Background Increasing the activity of defective cystic fibrosis transmembrane conductance
regulator (CFTR) protein is a potential treatment for cystic fibrosis. Methods We conducted a …

Current viral vectors for gene therapy 1 , 2 , 3 are associated with serious safety concerns,
including leukemogenesis 4 , and nonviral vectors are limited by low gene transfer efficiency 5 …

BACKGROUND: Asthma is characterized by a T H 2 immune response. CD4 + CD25 hi
regulatory T cells (Tregs) have been proposed to prevent allergic diseases through suppression …

Interleukin-8 (IL-8) activates neutrophils via the chemokine receptors CXCR1 and CXCR2.
However, the airways of individuals with cystic fibrosis are frequently colonized by bacterial …

The Human Phenotype Ontology (HPO, https://hpo.jax.org ) was launched in 2008 to provide
a comprehensive logical standard to describe and computationally analyze phenotypic …

Rationale: Primary ciliary dyskinesia (PCD) is characterized by recurrent airway infections
and randomization of left–right body asymmetry. To date, autosomal recessive mutations have …

Asthma is among the most frequent chronic diseases in childhood. Although numerous
environmental risk factors have already been identified, the basis for familial occurrence of …

Rationale: Acute lung dysfunction of noninfectious etiology, known as idiopathic pneumonia
syndrome (IPS), is a severe complication following hematopoietic stem cell transplantation (…

Background Lumacaftor and ivacaftor combination treatment showed efficacy in patients
aged 12 years or older with cystic fibrosis homozygous for F508del-cystic fibrosis …

DYX1C1 has been associated with dyslexia and neuronal migration in the developing
neocortex. Unexpectedly, we found that deleting exons 2–4 of Dyx1c1 in mice caused a …