Found inside – Page 44By altering an organism ' s genes , scientists can change particular characteristics of the organism . ... Although still in its infancy , gene therapy offers hope for people with inherited disorders like cystic fibrosis and sickle cell ... Some medical researchers fear that implanting genetically altered cells in humans might result in unplanned effects , such as an increased risk of cancers or other diseases . The book highlights important recent developments and discusses the next steps that will be required for further improvement of the life expectancy and quality of life of CF patients. Written by world authorities, this reference explores the utilization and advancement of state-of-the-art imaging modalities for the analysis of pulmonary function-providing an up-to-date and global perspective of imaging applications with ... Found inside – Page 18Fetal therapy means that preventive medicine needn ' t eliminate the patient at risk . ... Considered individually rather than in the aggregate , single - gene defects are quite rare , with cystic fibrosis , for example , one of the commonest of the ... "The purpose of this thesis is to investigate the social cost motivations associated with paying for genetic therapies at the beginning of life for monogenic diseases from various perspectives including the social perspective and the ... Found insideRecognizing the potential design complexities and ethical issues associated with clinical trials for gene therapies, the Forum on Regenerative Medicine of the National Academies of Sciences, Engineering, and Medicine held a 1-day workshop ... Found inside – Page 54Other Biological Risk Factors We have already said that the brain is a complex organ . ... For example , gene replacement or gene therapy may be possible now or in the future for some chronic health conditions ( e . g . , cystic fibrosis ) , but it ... Found inside – Page 166... if somatic cell therapy develops into a successful cure for single gene defects with high prevalence , as in cystic fibrosis ... to evaluate not only technical risks of effects on the human gene pool but political , social , and moral impacts as well . Found insideThis book will be invaluable and entertaining for anyone who is involved in the care of patients with cystic fibrosis. The emerging field of prenatal gene therapy is founded on scientific and technical advances in fetal medicine, molecular biology and gene therapy. Found inside – Page 108Gene therapy with plasmid vectors has been used to attempt to treat cystic fibrosis (see Chapter 3) and cancer in humans (see Chapter 10). Risks of Nonviral ... Found inside – Page 11I battle every day with cystic fibrosis just to be able to breathe . ... With gene therapy , I am also willing to take calculated risks . Found inside – Page 2954Gene dose of apolipoprotein E type 4 allele and the risks of Alzheimer's disease in late onset families . Science . 1993 ; 261 ... Wilfond BS , Fost N. The cystic fibrosis gene : medical and social implications for heterozygote detection . JAMA . ... Walters L. Human gene therapy : ethics and public policy . Hum Gene Ther . Found inside – Page 86At this point, because gene therapy is "still essentially experimental, ... cells in the lungs and the pancreas in cystic fibrosis" (Davis, 1991b, p. Herpes simplex virus vectors / William F. Goins [und weitere] -- 6. Adeno-Associated Viral (AAV) vectors / Nicholas Muzyczka -- 7. Regulatory RNA in gene therapy / Alfred. S. Lewin -- 8. Found inside – Page 93GENE THERAPY RISKS The first death associated with gene therapy occurred on September 18 , 1999 , at the ... This has been observed in several clinical trials with cystic fibrosis and ADA deficiency patients , whose cure faded after a few ... Human Genome Editing considers important questions about the human application of genome editing including: balancing potential benefits with unintended risks, governing the use of genome editing, incorporating societal values into clinical ... Found inside – Page 288Gene transfer studies indicate that “ the procedure appears to carry a very low risk of adverse reactions ” ( 17 ) . ... research with animals , for example , the apparently successful reversal of cystic fibrosis in mice by gene therapy in utero ( 16 ) . Found inside – Page 14For example , the efficiency of activated , cancer would not necessarily result since gene transfer through physical , chemical , and ... to be useful in treatment of such genetic therapeutic genes . disorders as cancer , cystic fibrosis , and hemophilia , Because safety is such ... models , allowing the methods to be better I sincerely thank Rena Kono and Dr. Janette Lewis studied to ensure less risk to humans . Found inside – Page 46... diabetes , and cystic fibrosis fuels boundless optimism . Yet much about gene therapy is still unknown ; its short- and long - term risks are still the ... Found inside – Page 258The diagnosis of genetic diseases is achieved by examining the patient and obtaining a family pedigree . ... The patient and the family should be informed , according to their specific inheritance patterns , of the following risks of future occurrence of disorders ... THERAPY Dietary replacement of enzymes ( e.g. , patients having cystic fibrosis with damage to the pancreas are treated with lipase , trypsin , etc. ) ... Found inside – Page 1257Gene therapy , introducing genes to cure a genetic disease such as cystic fibrosis , carries risks as does all new medical treatments . There may well be problems with insurance and testing but are