Gene Therapy

GENE THERAPY Name Institution Affiliation Course Date of Submission Gene and cell therapy for cystic fibrosis: From bench to bedside Current therapies for human are inadequate, and the need for effective therapies for most human molecular genetic provides a tool for unprecedented approach to the treatment of diseases through attack to the mutant genes. Results have indicated various target organs and gene transfer techniques resulting to a wide scientific and medical acceptance regarding the feasibility of gene therapy for disorder of various organs such as liver, lungs and some kind of cancer in addition to enzymes and hormones. There are well developed models that involve alteration of mutant target genes through gene transfer with recombinant pathogenic virus to express new genetic information hence, correcting disease phenotypes. How the technology works This is a technology of recent development of gene transfer vectors in utero gene therapy and the application of stem cells as experimental therapy for CF.similarly, it is also a a vector of genomes and the artificial chromosomes of the human body are usually dependent on the segregation or division of the hot cell and cell replication. The CFTR is expressed in airways epithelia to serves phosphorylation-regulated Cl- channel and a regulator of channels and transporters. Activation of CFTR leads to parallel inhibition of epithelia Na+ channel (ENaC) that is lost when CFTR is dysfunctional. Similarly, according to Conese, et al., (2011) the rise in rate of absorption of the Na+ as well as the reduction and total elimination of Cl- leads to a reduced thickness of the airways surface liquid and this in turn results to the destruction of the mucociliary. The loss of CFTR is expressed in sub-mucosal glands playing the role of defense and thus preventing secretion of the mucus and anti-microbial factors by sub-mucosal glands. However, the limited efficacy of gene transfer vectors as well as extra- and intrace...