Results 61 to 70 of about 459,657 (249)

Alpha-1 antitrypsin deficiency: outstanding questions and future directions

Orphanet Journal of Rare Diseases, 2018
BackgroundAlpha-1 antitrypsin deficiency (AATD) is a rare hereditary condition that leads to decreased circulating alpha-1 antitrypsin (AAT) levels, significantly increasing the risk of serious lung and/or liver disease in children and adults, in which ...
M. Torres-Durán, J. López-Campos, M. Barrecheguren, M. Miravitlles, B. Martínez-Delgado, Silvia Castillo, A. Escribano, A. Baloira, María Mercedes Navarro-García, D. Pellicer, Lucía Bañuls, María Magallón, F. Casas, F. Dasí   +13 more
semanticscholar   +1 more source

Engineering Lipid Nanoparticles for Precision RNA Delivery: Design Principles, Targeting Strategies, and Clinical Prospects

Cancer Nexus, EarlyView.
ABSTRACT Lipid nanoparticles (LNPs) represent the most clinically advanced platform for RNA delivery and have enabled major breakthroughs in vaccines and gene therapies. However, their broader application is still limited by inefficient extrahepatic delivery, immunogenicity, and insufficient control over tissue‐ and cell‐specific targeting. This review
Yu Han, Ying Wang, Xintao Gao, Yan Lei, Yantao Li, Zhiyong Tang   +5 more
wiley   +1 more source

Alpha-1 antitrypsin Pi*Z gene frequency and Pi*ZZ genotype numbers worldwide: an update

International Journal of COPD, 2017
In alpha-1 antitrypsin deficiency (AATD), the Z allele is present in 98% of cases with severe disease, and knowledge of the frequency of this allele is essential from a public health perspective.
I. Blanco, P. Bueno, I. Diego, S. Pérez-Holanda, F. Casas-Maldonado, C. Esquinas, M. Miravitlles   +6 more
semanticscholar   +1 more source

Exercise‐specific plasma proteomic signatures in racehorses: Candidates for training adaptation and peak load monitoring

Equine Veterinary Journal, EarlyView.
Abstract Background Racehorses undergo profound physiological changes with training and competition, but current biomarkers inadequately capture the complex molecular dynamics of exercise. This study aimed to identify novel plasma biomarkers of training adaptation and peak load using high‐throughput proteomics.
Jowita Grzędzicka, Bianka Świderska, Ewa Sitkiewicz, Izabela Dąbrowska, Olga Witkowska‐Piłaszewicz   +4 more
wiley   +1 more source

Rare variants in alpha 1 antitrypsin deficiency: a systematic literature review

Orphanet Journal of Rare Diseases
Background Alpha 1 Antitrypsin Deficiency (AATD) is a largely underrecognized genetic condition characterized by low Alpha 1 Antitrypsin (AAT) serum levels, resulting from variations in SERPINA1.
Ilaria Ferrarotti, Marion Wencker, Joanna Chorostowska-Wynimko   +2 more
doaj   +1 more source

The first report of two cases of fatal liver injury due to anti-tuberculosis drugs in the presence of alpha-1 antitrypsin deficiency

International Journal of Mycobacteriology, 2017
Tuberculosis (TB) is a major global health problem. Awareness of liver injury due to anti-TB therapy is vital because fulminant hepatic failure is a devastating and often fatal condition without liver transplantation.
Shahram Habibzadeh, Jafar Mohammad Shahi, Hassan Ghobadi, Nasrollah Maleki   +3 more
doaj   +1 more source

Microengineering the Liver: Strategies for Constructing Functional Liver‐on‐a‐Chip Devices

Exploration, EarlyView.
This review summarizes recent advances in liver‐on‐a‐chip (LOC) technologies, including fabrication strategies and functional integration approaches, and discusses their promising applications in drug screening and related biomedical fields. ABSTRACT Reliable in vitro liver models are indispensable for researching liver diseases and developing ...
Jie Wang, Ziwei Liang, Jiapu Wang, Zongyi Li, Shaojie Wang, Yan Wei, Xin Xie, Di Huang   +7 more
wiley   +1 more source

Experimental and investigational drugs for the treatment of alpha-1 antitrypsin deficiency

Expert Opinion on Investigational Drugs, 2019
Introduction: Alpha-1 antitrypsin deficiency (AATD) is most often associated with chronic lung disease, early onset emphysema, and liver disease. The standard of care in lung disease due to AATD is alpha-1 antitrypsin augmentation but there are several ...
A. Pye, A. Turner
semanticscholar   +1 more source

Why has it been so difficult to prove the efficacy of alpha-1-antitrypsin replacement therapy? Insights from the study of disease pathogenesis

Drug Design, Development and Therapy, 2011
Jennifer A Dickens, David A LomasDepartment of Medicine, University of Cambridge, Cambridge Institute for Medical Research, Wellcome Trust/MRC Building, Cambridge, UKAbstract: Alpha-1-antitrypsin is the most abundant circulating protease inhibitor. It is
Dickens JA, Lomas DA
doaj  

Protective role of the alpha-1-antitrypsin in intervertebral disc degeneration

Journal of Orthopaedic Surgery and Research, 2021
Background Intervertebral disc degeneration is a complex disease with high prevalence. It suggests that cell death, senescence, and extracellular matrix degradation are involved in the pathogenesis. Alpha-1 antitrypsin (AAT), a serine protease inhibitor,
Weikun Liu, Yanfu Wang
doaj   +1 more source