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Alpha-1 antitrypsin (AAT) deficiency, an autosomal co-dominant inherited condition, significantly impacts lung and liver functions, with mutations in the SERPINA1 gene, notably the Z allele, playing a pivotal role in disease susceptibility. This retrospective descriptive study from a rural Eastern Kentucky pulmonary clinic aimed to characterize patients with AAT deficiency, focusing on demographic, clinical, and laboratory parameters extracted from electronic health records (EHR) of Appalachian Regional Healthcare (ARH). Among 100 patient encounters, 56 were analyzed, revealing notable sex-based differences in smoking rates and co-existing conditions, with males showing higher rates of black lung and chronic obstructive pulmonary disease. In comparison, females exhibited higher rates of asthma, COVID-19, pneumothorax, and obstructive sleep apnea. The study emphasizes the importance of understanding genotype-phenotype correlations and demographic factors in assessing AAT deficiency, advocating for further research to refine management strategies and elucidate causal relationships.
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http://dx.doi.org/10.7759/cureus.56395 | DOI Listing |
Gene
August 2025
Genetics Laboratory, Instituto Universitario de Enfermedades Tropicales y Salud Pública de Canarias (IUETSPC), Universidad de La Laguna (ULL), Tenerife, Spain; Department of Biochemistry, Microbiology, Cell Biology and Genetics, Universidad de La Laguna (ULL), Tenerife, Spain.
Introduction: Certain variants in the SERPINA1 gene cause Alpha-1 antitrypsin deficiency (AATD). Null SERPINA1 alleles result in the full absence of circulating AAT, which increases the severity of AATD-related respiratory illnesses. PI*S-plus alleles are the combination in cis of the PI*S allele with another variant that confers more deleterious features to the haplotype.
View Article and Find Full Text PDFLaryngoscope
September 2025
Department of Otorhinolaryngology, Zealand University Hospital, Køge, Denmark.
Objective: To estimate the risk of cholesteatoma in patients with alpha-1 antitrypsin deficiency (AATD) compared to the general population using time-to-event analysis.
Methods: We conducted a nationwide nested cohort study identifying all patients with AATD in Denmark and subsequently matching each case with up to 10 controls based on age, sex, and municipality. Hazard ratios (HR) of cholesteatoma by AATD were calculated using Cox regression analysis adjusted for age and sex.
Mol Ther
August 2025
RNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, MA 01605, USA; Department of Molecular, Cell and Cancer Biology, University of Massachusetts Chan Medical School, Worcester, MA 01605, USA; Department of Molecular Medicine, University of Massachusetts Chan Medic
Alpha-1 antitrypsin deficiency (AATD) is commonly caused by a G-to-A mutation in the SERPINA1 gene (the PiZ mutation). The mutant PiZ AAT protein is sequestered in hepatocytes, causing lung emphysema due to insufficient AAT protein to inhibit neutrophil elastase in the lung. Here, we show that a compact adenine base editor (ABE) with an evolved Cas9 nickase derived from Neisseria meningitidis (eNme2.
View Article and Find Full Text PDFPharmaceuticals (Basel)
August 2025
Plasma Derived Therapies R&D, Baxalta Innovations GmbH, Part of Takeda, 1220 Vienna, Austria.
: The quality of clinical studies is largely determined by the bioanalytical methods used for testing study samples. Rigorous assay validation following defined criteria, for example, the European Medicines Agency guideline for bioanalytical method validation, is a prerequisite for such assays. Alpha1-antitrypsin (AAT) measurement, i.
View Article and Find Full Text PDFAnn Med Surg (Lond)
July 2025
Voice of Doctors Research School, Dhaka, Bangladesh.
Alpha-1 antitrypsin deficiency (AATD) is a prevalent autosomal recessive disorder affecting approximately 3 million people worldwide, caused by mutations in the SERPINA1 gene leading to low alpha-1 antitrypsin (AAT) levels. This deficiency predisposes individuals to chronic obstructive pulmonary disease, bronchiectasis, neonatal cholestasis, and liver cirrhosis. The most common pathogenic mutation, PI*ZZ allele, produces misfolded Z-AAT protein accumulating in hepatocytes.
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