Essays on Gene Therapy

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Introduction Gene therapy is a genetic technology in which genes and nucleic acids (RNA or DNA) are used for the cure, treatment, or prevention of a disease. This technology can be accomplished by replacing a defective or mutated gene with a healthy copy to reduce the problem; removing or inactivating a mutated or defective gene to reduce or eliminate improper functioning, and/or introducing a new kind of gene in the body to help in the fight against disease. Initially, the technology was used for orphan diseases such as primary immunodeficiencies, but with the passage of time, ...

The purpose of this paper is to review the etiology, pathophysiology, clinical manifestations, inheritance patterns, and recent advances in disease management of cystic fibrosis. Cystic fibrosis is a progressive and lethal disease resulting from an autosomal recessive gene (Singh, Rebordosa, Bernholz, & Sharma, 2015). The major problem associated with cystic fibrosis is the repeated lung infections that over time severely damage the lungs, scar and widen the passageways and limit the ability to breathe (Cystic Fibrosis Foundation, n.d.). As of 2013, there are an estimated 70,000 cystic fibrosis patients globally with 33,000 of them in the United States ( ...

Cystic fibrosis is a chronic, progressive and often deadly genetic disease that affects the body’s mucous glands. It follows that cystic fibrosis (CF) is evident in the body’s digestive and respiratory systems, particularly in children and young adults (Flume et al., 2007). In some cases, it affects the reproductive system and the sweat glands. A CF-like disease has been known over the past two centuries, but it was in 1938 that the term cystic fibrosis was first used. CF may also be considered to be an autosomal-recessive disease. Its estimated heterozygote occurrence is one for every twenty ...

Gene replacement refers to a technique which utilizes homologous recombination (Carter & Shieh, 2010, p. 265), to make alterations by either adding a gene, deleting a gene or including point mutations. Gene replacement attempts to cure most of the genetic diseases at molecular level. A successful gene replacement prevents a protein from causing harm, giving new functions to proteins, and recovering or enhancing the normal function of a protein. This technique is commonly used to prevent, treat or improve animals. This is a permanent process that is very beneficial to animals. The process of animal gene replacement involves several processes ...

Coursename Sickle-cell anemia is a genetic blood disorder, which affects the red blood cells. It was first described in the medical literature in 1846. In 1910, Ernest Irons became first to write about the pathologically altered red blood cells of the patient with sickle-cell anemia (Serjeant 425-429). It is caused by the mutation of the gene encoding the synthesis of the hemoglobin. As a result, the hemoglobin of the affected people is defective (hemoglobin S). Glutamic acid in it is substituted with valine in the sixth position of the β-chain. This change greatly reduces the solubility of hemoglobin in ...

Introduction

The treatment for multitudes of diseases and disorders resulting from genetic reasons have been ambiguous since long as the only cure could be the correction of genes and DNA in the specific region. Gene therapy gave a promising horizon to the correction of such genetic defects whereby molecular tools are applied to reverse the mutation or any other changes in the DNA. The advancing biomedical research have offered several tools for gene therapy since last few decades that have been able to perform the desired roles but with one or the other limitations. Lately, CRISPR-Cas9 based tools for gene ...

Bioethical Issues behind Gene Therapy Trials

Gene therapy is a prospective therapy for curing many genetic defects by correcting the mistakes happening at gene level. It can be somatic or germline targeting either blood or bone marrow in former and eggs or sperms in latter. Ethical issues prevail with the germline gene therapy and it has not been approved yet. Similarly, the gene therapy trials need to be regulated and is governed by many bioethical issues. In US, the Department of Health and Human Services (DHHS) regulates the gene therapy clinical trials. This organization has further sub-divisions that regulate the human subjects under trials through ...

A significant population in the world smoke cigarettes, which is a harmful behavior. Various studies have indicated that smoking is among the leading but preventable causes of death and diseases around the world. Apart from health issues produced by smoking, financial issues also prevail and they are among the main predictors of quitting smoking. For instance, Sindelar and O'Malley (2013) found out that quitting smoking could help an individual to save about $3000 per year. Despite the financial, environmental, and health problems created by smoking cigarettes, many people argue that smoking provides various benefits, and they cannot imagine the ...