Current trends of clinical trials involving CRISPR/Cas systems

Frontiers in Medicine, Год журнала: 2023, Номер 10

Опубликована: Ноя. 10, 2023

The CRISPR/Cas9 system is a powerful genome editing tool that has made enormous impacts on next-generation molecular diagnostics and therapeutics, especially for genetic disorders traditional therapies cannot cure. Currently, CRISPR-based gene widely applied in basic, preclinical, clinical studies. In this review, we attempt to identify trends studies involving CRISPR techniques gain insights into the improvement contribution of CRISPR/Cas technologies compared modified modalities. review trials focused applications systems treatment cancer, hematological, endocrine, immune diseases, as well diagnostics. scientific basis underlined analyzed. addition, challenges application disease recent advances expand improve precision medicine are discussed.

Язык: Английский

---

CRISPR/Cas9 System as a Promising Therapy in Thalassemia and Sickle Cell Disease: A Systematic Review of Clinical Trials

Molecular Biotechnology, Год журнала: 2025, Номер unknown

Опубликована: Янв. 10, 2025

Язык: Английский

---

A review on molecular scissoring with CRISPR/Cas9 genome editing technology

Toxicology Research, Год журнала: 2024, Номер 13(4)

Опубликована: Июль 1, 2024

Genome editing is a technology to make specific changes in the DNA of cell or an organism. It has significantly altered landscape life sciences, facilitating establishment exceedingly customized genetic modifications. Among various genome technologies, CRISPR/Cas9 system, endonuclease induces double stranded break and enabling modifications genome, surfaced as formidable adaptable instrument. Its significance cannot be overstated, it not only allows for manipulation genomes model organisms but also holds great potential revolutionary advances medicine, particularly treating diseases. This review paper explores remarkable journey CRISPR/Cas9, its natural function, mechanisms, transformative impact on finally use artificial intelligence other intelligent manufacturing tools used. The introduction provides background editing, emphasizing emergence CRISPR/Cas9. Subsequent sections comprehensively elucidate disease modeling, agriculture, biotechnology, address therapeutic applications, ongoing clinical trials while discussing prospects ethical implications. We summarized key findings, indicating that empowered creation disease-specific animal models. invaluable insights into pathogenic mechanisms opens new avenues drug discovery, reaffirming editing. Finally we discussed importance continued research collaboration comprehensive utilization inherent capabilities this molecular precision tool shaping forthcoming advancements.

Язык: Английский

---

Breaking the Silence: Regulation of HIV Transcription and Latency on the Road to a Cure

Viruses, Год журнала: 2023, Номер 15(12), С. 2435 - 2435

Опубликована: Дек. 15, 2023

Antiretroviral therapy (ART) has brought the HIV/AIDS epidemic under control, but a curative strategy for viral eradication is still needed. The cessation of ART results in rapid rebound from latently infected CD4+ T cells, showing that control replication alone does not fully restore immune function, nor it eradicate reservoirs. With better understanding factors and mechanisms promote latency, current approaches are primarily focused on permanent silencing cells ("block lock") or reactivating HIV-1 gene expression combination with restoration strategies to eliminate HIV host ("shock kill"). In this review, we provide summary current, most promising cure strategies, including an analysis both latency-promoting agents (LPA) latency-reversing (LRA) have shown promise vitro, ex vivo, human clinical trials reduce reservoir.

Язык: Английский

---

Gene-Editing and RNA Interference in Treating Hepatitis B: A Review

Viruses, Год журнала: 2023, Номер 15(12), С. 2395 - 2395

Опубликована: Дек. 8, 2023

The hepatitis B virus (HBV) continues to cause substantial health and economic burdens, its target of elimination may not be reached in 2030 without further efforts diagnostics, non-pharmaceutical prevention measures, vaccination, treatment. Current therapeutic options chronic HBV, based on interferons and/or nucleos(t)ide analogs, suppress the replication but do eliminate pathogen suffer from several constraints. This paper reviews progress biotechnological approaches functional definitive HBV treatments, including gene-editing tools, i.e., zinc-finger proteins, transcription activator-like effector nucleases, CRISPR/Cas9, as well therapeutics RNA interference. advantages challenges these are also discussed. Although safety efficacy tools therapies yet demonstrated, they show promise for revitalization a much-needed advance field offer viral eradication. Particular hopes related CRISPR/Cas9; however, employing this system enter clinical testing phases. In contrast, number candidates interference, intending confer cure, have already been introduced human studies. However, larger longer trials required assess their safety. Considering that is always superior treatment, it essential pursue global vaccination.