these any different from those related to ... Given the general interest in CFTR, this collection will appeal to a broad readership with interests in CFTR, cystic fibrosis, ion channels and ABC transporters. The multifaceted topics surrounding gene therapy DNA, techniques, challenges, and risks are presented in this clear, understandable text, so that readers can grasp the ramifications and ethical concerns of this incredible technology. Is it ever permissible to use a child as a means to an end? How much authority should parents have over decisions about research involving their children? Should children or their parents be paid for participation in research? Found insidepulmonary complications of cystic fibrosis. ... Institute of Health), the first human gene therapy trial on cystic fibrosis was initiated on April 17, 1993. Found inside – Page 191Now, with data that clearly show serious health risks of SCT and benefits of early recognition, the advantages of heterozygote detection are generally ... This book presents a current assessment of this rapidly evolving field, offering principles for actions and research and recommendations on key issues in genetic testing and screening. Found inside – Page 129Gene therapy is the technology that aims to alter the genes that are responsible for certain disorders — the insertion or removal of target genes for therapeutic purposes . ... These include AIDS , lung cancer , cystic fibrosis , glioblastoma , and kidney cancer . ... that compromise patient safety to ensure that there is adequate control of risks and that the process of informed consent is stringently followed . Offers a reference guide to pregnancy and childbirth, discussing morning sickness, back pain, gestational diabetes and breast-feeding with a week-by-week guide to changes in both mother and baby. Original. Found inside – Page 679Weighed against these potential benefits are certain risks The privacy of the patients and all confidentiality issues will ... Cystic fibrosis . J Thorac Cardiovasc Surg 1992 ; protein produced by the CFTR gene in AdayCFTR . 10 may be 103 ... Found inside – Page 260G GENE THERAPY Ethical and Social Issues During the 1970s , biologists began to map DNA directly from the entire genomes of viruses and bacteria ... in addition to classic genetic diseases such as cystic fibrosis and Gaucher's disease ( a disorder in which fats build up in the cells ) ... therapy review process was based . questions about gene therapy have to do with the anticipated benefits and risks of ... Found inside – Page 1267Normal results Successful gene therapy would cure the disease being treated . Resources BOOKS ... These ailments include acquired immunodeficiency syndrome ( AIDS ) , cystic fibrosis , and muscular dystrophy . Adenosine ... The brain , heart , eyes , Risks The risks of gene therapy are largely unknown . Some agents ... Found insideThis book is a collection of chapters dealing with examples of RNA and DNA viruses, and issues such as how these gene packages have learnt to take advantage of their hosts, molecular recognition events that hosts may use to counterattack ... Found inside – Page 32Juengst ( 1995 ) has criticized Gene Marking the argument that germ - line gene therapy is rendered superfluous by the availability of ... Germline gene therapy is the only definite measure one can offer to prevent fatal diseases like cystic fibrosis and sickle cell disease . ... can produce a non - defective embryo as there are no functional copies of the defective gene to select ( Latchman , 1994 ) . Risks . Found insideThis book provides detailed, up-to-date topics addressing basic principles of gene therapy and discussing some of the challenges encountered by scientists in developing this relatively novel technology. This book is the first to cover the entire field of this complex disease, and encompasses the rapidly moving topics of the basic molecular and cellular biology as well as the recent multi-system, multi-disciplinary advances in the clinical ... Karl Drlica's Double-Edged Sword: The Promises and Risks of the Genetic Revolution explores both the positive and negative aspects of modern genetics and its potential impact on us as individuals. The book provides most basic principles of immunopharmacology by equally emphasizing the disciplines of immunology and pharmacology, which will be a very valuable tool for both the professional as well as graduate students. Found insideSome gene therapies target stem or progenitor cells , because they can divide and move . 13. ... What are some of the complications that have slowed the development of gene therapy ? 13. ... Cell Type Vector fibroblast AV Disease Target Duchenne muscular dystrophy Alzheimer disease sickle cell disease cystic fibrosis 9. The title of this book derives from C. Wright Mills’ classic The Sociological Imagination (Penguin, 1970), in which he sees the essential project of social science as the use of the imagination to 'grasp history and biography and the ... Found inside – Page 177Women who inherit a mutation in one of these genes face a high risk of eventually developing breast or ovarian cancer ... with cystic fibrosis has been increasing gradually with better treatments for chronic lung disease ; dietary therapy is ... It emphasizes methods, tools, and experimental approaches used by leaders in the field of translational gene therapy. The book promotes cross-disciplinary communication between the sub-specialties of medicine, and remains unified in theme. 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