The New Frontiers of Gene Therapy and Gene Editing in Inflammatory Diseases.

Hum Gene Ther

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.

Published: April 2024

Inflammatory diseases are conditions characterized by abnormal and often excessive immune responses, leading to tissue and organ inflammation. The complexity of these disorders arises from the intricate interplay of genetic factors and immune responses, which challenges conventional therapeutic approaches. However, the field of genetic manipulation has sparked unprecedented optimism in addressing these complex disorders. This review aims to comprehensively explore the application of gene therapy and gene editing in the context of inflammatory diseases, offering solutions that range from correcting genetic defects to precise immune modulation. These therapies have exhibited remarkable potential in ameliorating symptoms, improving quality of life, and even achieving disease remission. As we delve into recent breakthroughs and therapeutic applications, we illustrate how these advancements offer novel and transformative solutions for conditions that have traditionally eluded conventional treatments. By examining successful case studies and preclinical research, we emphasize the favorable results and substantial transformative impacts that gene-based interventions have demonstrated in patients and animal models of inflammatory diseases such as chronic granulomatous disease, cryopyrin-associated syndromes, and adenosine deaminase 2 deficiency, as well as those of multifactorial origins such as arthropathies (osteoarthritis, rheumatoid arthritis) and inflammatory bowel disease. In conclusion, gene therapy and gene editing offer transformative opportunities to address the underlying causes of inflammatory diseases, ushering in a new era of precision medicine and providing hope for personalized, targeted treatments.

Download full-text PDF

Source
http://dx.doi.org/10.1089/hum.2023.210DOI Listing

Publication Analysis

Top Keywords

inflammatory diseases
20
gene therapy
12
therapy gene
12
gene editing
12
immune responses
8
inflammatory
6
gene
5
diseases
5
frontiers gene
4
editing inflammatory
4

Similar Publications

Mannose oligosaccharide (MOS) has been shown to promote animal growth, maintain intestinal health, and activate the intestinal immune system. However, the question of whether MOS can stimulate the immune system and alleviate acetylsalicylic acid (ASA)-induced gut damage remains unresolved. The purpose of this study was to investigate the impact of MOS pretreatment on the immunological and anti-inflammatory capabilities of rats with ASA-induced intestinal injury.

View Article and Find Full Text PDF

A comparative approach on the prophylactic impact of fermented beverages on acute ulcerative colitis in mouse model.

Pol J Vet Sci

December 2024

Department of Pathology, Faculty of Veterinary Medicine, Burdur Mehmet Akif Ersoy University, Burdur, 15030, İstiklal Campus, Burdur, Turkey.

Acute ulcerative colitis is an inflammatory disease of the colon that is becoming increasingly prevalent. Yet, a growing body of evidence supports the efficacy of dietary interventions in preventing acute ulcerative colitis. Fermented beverages have been the focus of research in humans and animals for several years due to their potential to influence overall health functions with an emphasis on gut health.

View Article and Find Full Text PDF

Effect of commercial prescription diets containing prebiotics on clinical signs and fecal microbiome in dogs with intestinal disease.

Pol J Vet Sci

December 2024

School of Veterinary Nursing and Technology, Faculty of Veterinary Science, Nippon Veterinary and Life Science University, 1-7-1 Kyonancho, Musashino, Tokyo 180-8602, Japan.

Diet has emerged as a key modulator of the gut microbiota, offering a potential strategy for disease prevention and management. This study investigated the effects of the Prescription Diet Gastrointestinal Biome (GB) on 7 healthy dogs and 16 dogs with chronic gastrointestinal diseases (GI dogs). Our investigation monitored changes in body weight and the Canine Inflammatory Bowel Disease Activity Index (CIBDAI) in 16 GI dogs fed a GB diet.

View Article and Find Full Text PDF

HSP90 Family Members, Their Regulators and Ischemic Stroke Risk: A Comprehensive Molecular-Genetics and Bioinformatics Analysis.

Front Biosci (Schol Ed)

December 2024

Laboratory of Genomic Research, Research Institute for Genetic and Molecular Epidemiology, Kursk State Medical University, 305041 Kursk, Russia.

Background: Disruptions in proteostasis are recognized as key drivers in cerebro- and cardiovascular disease progression. Heat shock proteins (HSPs), essential for maintaining protein stability and cellular homeostasis, are pivotal in neuroperotection. Consequently, deepening the understanding the role of HSPs in ischemic stroke (IS) risk is crucial for identifying novel therapeutic targets and advancing neuroprotective strategies.

View Article and Find Full Text PDF

GWAS-Significant Loci and Uterine Fibroids Risk: Analysis of Associations, Gene-Gene and Gene-Environmental Interactions.

Front Biosci (Schol Ed)

December 2024

Laboratory of Genomic Research, Research Institute for Genetic and Molecular Epidemiology, Kursk State Medical University, 305041 Kursk, Russia.

Background: Uterine fibroids (UF) is the most common benign tumour of the female reproductive system. We investigated the joint contribution of genome-wide association studies (GWAS)-significant loci and environment-associated risk factors to the UF risk, along with epistatic interactions between single nucleotide polymorphisms (SNPs).

Methods: DNA samples from 737 hospitalised patients with UF and 451 controls were genotyped using probe-based PCR for seven common GWAS SNPs: rs117245733 , rs547025 rs2456181 , rs7907606 , , rs58415480 , rs7986407 , and rs72709458 .

View Article and Find Full Text PDF

Want 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!