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What is Cystic Fibrosis What is SCID4/12/2024 ![]() Recently, targeted therapies have been approved for CF treatment. Symptomatic therapies such as airway clearance by physiotherapy, mucus thinning agents, antibiotics and anti-inflammatories remain crucial for the management of CF airways ( Flume et al., 2007). CFTR dysfunction disrupts ion transport equilibrium, deregulating fluid absorption and secretion processes in epithelial tissue such as the airways, resulting in mucus accumulation and recurrent bacterial infections ( Ratjen et al., 2015). The CFTR protein, an anion channel, is expressed in a diverse range of epithelial tissues ( Riordan, 2008). Mortality in CF patients is mostly due to respiratory failure ( Elborn, 2016). CF is progressive, with its major pathology impacting the lung, liver, pancreas and intestine. Progress towards identification, correction, and expansion of a suitable regenerative cell, as well as refinement of pre-cell transplant lung conditioning protocols is discussed.Ĭystic fibrosis (CF) is an inherited, multi-organ disease caused by mutations in the CF transmembrane conductance regulator ( CFTR) gene ( Rowe et al., 2005). We review CF gene therapies using viral and non-viral delivery strategies and discuss current advances towards autologous cell-based therapies. However, applying a cell-based therapy to the human airways has distinct challenges. For CF, pioneering research has demonstrated proof-of-principle for allogenic transplantation of cultured human airway stem cells into mouse airways. Cell-based therapy–which relies on functional modification of allogenic or autologous cells ex vivo, prior to transplantation into the patient–is now a therapeutic reality for various diseases. Improved delivery vectors are needed to overcome the body’s defense system and ensure an efficient and consistent clinical response before gene therapy is suitable for clinical care. Progress towards a curative treatment strategy that implements a CFTR gene addition-technology to the patients’ lungs has been slow and not yet developed beyond clinical trials. ![]() Despite being a multi-organ disease, extensive lung tissue destruction remains the major cause of morbidity and mortality. Prognosis of patients with cystic fibrosis (CF) varies extensively despite recent advances in targeted therapies that improve CF transmembrane conductance regulator (CFTR) function. 6Department of Respiratory Medicine, Sydney Children’s Hospital, Sydney, Australia.5Adelaide Medical School, The University of Adelaide, Adelaide, Australia.4Robinson Research Institute, The University of Adelaide, Adelaide, Australia.3Respiratory and Sleep Medicine, Women’s and Children’s Health Network, Adelaide, Australia.2Molecular and Integrative Cystic Fibrosis Research Centre (miCF_RC), University of New South Wales and Sydney Children’s Hospital, Sydney, Australia.1School of Women’s and Children’s Health, Faculty of Medicine, University of New South Wales, Sydney, Australia.Allan 1,2 Nigel Farrow 3,4,5 Martin Donnelley 3,4,5 Adam Jaffe 1,2,6 Shafagh A.
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