Developing delivery vectors capable of transducing genetic material across the lung epithelia and mucus barrier is a major challenge and of great interest to enable gene therapies to treat pulmonary diseases. Recombinant Adeno-associated Viruses (rAAVs) have emerged as attractive candidates among viral and non-viral vectors due to their broad tissue tropism, ability to transduce dividing and quiescent cells, and their safety profile in current human applications. While rAAVs have demonstrated safety in earlier clinical trials for lung disease applications, there are still some limitations regarding rAAV-transgene delivery in pulmonary cells. Thus, further improvements in rAAV engineering are needed to enhance the effectiveness of rAAV-based therapies for lung diseases. Such therapies could benefit patients with chronic lung diseases, such as asthma, chronic obstructive pulmonary disease, pulmonary hypertension, and cystic fibrosis, among others, by regulating hereditary gene mutations or acquired gene deregulations causing these conditions. Alongside therapeutic development, advances in the rAAV production process are essential to meet increasing production demands, while reducing manufacturing costs. This review discusses current challenges and recent advances in the field of rAAV engineering and manufacturing to encourage the clinical development of new pulmonary gene therapy treatments.
Download full-text PDF |
Source |
|---|---|
| http://dx.doi.org/10.2174/0115665232294935240826061311 | DOI Listing |
Publication Analysis
Top Keywords
Similar Publications
Insights into NEK2 inhibitors as antitumor agents: From mechanisms to potential therapeutics.
Eur J Med Chem
January 2025
Department of Respiratory and Critical Care Medicine, Targeted Tracer Research and Development Laboratory, Institute of Respiratory Healthand, Department of Frontiers Science Center for Disease-related Molecular Network, Core Facilities, West China Hospital, Sichuan University, Chengdu, 610041, Sichuan, China. Electronic address:
NEK2, a serine/threonine protein kinase, is integral to mitotic events such as centrosome duplication and separation, microtubule stabilization, spindle assembly checkpoint, and kinetochore attachment. However, NEK2 overexpression leads to centrosome amplification and chromosomal instability, which are significantly associated with various malignancies, including liver, breast, and non-small cell lung cancer. This overexpression could facilitate tumor development and confer resistance to therapy by promoting aberrant cell division and centrosome amplification.
View Article and Find Full Text PDFEra of Generalist Conversational Artificial Intelligence to Support Public Health Communications.
J Med Internet Res
January 2025
School of Computer Science, University of Technology Sydney, Sydney, Australia.
The integration of artificial intelligence (AI) into health communication systems has introduced a transformative approach to public health management, particularly during public health emergencies, capable of reaching billions through familiar digital channels. This paper explores the utility and implications of generalist conversational artificial intelligence (CAI) advanced AI systems trained on extensive datasets to handle a wide range of conversational tasks across various domains with human-like responsiveness. The specific focus is on the application of generalist CAI within messaging services, emphasizing its potential to enhance public health communication.
View Article and Find Full Text PDFThe clinical assessment of lung involvement in patients with Still's disease, results from the multicentre international AIDA Network Still's Disease Registry.
Rheumatology (Oxford)
January 2025
Department of Medical Sciences, Surgery and Neurosciences, Research Center of Systemic Autoinflammatory Diseases and Behçet's Disease Clinic, University of Siena, Siena, Italy.
Objectives: To assess the lung involvement in patients with Still's disease, an inflammatory disease assessing both children and adults. To exploit possible associated factors for parenchymal lung involvement in these patients.
Methods: A multicentre observational study was arranged assessing consecutive patients with Still's disease characterized by the lung involvement among those included in the AIDA (AutoInflammatory Disease Alliance) Network Still's Disease Registry.
Suspected heparin-induced thrombocytopaenia in pulmonary thromboendarterectomy: retrospective cohort.
Interdiscip Cardiovasc Thorac Surg
January 2025
Department of Thoracic Surgery and Heart-Lung Transplantation, Paris-Saclay University, Marie-Lannelongue Hospital, 92350, Le Plessis-Robinson, France.
Objectives: Heparin is given for anticoagulation during and after pulmonary thromboendarterectomy for chronic thromboembolic pulmonary hypertension. Our objective was to add to the limited data available on the incidence, management, and outcomes of suspected heparin-induced thrombocytopaenia after pulmonary thromboendarterectomy for chronic thromboembolic pulmonary hypertension.
Methods: This retrospective single-centre study included consecutive patients with suspected heparin-induced thrombocytopaenia after pulmonary thromboendarterectomy done in 2005-2018.
Ongoing post-COVID-19 symptoms and complaints among healthcare professionals.
J Infect Dev Ctries
December 2024
Family Medicine, Merkezefendi District Health Directorate, Denizli, Turkey.
Introduction: Post-COVID-19 syndrome refers to the occurrence of symptoms lasting more than 4 weeks in individuals who have recovered from COVID-19. This study aims to investigate the post-COVID-19 symptoms in healthcare professionals.
Methodology: This descriptive study included 166 healthcare professionals who had tested positive for COVID-19 via PCR at least four weeks prior and subsequently presented to the Family Medicine Clinic at Pamukkale University Training and Research Hospital.
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!