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Understanding the Technologies Involved in Gene Therapy
Published in Yashwant V. Pathak, Gene Delivery Systems, 2022
Manish P. Patel, Jayvadan K. Patel, Mukesh Patel, Govind Vyas
To overcome the effect of the mutated gene or disease-causing gene, there are three prominent methods of gene editing by nucleases: zinc finger nucleases, transcription activator-like effectors nucleases and clustered regularly interspaced short palindromic repeats (Jinek et al. 2012). All three have a general mechanism for cutting and modifying DNA by joining through homologous end joining or nonhomologous end joining (Takata et al. 1998). After gene modification, there is a need to transfer the gene to the targeted cell or tissue, which can be done by two methods: viral vectors and non-viral vectors. Viral vectors are the viruses that were modified, leaving its pathological condition to causes disease. Non-viral vectors consist of a physical, inorganic and particulate system which transfers the DNA to the cell (Keller et al. 2019).
Deep Learning and Economic Prospects in Medical and Pharmaceutical Biotechnology
Published in Hajiya Mairo Inuwa, Ifeoma Maureen Ezeonu, Charles Oluwaseun Adetunji, Emmanuel Olufemi Ekundayo, Abubakar Gidado, Abdulrazak B. Ibrahim, Benjamin Ewa Ubi, Medical Biotechnology, Biopharmaceutics, Forensic Science and Bioinformatics, 2022
Charles Oluwaseun Adetunji, Kingsley Eghonghon Ukhurebor, Olugbemi Tope Olaniyan, Juliana Bunmi Adetunji, Gloria E. Okotie, Julius Kola Oloke
Most of the traditional vaccines are used to inactivate microorganisms, kill bacteria, or immobilize toxic organisms. This may generate adverse effects on the immune system by rendering it redundant; therefore, newer technologies are emanating as alternative solutions to the classical ones. Bacteria or viruses can be used as vectors or carriers of vaccine or act as living genetic modified vaccines. Also, synthetic polypeptides extracted from cloned genes or disease pathogens as recombinant vaccines are also being developed to induce immune response. Most subunit vaccines are given with an adjuvant mainly because they cannot replicate in the host, for instance virus-like particles which are immunogenic. Genetic vaccines are made up of spherical pieces of DNA referred to as plasmids made up of a promoter gene and foreign gene for protein expression in the host. Recombinant plasmids are purified foreign naked gene from bacteria given to a host directly via intramuscular means. Genetic vaccines can stimulate any part of immune compartment to bring about the desired change. The importance of DNA vaccines is the unique sequence referred to as CpG motifs having ability to act as adjuvant activating the immune system to promote the efficiency of the vaccine (Jackwood et al., 2008).
Dendrimers in Gene Delivery
Published in Neelesh Kumar Mehra, Keerti Jain, Dendrimers in Nanomedicine, 2021
Dnyaneshwar Baswar, Ankita Devi, Awanish Mishra
Various types of vectors are available for delivering therapeutic genomic molecules (such as DNA, siRNA and oligonucleotides) for treating various genetic diseases. Generally, both viral and non-viral systems play a role as vectors/vehicles for transferring genes/nucleic acids into the target cells. However, cationic polymer-based vector systems and their modified multi-functional forms, nanocomposites, are more relevant for the safe and efficient transfer of therapeutic genes (Zhang and Wagner 2017). These vectors appear as most promising agents in achieving personalised gene therapy against various genetic diseases. However, due to the highly negatively charged surface of plasmid DNA, gene delivery systems having high transfection efficiency are required for successful nucleic acid delivery (Shim et al. 2018). Gene delivery vectors are generally divided into viral and non-viral vector systems.
Nonviral gene delivery using PAMAM dendrimer conjugated with the nuclear localization signal peptide derived from human papillomavirus type 11 E2 protein
Published in Journal of Biomaterials Science, Polymer Edition, 2021
Jeil Lee, Yong-Eun Kwon, Jaegi Kim, Dong Woon Kim, Hwanuk Guim, Jehyeong Yeon, Jin-Cheol Kim, Joon Sig Choi
Gene therapy is an advanced method of genetic modification, allowing the revision and editing of abnormal genes and suppression of unwanted genes for the treatment of diseases [1]. Generally, vectors, the gene carriers used for gene therapy, are of two types, viral and nonviral. Viral vectors are used in the treatment of diseases and have been developed as gene medicines owing to their excellent transduction and infection capacity in host cells. Viral vectors are used in the treatment of diseases and have been developed as therapeutics owing to their excellent transduction and infection capacity in host cells. Viral vectors have some problems, notably high immunogenicity, recoverability of pathogenicity, induction of point mutations, and high treatment costs, despite their excellent efficiency [2].
Applications and hazards associated with carbon nanotubes in biomedical sciences
Published in Inorganic and Nano-Metal Chemistry, 2020
Ali Hassan, Afraz Saeed, Samia Afzal, Muhammad Shahid, Iram Amin, Muhammad Idrees
High-efficiency screening techniques and several biochemical assays have paved the way for identification of genomic targets that are responsible for many genetic and other lethal diseases. This directed the modern research to treat diseases by administering the nucleic acid to add, remove, replace, regulate, and repair the target gene that causes the disease. Gene therapy uses a variety of nucleic acids which include plasmid DNA, small RNA, aptamers, and antisense oligodeoxynucleotides (ODN) that control the expression of genes at co-transcriptional and protein level.[57] Major obstruction in gene therapy is delivery of gene. That complication is elucidated by use of vector system. An ideal vector system should have eccentric properties such as an ability to cross the membrane barrier, prevention of nucleic acid degradation, high specificity, and greater transfection efficacy. Two types of vector used for nucleic acids delivery, one is viral and other is the non-viral vector. Some drawbacks associated with viral vectors involve limited payload capacity and low specificity.[58] Although non-viral vectors have some advantages over viral vectors, as they have safety problems.[59] All these issues improved by making use of the CNTs as delivery vectors that exhibit the properties of a nearly ideal vector system.
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
Even though nonviral vectors of high transfection efficiency have been developed, the toxicity of vectors has to be minimized for successful disease therapy. We evaluated cytotoxic effects of polymers and polyplexes to verify the applicability of RRILH- and RRLHL-PAMAM G2 in the respective cell lines (Figure 7).