘anticipatory gap’ has not been sufficiently addressed discussion editing, namely, uncertainty about visions applications currently attempting now. This paper motivates relevance gap, identifying challenges it generates offering various recommendations so does lead paralysis with regard germline

Язык: Английский

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Nanomedicine for cancer patient‐centered care

MedComm, Год журнала: 2024, Номер 5(11)

Опубликована: Окт. 20, 2024

Abstract Cancer is a leading cause of morbidity and mortality worldwide, an increase in incidence estimated the next future, due to population aging, which requires development highly tolerable low‐toxicity cancer treatment strategies. The use nanotechnology tailor treatments according genetic immunophenotypic characteristics patient's tumor, allow its targeted release, can meet this need, improving efficacy minimizing side effects. Nanomedicine‐based approach for diagnosis rapidly evolving field. Several nanoformulations are currently clinical trials, some have been approved marketed. However, their large‐scale production still hindered by in‐depth debate involving ethics, intellectual property, safety health concerns, technical issues, costs. Here, we survey key approaches, with specific reference organ‐on chip technology, cutting‐edge tools, such as CRISPR/Cas9 genome editing, through nanosystems needs personalized diagnostics therapy patients. An update provided on nanopharmaceuticals marketed those undergoing trials. Finally, discuss emerging avenues field challenges be overcome transfer nano‐based precision oncology into daily life.

Язык: Английский

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