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 intracellular barriers have prevented the development of a gene therapy-based treatment for CF. In a recent study a review of the applications of the new non-viral and viral gene therapy vectors was carried out as well as overcoming barriers through the use of artificial chromosomes derived from human beings so as to find solutions to expression of CFTR.The scientific research has brought a lot of positive impacts to the medicine industry as seen using the models of animals including ferrets and pigs. Prenatal gene therapy is a potential alternative to gene transfer to fully developed lungs. However, unsettled problems, as well as the likelihood of adversative impacts on both postnatal and prenatal development, germ line transmission require further investigation and the risk of initiating oncogenic or degenerative processes. How it was applied to solve a particular problem The therapy of the genes and cells is largely reliant on on the progress in a safe as well as an efficient method to achieve transfer of gene. The comprehensive CFTR of genomes locus offers the ordinary context of genomes which are the same as those of the identified positive chromosomes. T...