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Gene therapy is a medical field which focuses on the genetic modification of cells to produce a therapeutic effect or the treatment of disease by repairing or reconstructing defective genetic material. The first attempt at modifying human DNA was performed in 1980 by Martin Cline, but the first successful nuclear gene transfer in humans, approved by the National Institutes of Health, was performed in May 1989. The first therapeutic use of gene transfer as well as the first direct insertion of human DNA into the nuclear genome was performed by French Anderson in a trial starting in September 1990. It is thought to be able to cure many genetic disorders or treat them over time.
Aplastic anemia is a disease in which the body fails to produce blood cells in sufficient numbers. Blood cells are produced in the bone marrow by stem cells that reside there. Aplastic anaemia causes a deficiency of all blood cell types: red blood cells, white blood cells, and platelets.
Hematopoietic stem-cell transplantation (HSCT) is the transplantation of multipotent hematopoietic stem cells, usually derived from bone marrow, peripheral blood, or umbilical cord blood. It may be autologous, allogeneic or syngeneic.
Wiskott–Aldrich syndrome (WAS) is a rare X-linked recessive disease characterized by eczema, thrombocytopenia, immune deficiency, and bloody diarrhea. It is also sometimes called the eczema-thrombocytopenia-immunodeficiency syndrome in keeping with Aldrich's original description in 1954. The WAS-related disorders of X-linked thrombocytopenia (XLT) and X-linked congenital neutropenia (XLN) may present with similar but less severe symptoms and are caused by mutations of the same gene.
Virotherapy is a treatment using biotechnology to convert viruses into therapeutic agents by reprogramming viruses to treat diseases. There are three main branches of virotherapy: anti-cancer oncolytic viruses, viral vectors for gene therapy and viral immunotherapy. These branches utilize three different types of treatment methods: gene overexpression, gene knockout, and suicide gene delivery. Gene overexpression adds genetic sequences that compensate for low to zero levels of needed gene expression. Gene knockout utilizes RNA methods to silence or reduce expression of disease-causing genes. Suicide gene delivery introduces genetic sequences that induce an apoptotic response in cells, usually to kill cancerous growths. In a slightly different context, virotherapy can also refer more broadly to the use of viruses to treat certain medical conditions by killing pathogens.
Metachromatic leukodystrophy (MLD) is a lysosomal storage disease which is commonly listed in the family of leukodystrophies as well as among the sphingolipidoses as it affects the metabolism of sphingolipids. Leukodystrophies affect the growth and/or development of myelin, the fatty covering which acts as an insulator around nerve fibers throughout the central and peripheral nervous systems. MLD involves cerebroside sulfate accumulation. Metachromatic leukodystrophy, like most enzyme deficiencies, has an autosomal recessive inheritance pattern.
CD34 is a transmembrane phosphoglycoprotein protein encoded by the CD34 gene in humans, mice, rats and other species.
The European Society of Gene and Cell Therapy is a non-profit organisation for educational and scientific purposes. The aim of ESGCT is to promote fundamental and clinical research in gene therapy, cell therapy, and genetic vaccines by facilitating education, the exchange of information and technology and by serving as a professional adviser to stakeholder communities and regulatory bodies in Europe.
Stem-cell therapy is the use of stem cells to treat or prevent a disease or condition. As of 2016, the only established therapy using stem cells is hematopoietic stem cell transplantation. This usually takes the form of a bone-marrow transplantation, but the cells can also be derived from umbilical cord blood. Research is underway to develop various sources for stem cells as well as to apply stem-cell treatments for neurodegenerative diseases and conditions such as diabetes and heart disease.
Molecular oncology is an interdisciplinary medical specialty at the interface of medicinal chemistry and oncology that refers to the investigation of the chemistry of cancer and tumors at the molecular scale. Also the development and application of molecularly targeted therapies.
