Multi-Organ Pathogenesis and Therapeutic Strategies for Cystic Fibrosis  

作　　者：Oluwaseun Babatunde Ogonna William David Richard Ifeanyichukwu Ikwugbado Oluwaseyi Oyewole Oluwaseun Babatunde;Ogonna William David;Richard Ifeanyichukwu Ikwugbado;Oluwaseyi Oyewole(Fetal Research and Therapy Program, Wake Forest Institute for Regenerative Medicine, Winston-Salem, NC, USA;Department of Chemistry, University of Houston, Houston, TX, USA;Biotechnology Graduate Program, School of Sciences and Engineering, The American University in Cairo, New Cairo, Egypt)

机构地区：[1]Fetal Research and Therapy Program, Wake Forest Institute for Regenerative Medicine, Winston-Salem, NC, USA [2]Department of Chemistry, University of Houston, Houston, TX, USA [3]Biotechnology Graduate Program, School of Sciences and Engineering, The American University in Cairo, New Cairo, Egypt

出　　处：《Journal of Biosciences and Medicines》2024年第5期312-329,共18页生物科学与医学（英文）

摘　　要：Cystic Fibrosis (CF) is the most common lethal autosomal inherited disorder that affects all races and ethnicities in the United States. However, it is mostly predominant in the Caucasian populace accounting for about 80% of all CF cases. CF most severe complication can be referred to as pulmonary bronchiectasis and infections of the airways, nonetheless, the devastating effects of the disease have far-reaching consequences beyond lung damage. CF is a heterogeneous disease that is caused by mutations in Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene. The impairment or absence of this gene can affect multiple organs and systems and is characterized not only by chronic lung blockage, infections, and inflammation but also by exocrine gland dysfunction, intestinal obstruction, liver pathology, elevated sweat chloride concentration, and in males, infertility due to the congenital bilateral absence of the vas deferens. To this end, we briefly explore the pathological effects of CF and how CF mediates the destruction of several critical organs in the body and some of the gene therapeutical approaches such as gene editing and viral-based strategies available for the treatment of this multi-organ disease.Cystic Fibrosis (CF) is the most common lethal autosomal inherited disorder that affects all races and ethnicities in the United States. However, it is mostly predominant in the Caucasian populace accounting for about 80% of all CF cases. CF most severe complication can be referred to as pulmonary bronchiectasis and infections of the airways, nonetheless, the devastating effects of the disease have far-reaching consequences beyond lung damage. CF is a heterogeneous disease that is caused by mutations in Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene. The impairment or absence of this gene can affect multiple organs and systems and is characterized not only by chronic lung blockage, infections, and inflammation but also by exocrine gland dysfunction, intestinal obstruction, liver pathology, elevated sweat chloride concentration, and in males, infertility due to the congenital bilateral absence of the vas deferens. To this end, we briefly explore the pathological effects of CF and how CF mediates the destruction of several critical organs in the body and some of the gene therapeutical approaches such as gene editing and viral-based strategies available for the treatment of this multi-organ disease.

关 键 词：Cystic Fibrosis Gene Therapy Organ Damage 

分 类 号：R57[医药卫生—消化系统]