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Novel Gene Therapies for Cystic Fibrosis Cystic Fibrosis Cystic fibrosis (CF) is an autosomal recessive, life-limiting disease resulting from gene mutations that encode the cystic fibrosis transmembrane conductance regulator (CFTR). This gene is comprised of 27 exons and is located on chromosome 7. CFTR is a cAMP-regulated chloride channel located on the apical membrane of exocrine epithelial cells. CFTR is involved in regulating the epithelial sodium channel and bicarbonate transport. There are many different types of CFTR mutations that result in CF; lack of production; failure to reach the site of action; defects in gating; conductance; abnormally low channel numbers; and …

Pyroptosis is an immunogenic form of cell death, meaning that it triggers an immune response. This makes pyroptosis a potentially promising target for immunotherapies that aim to boost the immune system's response to cancer cells and other invaders. In this article, we will discuss pyroptosis in more detail, compare it to apoptosis and explore its potential as a therapeutic target. What is Pyroptosis ? Pyroptosis is a type of programmed cell death that occurs in response to infection or other cellular stresses. It is characterized by inflammatory cytokine release, membrane rupture, and cell lysis. Pyroptosis was first described in 2004 by Steinman and …

PD-1 & PD-L1 interaction between T-cells and tumour cells. What are PD-1 and PD-L1? PD-1, also known as programmed cell death 1, is a receptor expressed on the surface of T cells and plays a pivotal role in immune homeostasis, by keeping overstimulated responses at bay while safeguarding tissues from any unintended destruction. In essence, Programmed Cell Death Protein 1 (PD-1) keeps the body's immunity balanced. The ligands for PD-1 are programmed death-ligand 1 (PD-L1) and PD-L2, which are expressed on various cells including cancer cells, antigen-presenting cells, and non-immune cells. PD-1 and its ligands are powerful players in suppressing …

CD Markers Cluster of Differentiation (CD) markers play an important role in the differentiation of B cells, T cells and NK (natural killer) cells. These markers are often responsible for the identification of certain cancers and tumors, resulting in CD markers being good candidates for cancer therapies. CTLA-4 Cytotoxic T lymphocyte-associated antigen-4 (CTLA-4)(CD152) is a protein receptor found on T cells. Both CTLA-4 and CD28 bind to B7-1/2 on antigen-presenting cells (APCs) such as dendritic cells and B cells. CTLA-4 has been shown to bind B7-1/2 with a higher affinity than CD28 d …

Idiopathic Pulmonary Fibrosis Idiopathic Pulmonary Fibrosis (IPF) is a chronic, irreversible and ultimately fatal disease characterized by progressive scaring (fibrosis) of the lungs. Affecting older adults, it targets the tissue between the lungs alveoli and the bloodstream, severely compromising gas exchange leading to worsening dyspnoea (difficulty breathing). It is associated with a poor prognosis, with several retrospective longitudinal studies suggesting a median survival of 2-3 years from the time of diagnosis (1). There is no known cause of IPF (thus the term “idiopathic” – a disease/condition which arises spo …

Danka Kozareva PhD Candidate, University College Cork The generation of neurons from stem cells The process of generating functional neurons from stem cells in the central nervous system (CNS) was believed to occur strictly during embryonic development and the early postnatal days in mammals (Ming and Song, 2011). A century ago this idea was challenged with the discovery of adult neurogenesis. Ezra Allen was the first to demonstrate that mitosis persisted in the lateral walls of adult albino rats (Allen, 1912). Several decades later Altman and Das (1965) followed up this research and determined that neurogenesis oc …