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Gene therapy is a medical technology that aims to produce a therapeutic effect through the manipulation of gene expression or through altering the biological properties of living cells.
A DNA vaccine is a type of vaccine that transfects a specific antigen-coding DNA sequence into the cells of an organism as a mechanism to induce an immune response.
Adenoviruses are medium-sized, nonenveloped viruses with an icosahedral nucleocapsid containing a double-stranded DNA genome. Their name derives from their initial isolation from human adenoids in 1953.
Transplant rejection occurs when transplanted tissue is rejected by the recipient's immune system, which destroys the transplanted tissue. Transplant rejection can be lessened by determining the molecular similitude between donor and recipient and by use of immunosuppressant drugs after transplant.
Adeno-associated viruses (AAV) are small viruses that infect humans and some other primate species. They belong to the genus Dependoparvovirus, which in turn belongs to the family Parvoviridae. They are small replication-defective, nonenveloped viruses and have linear single-stranded DNA (ssDNA) genome of approximately 4.8 kilobases (kb).
Viral vectors are modified viruses designed to deliver genetic material into cells. This process can be performed inside an organism or in cell culture. Viral vectors have widespread applications in basic research, agriculture, and medicine.
Allograft inflammatory factor 1 (AIF-1) also known as ionized calcium-binding adapter molecule 1 (IBA1) is a protein that in humans is encoded by the AIF1 gene.
ICOS ligand is a protein that in humans is encoded by the ICOSLG gene located at chromosome 21. ICOSLG has also been designated as CD275.
Gene therapy for osteoarthritis is the application of gene therapy to treat osteoarthritis (OA). Unlike pharmacological treatments which are administered locally or systemically as a series of interventions, gene therapy aims to establish sustained therapeutic effect after a single, local injection.
Adenovirus varieties have been explored extensively as a viral vector for gene therapy and also as an oncolytic virus.
Lentiviral vectors in gene therapy is a method by which genes can be inserted, modified, or deleted in organisms using lentiviruses.
DNA-directed RNA interference (ddRNAi) is a gene-silencing technique that utilizes DNA constructs to activate an animal cell's endogenous RNA interference (RNAi) pathways. DNA constructs are designed to express self-complementary double-stranded RNAs, typically short-hairpin RNAs, that bring about the silencing of a target gene or genes once processed. Any RNA, including endogenous messenger RNA (mRNAs) or viral RNAs, can be silenced by designing constructs to express double-stranded RNA complementary to the desired mRNA target.
James M. Wilson is an American biomedical researcher who serves as the Director of the Gene Therapy Program, Rose H. Weiss Professor and Director of the Orphan Disease Center, and Professor of Medicine and Pediatrics at the Perelman School of Medicine at the University of Pennsylvania. Previously, he held the John Herr Musser endowed professorship at the Perelman School of Medicine.
Shaf Keshavjee is a Canadian surgeon and the current Surgeon-in-Chief at University Health Network in Toronto, the Director of the Toronto Lung Transplant Program, as well as a clinical scientist and professor with the University of Toronto.
Isolated organ perfusion technique is employed to precipitate an organ's perfusion and circulation that are independent/isolated from the body's systemic circulation for various purposes such as organ-localized chemotherapy, organ-targeted delivery of drug, gene or anything else, organ transplantation, and organ injury recovery. The technique has been widely studied in animal and human for decades. Before the implementation, the perfusion system will be selected and the process can be similar to organ bath. Isolated organ perfusion technique, nevertheless, is averagely conducted in vivo without leaving the organ alone as a whole out of the body.
Rainer Blasczyk is a German physician, university professor, entrepreneur and philanthropist. He specializes in transfusion medicine with a focus on transplantation and immunogenetics. He is considered a pioneer in genetic engineering of allografts to increase histocompatibility and prevent organ rejection after organ transplantation.
Jean Bennett is the F. M. Kirby Professor of Ophthalmology in the Perelman School of Medicine at the University of Pennsylvania. Her research focuses on gene therapy for retinal diseases. Her laboratory developed the first FDA approved gene therapy for use in humans, which treats a rare form of blindness. She was elected a member of the National Academy of Sciences in 2022.
