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Genetic therapy for congenital myopathies
Current Opinion in Neurology, 2021Purpose of review There has been an explosion of advancement in the field of genetic therapies. The first gene-based treatments are now in clinical practice, with several additional therapeutic programs in various stages of development.
Nika, Maani +2 more
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Gene Therapy for Genetic Diseases
Cancer Investigation, 1989(1989). Gene Therapy for Genetic Diseases. Cancer Investigation: Vol. 7, No. 2, pp. 179-192.
D B, Kohn, W F, Anderson, R M, Blaese
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Genetic therapies for cardiovascular diseases
Trends in Molecular Medicine, 2005Recent advances in understanding the molecular and cellular basis of cardiovascular diseases, together with the availability of tools for genetic manipulation of the cardiovascular system, offer possibilities for new treatments. Gene therapies have demonstrated potential usefulness for treating complex cardiovascular diseases, such as hypertension ...
Luis G, Melo +3 more
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New England Journal of Medicine, 1987
Inherited disorders have been perceived by the general medical profession as unfortunate accidents of nature that are essentially untreatable.
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Inherited disorders have been perceived by the general medical profession as unfortunate accidents of nature that are essentially untreatable.
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Expert Opinion on Biological Therapy, 2003
Despite the tremendous success of highly active antiretroviral treatment (HAART) introduced nearly 8 years ago for the treatment of human immunodeficiency virus (HIV), innovative therapies, including gene transfer approaches, are still required for nearly half of the general patient population.
Ananthalakshmi, Poluri +2 more
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Despite the tremendous success of highly active antiretroviral treatment (HAART) introduced nearly 8 years ago for the treatment of human immunodeficiency virus (HIV), innovative therapies, including gene transfer approaches, are still required for nearly half of the general patient population.
Ananthalakshmi, Poluri +2 more
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Genetic therapies and xenotransplantation
Expert Opinion on Investigational Drugs, 1999There is an acute shortage of human organs available for transplantation. Transplanting animal organs or tissues is a potential solution and could also provide a novel means of gene delivery. Xenotransplantation faces many challenges, including the immune response of the recipient against the transplant, the physiological limitations of the transplant ...
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Gene therapy for genetic diseases
Pediatrics International, 1998Abstract Gene therapy provides the potential to permanently cure selected genetic diseases. However, a major obstacle is the effective delivery of the normal gene to specific target sites of pathology and continuous expression at therapeutic levels. A variety of viral and non‐viral vectors have been developed to deliver genes to various cells, tissues ...
R J, Desnick, E H, Schuchman
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Genetic prodrug activation therapy
Molecular Medicine Today, 1997Genetic prodrug activation therapy shows promise as a therapeutic option for the treatment of cancer as well as a variety of other diseases. It involves the insertion of a gene encoding a drug-metabolizing enzyme into cells and the systemic administration of a prodrug. The prodrug is converted to a cytotoxic agent by the action of the expressed enzyme.
A, Rigg, K, Sikora
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Genetic therapies to lower cholesterol
Vascular Pharmacology, 2015This review surveys the state-of-the-art in genetic therapies for familial hypercholesterolaemia (FH), caused most commonly by mutations in the LDL receptor (LDLR) gene. FH manifests as highly elevated low density lipoprotein (LDL) cholesterol levels and consequently accelerated atherosclerosis.
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Ethical Issues in Genetic Therapy
Journal of Obstetric, Gynecologic & Neonatal Nursing, 1994Ethical issues in genetic therapy involve the use of genetic science for human good and the avoidance of preventable harms. Immediate issues are the control and confidentiality of results of genetic testing, possible discrimination against those found to have genetic diseases, and the just allocation of beneficial genetic therapies. Less practical, but
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