Язык: Английский

---

Engineered ClearColi™-derived outer membrane vesicles as functional carriers for development of HIV-1 therapeutic vaccine candidate

Microbial Pathogenesis, Год журнала: 2024, Номер 193, С. 106749 - 106749

Опубликована: Июнь 13, 2024

Язык: Английский

---

Immune-mediated strategies to solving the HIV reservoir problem

Nature reviews. Immunology, Год журнала: 2025, Номер unknown

Опубликована: Фев. 13, 2025

Язык: Английский

---

CRISPR/Cas13d mediated inhibition of Siniperca chuatsi rhabdovirus infection in fish cells

Aquaculture, Год журнала: 2025, Номер unknown, С. 742355 - 742355

Опубликована: Фев. 1, 2025

Язык: Английский

---

A targeted CRISPR screen identifies ETS1 as a regulator of HIV-1 latency

PLoS Pathogens, Год журнала: 2025, Номер 21(4), С. e1012467 - e1012467

Опубликована: Апрель 8, 2025

Human Immunodeficiency virus (HIV) infection is regulated by a wide array of host cell factors that combine to influence viral transcription and latency. To understand the complex relationship between HIV-1 latency, we performed lentiviral CRISPR screen targeted set genes whose expression or activity correlates with expression. We further investigated one identified - factor ETS1, found it required for maintenance latency in both latently infected lines primary CD4 T model. Interestingly, ETS1 played divergent roles actively cells, knockout leading reduced but increased indicating can play positive negative role CRISPR/Cas9 cells from ART-suppressed people (PWH) confirmed maintains transcriptional repression clinical reservoir. Transcriptomic profiling ETS1-depleted PWH pathways involved are controlled resting cells. In particular, observed long non-coding RNA MALAT1 has been previously as regulator Furthermore, impact depletion on was partially dependent MALAT1. Additionally, demonstrate resulted enhanced abundance activating modifications (H3K9Ac, H3K27Ac, H3K4me3) histones located at terminal repeat (LTR), regulates chromatin-targeting complexes LTR. Overall, these data an important impacts through repressing regulating modification proviral histones.

Язык: Английский

---

Precision in Action: The Role of Clustered Regularly Interspaced Short Palindromic Repeats/Cas in Gene Therapies

Vaccines, Год журнала: 2024, Номер 12(6), С. 636 - 636

Опубликована: Июнь 7, 2024

Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR)-associated enzyme-CAS holds great promise for treating many uncured human diseases and illnesses by precisely correcting harmful point mutations disrupting disease-causing genes. The recent Food Drug Association (FDA) approval of the first CRISPR-based gene therapy sickle cell anemia marks beginning a new era in editing. However, delivering CRISPR specifically into diseased cells vivo is significant challenge an area intense research. identification CRISPR/Cas variants, particularly ultra-compact CAS systems with robust editing activities, paves way low-capacity delivery vectors to be used therapies. technology has evolved beyond DNA cover wide spectrum functionalities, including RNA targeting, disease diagnosis, transcriptional/epigenetic regulation, chromatin imaging, high-throughput screening, modeling. can engineer B-cells produce potent antibodies more effective vaccines enhance CAR T-cells precise efficient targeting tumor cells. challenges, off-target effects, toxicity, immune responses, inadequate tissue-specific delivery. Overcoming these challenges necessitates development specific system. This entails strategically utilizing gRNAs conjunction variants mitigate effects. review seeks delve intricacies mechanism, explore progress therapies, evaluate systems, highlight limitations, outline necessary precautions, scrutinize ethical considerations associated its application.

Язык: Английский

---