Alicia L. Bertone is an American academic, administrator, researcher, and veterinary surgeon. She is a professor and a provost in the Office of Academic Affairs at the Ohio State University. Bertone has worked as the Vice-Provost of Graduate Studies, Associate Vice Provost of Data and Analysis, the ENGIE-Axium Endowed Dean of the Graduate School, and the Trueman Family Endowed Chair at the Ohio State University. Bertone is a Professor of Veterinary Clinical Sciences, and, as the Trueman Endowed Chair, established and directed the Comparative Orthopedic Research Laboratory at the University.
Cellular adoptive immunotherapy is a type of immunotherapy. Immune cells such as T-cells are usually isolated from patients for expansion or engineering purposes and reinfused back into patients to fight diseases using their own immune system. A major application of cellular adoptive therapy is cancer treatment, as the immune system plays a vital role in the development and growth of cancer. The primary types of cellular adoptive immunotherapies are T cell therapies. Other therapies include CAR-T therapy, CAR-NK therapy, macrophage-based immunotherapy and dendritic cell therapy.
Ex vivo lung perfusion, EVLP, is a form of machine perfusion aimed at sustaining the active aerobic cellular metabolism of donor lungs outside the donor's body prior to lung transplantation. This medical preservation technique typically occurs within a specialised machine engineered to mimic the conditions of the natural circulatory system. The machine supplies the lung with perfusate and ventilates it using a protective mechanical ventilator under human body temperature. This allows the delivery of essential nutrients and oxygen to the donor lung, supporting metabolic functions and allowing for prolonged preservation up to 17 hours. The three major EVLP protocols at present are the Toronto protocol, Lund protocol, and Organ Care System protocol. These EVLP protocols can be effective in rendering initially rejected donor lungs suitable for transplantation through reassessment and damage repair, thus widening the donor lung pools.
References
- ↑ Cypel, Marcelo; Liu, Mingyao; Rubacha, Matt; Yeung, Jonathan C.; Hirayama, Shin; Anraku, Masaki; Sato, Masaaki; Medin, Jeffrey; Davidson, Beverly L.; Perrot, Marc de; Waddell, Thomas K. (2009-10-28). "Functional Repair of Human Donor Lungs by IL-10 Gene Therapy". Science Translational Medicine. 1 (4): 4ra9. doi:10.1126/scitranslmed.3000266. ISSN 1946-6234. PMID 20368171. S2CID 13366956.
- ↑ Machuca, Tiago N.; Cypel, Marcelo; Bonato, Riccardo; Yeung, Jonathan C.; Chun, Yi-Min; Juvet, Stephen; Guan, Zehong; Hwang, David M.; Chen, Manyin; Saito, Tomohito; Harmantas, Constantine (2017-09-01). "Safety and Efficacy of Ex Vivo Donor Lung Adenoviral IL-10 Gene Therapy in a Large Animal Lung Transplant Survival Model". Human Gene Therapy. 28 (9): 757–765. doi:10.1089/hum.2016.070. ISSN 1043-0342. PMID 28052693.
- ↑ Figueiredo, Constanca; Carvalho Oliveira, Marco; Chen-Wacker, Chen; Jansson, Katharina; Höffler, Klaus; Yuzefovych, Yuliia; Pogozhykh, Olena; Jin, Zhu; Kühnel, Mark; Jonigk, Danny; Wiegmann, Bettina (2019-04-04). "Immunoengineering of the Vascular Endothelium to Silence MHC Expression During Normothermic Ex Vivo Lung Perfusion". Human Gene Therapy. 30 (4): 485–496. doi:10.1089/hum.2018.117. ISSN 1043-0342. PMID 30261752.
- ↑ Yuzefovych, Yuliia; Valdivia, Emilio; Rong, Song; Hack, Franziska; Rother, Tamina; Schmitz, Jessica; Bräsen, Jan Hinrich; Wedekind, Dirk; Moers, Cyril; Wenzel, Nadine; Gueler, Faikah (2020). "Genetic Engineering of the Kidney to Permanently Silence MHC Transcripts During ex vivo Organ Perfusion". Frontiers in Immunology. 11: 265. doi: 10.3389/fimmu.2020.00265 . ISSN 1664-3224. PMC 7042208 . PMID 32140158.
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