Neal Stuart Young is an American physician and researcher, chief of the Hematology Branch of the National Institutes of Health (NIH), and Director of the Center for Human Immunology at the NIH in Bethesda, Maryland. He is primarily known for work in the pathophysiology and treatment of aplastic anemia, and is also known for his contributions to the pathophysiology of parvovirus B19 infection.
Elena Cattaneo is an Italian pharmacologist and co-founding director of the University of Milan's Center for Stem Cell Research.
Margaret (“Peggy”) A. Goodell is an American scientist working in the field of stem cell research. Goodell is Chair of the Department of Molecular and Cellular Biology at Baylor College of Medicine, Director of the Stem Cell and Regenerative Medicine (STaR) Center, and a member of the National Academy of Medicine. She is best known for her discovery of a novel method to isolate adult stem cells.
Helen Elisabeth Heslop is a physician-scientist from New Zealand whose clinical interests are in hematopoietic stem cell transplants. Heslop’s research interests are in immunotherapy to treat viral infections post transplant and hematologic malignancies. She is a professor in the Department of Medicine and Pediatrics at Baylor College of Medicine and the director of the Center for Cell and Gene Therapy at Baylor College of Medicine, Texas Children’s Hospital and Houston Methodist Hospital. She is also the Dan L. Duncan Chair and the associate director of clinical research at the Dan L. Duncan Cancer Center.
Betibeglogene autotemcel, sold under the brand name Zynteglo, is a medication for the treatment for beta thalassemia, a rare and potentially debilitating blood disorder. It was developed by Bluebird Bio and was given breakthrough therapy designation by the U.S. Food and Drug Administration in February 2015. It was approved for medical use in the European Union in May 2019.
Stefan Karlsson, M.D. Ph.D. is a Professor of Molecular Medicine and Gene Therapy at the Lund Stem Cell Center, in the Department of Laboratory Medicine, Lund University, Sweden. He is recognized for significant contributions to the fields of gene therapy and hematopoietic stem cell biology and in 2009 was awarded the Tobias Prize by The Royal Swedish Academy of Sciences.
Autologous CD34+ enriched cell fraction that contains CD34+ cells transduced with retroviral vector that encodes for the human ADA cDNA sequence, sold under the brand name Strimvelis, is a medication used to treat severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID).
Shimon Slavin, M.D., is an Israeli professor of medicine. Slavin pioneered the use of immunotherapy mediated by allogeneic donor lymphocytes and innovative methods for stem cell transplantation for the cure of hematological malignancies and solid tumors, and using hematopoietic stem cells for induction of transplantation tolerance to bone marrow and donor allografts.
Graziella Pellegrini is an Italian Professor of Cell Biology and the Cell Therapy Program Coordinator at the University of Modena and Reggio Emilia. She has developed and championed cell therapy protocols in hospitals across Italy.
Chen Hu was a Chinese military physician and stem cell researcher. He served as Director of the PLA Institute of Hematopoietic Stem Cell Research and the Beijing Hematopoietic Stem Cell Therapy Laboratory. Known for his research on hematopoietic stem cell therapy for leukemia, he was awarded the State Science and Technology Progress Award in 2015 and the Ho Leung Ho Lee Prize in 2016. In 2017, he and Deng Hongkui engineered resistance to HIV in mice using CRISPR gene editing, and for the first time used the technique on an AIDS patient. He died of a sudden heart attack before their findings were published.
References
- ↑ "Bordignon: «La terapia genica ci allungherà la vita (ma non troppo)»". Corriere della Sera (in Italian). 14 September 2018. Retrieved 13 November 2019.
- ↑ "Thirteenth World Conference - The Lives to Come - Claudio Bordignon". www.thefutureofscience.org. Retrieved 13 November 2019.
- ↑ "Claudio_Bordignon". researchgate.net.
- ↑ "Claudio Bordignon". www.thepharmaletter.com. Retrieved 13 November 2019.
- ↑ "ESGCT Awards".
- ↑ "Press corner". European Commission - European Commission. Retrieved 13 November 2019.