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Gene Therapy in Tissue Engineering: Prospects and Challenges
Published in Rajesh K. Kesharwani, Raj K. Keservani, Anil K. Sharma, Tissue Engineering, 2022
Indeed our genes define who we are, though sometimes a defect in the gene can produce an imperfection that can threaten the existence of an individual. In the last four decades, several genes have been identified that are responsible for different genetic disorders in human. A new field of biology has henceforth emerged called the gene therapy. Gene therapy can, therefore, be defined as a method of treating diseases by either modifying the expressions of an individual’s genes or alteration of abnormal genes or introduction of a normal healthy gene. The field holds great promise for treating an extensive range of human diseases. It has been found to be successful especially in single-gene diseases such as sickle cell anemia, hemophilia, etc. However, the therapy is still in its infancy but it brings a new ray of hope for patients who are living with difficult and incurable diseases.
Dendrimers in Gene Delivery
Published in Neelesh Kumar Mehra, Keerti Jain, Dendrimers in Nanomedicine, 2021
Dnyaneshwar Baswar, Ankita Devi, Awanish Mishra
Somatic and germline therapy are types of gene therapy. Somatic gene therapy is considered a much safer and most common therapy, which contains gene vectors, such as viral vectors and non-viral vectors (Nayerossadat et al. 2012). Vectors are basically transporters that may deliver the therapeutic gene to the infected cells. The general concept of gene therapy is inserting the exogenous gene into somatic cells that develop organs to produce a therapeutic effect. Germline gene therapy has the potential to manipulate reproductive sperm cells and eggs to create heritable changes (Misra 2013). Some therapeutic agents contain nucleic acid materials, which upon administration results in cellular adaptation, repairing, insertion or deletion of a gene sequence. Genetic materials such as DNA, mRNA, miRNA, siRNA and antisense oligonucleotides are commonly used in deficient target cells or tissue to re-establish the specific gene function for disease management (Chen et al. 2018). On this basis, gene therapy may appear as a promising pharmacotherapy tool (Klug et al. 2012; Ingusci et al. 2019).
Environmental Disease
Published in Gary S. Moore, Kathleen A. Bell, Living with the Earth, 2018
Gary S. Moore, Kathleen A. Bell
Genetic screening will rapidly advance the discovery of genetically based diseases while offering improved capabilities in gene therapy. Gene therapy may involve removing cells with defective genes from a person and replacing those genes with normal or healthy genes and then placing the cells back into the patient where the cells will begin to produce normal product and “cure” the disease. As fantastic as this sounds, these procedures are being attempted by such people as Dr. W. French Anderson, director of gene-therapy programs at the University of Southern California/Norris Comprehensive Cancer Center in Los Angeles. Dr. French and colleagues performed the first federally approved gene therapy procedure in 1990 when they replaced a faulty gene in Ashanthi DeSilva, a 4-year-old girl with severe acquired immune deficiency (SCID or “bubble-boy” disease). The disease results from a deficiency in the enzyme adenosine deaminase because of a rare and deadly mutation. Dr. French et al. extracted some white blood cells from the girl and exposed them to viruses that were genetically engineered to carry the normal gene for the production of adenosine deaminase. When the white blood cells were returned to her bloodstream, her cells began producing the necessary enzyme that restored immunity. The transplanted cells eventually die, requiring follow-up treatments.
Enhanced transfection efficiency of low generation PAMAM dendrimer conjugated with the nuclear localization signal peptide derived from herpesviridae
Published in Journal of Biomaterials Science, Polymer Edition, 2021
Jeil Lee, Yong-Eun Kwon, Younjin Kim, Joon Sig Choi
In various dendrimers, PAMAM dendrimers consisting of biodegradable peptide bonds have received attention because biodegradability is a key factor determining safety in the living system. PAMAM dendrimer was developed by Tomalia at al, and first used as the nonviral vector by Haensler and Szoka for application of gene therapy [2]. Gene Therapy is a technique to treat various illnesses at the genetic level. In the past, the definition of gene therapy was the replacement of deficient genes through the introduction of normal genes. Technical advances such as RNA interference (RNAi) and gene editing have extended the definition of gene therapy to include revision and editing of deficient genes and inhibition of unwanted gene expression [3]. There are two methods for gene therapy: physical delivery and use of a vector system. The vector system utilizes either the viral vector or nonviral vector [4].
Polyethylenimine-based nanocarriers in co-delivery of drug and gene: a developing horizon
Published in Nano Reviews & Experiments, 2018
Abbas Zakeri, Mohammad Amin Jadidi Kouhbanani, Nasrin Beheshtkhoo, Vahid Beigi, Seyyed Mojtaba Mousavi, Seyyed Ali Reza Hashemi, Ayoob Karimi Zade, Ali Mohammad Amani, Amir Savardashtaki, Esmail Mirzaei, Sara Jahandideh, Ahmad Movahedpour
Gene therapy is capable of treating human diseases caused by defective genes and its purpose is to transfer genetic material to specific cells for the treatment of the disease. Protecting DNA against temperature and pH changes, and the destruction of lysosomes and passing through the membrane to target it are the abilities of the gene as a clinical treatment, which is strongly dependent on the use of appropriate gene directory. Today, all gene delivery carriers have been developed, such as several viral and non-viral vectors, has been improved and used to successfully cure some diseases. In general, both viral and non-viral vectors, despite the advantages and disadvantages, are used in recent advances in gene therapy strategies somewhat bring the expectations of years of gene therapy closer to reality and the hope of achieving more success. It seems that the ultimate gene therapy solution for treating many diseases in this century is human. The result of a great deal of research on this subject in recent years is very promising, and polyethylene imine can be used as an appropriate vector.
Gene doping: Present and future
Published in European Journal of Sport Science, 2020
Rebeca Araujo Cantelmo, Alessandra Pereira da Silva, Celso Teixeira Mendes-Junior, Daniel Junqueira Dorta
According to Haisma and de Hon (2006), gene therapy can be defined as “the transfer of genetic material to human cells for treatment or prevention of a disease or disorder. Genetic materials can be DNA, RNA or genetically altered cells” (Haisma & de Hon, 2006, p. 259). Despite the great development of the gene therapy area over the years, this therapy still poses numerous limitations and risks, such as the patient’s immune response and consequent treatment rejection (Artioli et al., 2007; Gaffney & Parisotto, 2007; Haisma & de Hon, 2006; Karthikeyan & Pradeep, 2006).