Gene/Protein Disease Symptom Drug Enzyme Compound
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Query: UMLS:C0011849 (diabetes)
 277,896 document(s) hit in 31,850,051 MEDLINE articles (0.00 seconds)

Therapy of genetic diseases may be attempted at three different levels in the evolution of the disease process. At the first level, after clinical manifestations have appeared, methods are symptomatic and include diet, drugs, surgery, and avoidance of hazardous substances. At the second level, midway between the origin of the disease and the appearance of clinical manifestations, therapy consists of administration of a normal gene product, such as insulin in diabetes and factor VIII in hemophilia. At the third level, the origin of the disease, methods involve correcting the gene defect and are currently under investigation.
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PMID:Therapy of genetic diseases: a three-level approach. 692 69

An enzyme immuno assay kit has been developed to detect anti-HIV antibody in urine. In order to examine the clinical utility of the kit, 1333 urine samples were assayed. These samples consisted of 233 urine samples from HIV infected patients, 472 samples from HIV uninfected patients including 203 samples from patients with urogenital diseases, and 628 samples from normal subjects. Anti-HIV antibodies were detected in all the urine samples from HIV infected patients, and the diagnostic sensitivity for HIV infection was 100% with no false negative cases. A variety of anti-HIV antibody titers were found in the urine samples from HIV infected patients. However, no significant differences were found in the distribution patterns of urinary anti-HIV antibody titers among AC, ARC and AIDS patients. False positives were determined in only five samples in 628 healthy subjects (0.8%), one in 19 patients with hepatitis (5.3%), one in 45 patients with hemophilia (2.2%) and two in 105 pregnant women (1.9%). The antibody titers of all the false positive samples in these groups were less than the cut-off index multiplied by two. However, relatively high positive rates were demonstrated in the samples from urogenital diseases (11.8%), diabetes mellitus (20.0%) and auto-immune diseases (7.3%). False positive results were found to be directly correlated to the protein concentration of urinary protein, especially the immunoglobulin concentration in urine. The assay system was also evaluated by various reproducibility tests performed by different operators at different laboratories. The test results were satisfactory.(ABSTRACT TRUNCATED AT 250 WORDS)
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PMID:[Clinical usefulness of urinary anti HIV antibody test--a large scale study from 11 institutes in Japan]. 774 30

The authors established a means of effective gene transfer into human thyroid follicular cells via retroviral-mediated mechanisms. Using specific harvest and culture techniques, we investigated the selection of human thyroid cells in serum-free media. Normal adult human thyroid tissue was obtained after thyroidectomy from fresh specimens sent for frozen-section analysis. Follicular cells were harvested and grown in hormonally defined, serum-free media to prevent fibroblast growth with selection for differentiated function assessed by immunohistochemical staining for thyroglobulin. The efficiency of gene transfer into human thyroid cells was compared between the zen-beta-gal and LNL6 retroviral vectors. The zen-beta-gal retrovirus encodes the product beta-galactosidase, and gene expression was demonstrated by histochemical staining in 0.1% to 1% of the cells. An improved efficiency of 2% to 3% transduction was demonstrated using the LNL6 vector which carries the gene for neomycin resistance (NEO-R). Polymerase chain reaction (PCR) identification of the integrated proviral sequence (NEO-R gene) with Southern blot confirmation was used to quantitate LNL6 transductions and compare confluent versus actively dividing cell cultures. Follicular cell gene therapy has significant potential for treating congenital or acquired diseases of the thyroid as well as disorders of circulating proteins such as diabetes, hypopituitarism, and hemophilia. The ability to culture human follicular cells and perform effective gene transfer is paramount in the eventual realization of thyroid gene therapy.
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PMID:Gene transfer into human thyroid follicular cells. 796 61

The failure of patients to adhere adequately to prescribed medication and behavioral regimens is an important medical problem. Poor adherence is most common when the treatment regimen is preventive rather than curative, when patients are asymptomatic, and when the duration of treatment is long. For these reasons, adherence with dietary therapy for hypercholesterolemia is well recognized to be a significant clinical and research challenge. Medication adherence has been acknowledged to be a problem for those treatments with significant side effects, such as flushing and pruritus or the low palatability of bile acid sequestering agents. The availability of drugs that lack these effects has long been viewed as an important contribution to improving overall patient compliance. However, the literature on patient adherence with life-long treatment regimens that are simple and palatable (e.g., antihypertensives) suggests that while these improved treatments can enhance adherence, the overall rates of patient compliance still average only 50%. The fact that patients with heterozygous familial hypercholesterolemia are at high risk for early coronary artery disease and death if they fail to adhere to therapy is not sufficient to assure high rates of appropriate therapy over long periods of time, as demonstrated by the poor or erratic adherence commonly reported to treatments for other life-threatening diseases, such as advanced renal disease, hemophilia, and type I diabetes. The measurement of patient adherence to hypercholesterolemia therapy is often neglected in clinical practice and inadequate in hypercholesterolemia research.(ABSTRACT TRUNCATED AT 250 WORDS)
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PMID:Measuring adherence with therapy for chronic diseases: implications for the treatment of heterozygous familial hypercholesterolemia. 821 1

The authors investigate the in vitro component of an ex situ strategy for gene transfer into the thyroid gland using DNA complex and retroviral vectors. Canine follicular cells harvested by unilateral lobectomy and grown in low-serum media proliferated in culture and retained their differentiated state as evidenced by morphology and thyroglobulin expression. Transient and "stable" gene transfer in thyroid cells were evaluated by comparing DNA and retroviral transduction techniques. Effective gene transfer and expression was demonstrated by histochemical staining for the marker gene product beta-galactosidase. The efficiency of transduction was assessed using an amphotropic retroviral vector carrying the neomycin resistance gene and semiquantitative polymerase chain reaction (PCR) identification of integrated proviral sequences. This analysis demonstrated a proviral frequency in transduced cultures of 10% to 30%. Transduced cells showed no change in morphology or growth patterns and maintained differentiated function as assessed by antibody staining for thyroglobulin. The thyroid gland is an attractive target for somatic gene therapy because of its large protein-synthetic capacity, sensitivity to hormonal regulation, and proportionately high blood flow. Follicular cell gene therapy may be useful not only for treating congenital or acquired diseases of the thyroid, but also disorders of circulating proteins such as hypopituitarism, hemophilia, and diabetes.
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PMID:DNA- and viral-mediated gene transfer in follicular cells: progress toward gene therapy of the thyroid. 841 42

The ability to cross species lines will dramatically expand the number of patients and the scope of human diseases that can be treated successfully with transplantation. In addition to whole organs, the transplantation of cells and tissues with specific differentiated functions represents an important conceptual and medical advance. In the USA alone, over 15 million patients suffer from diabetes, over 7 million patients suffer from neurodegenerative diseases, and millions more suffer from liver failure, AIDS, hemophilia and other disorders caused by tissue loss or dysfunction. Clinical trials using animal cells to treat many of these diseases are already under way, and it seems likely that this list will continue to grow as researchers identify new bioactive molecules and expand their understanding of the role different cells play in the human disease process.
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PMID:Xenotransplantation of cells and tissues: application to a range of diseases, from diabetes to Alzheimer's. 949 69

Transplantation has become a successful method for the management of functional failure of a variety of tissues or organs. However, the majority of clinical transplantations use non-autologous allogeneic donor tissue implanted from one human to another. In order to prevent rejection of the allogeneic tissue, methods to overcome the immune barrier are necessary. Although prevention of organ rejection is currently achieved with pharmacological immune suppression, the undesirable side effects of this method have incited interest in novel methods to overcome the immune barrier. One such novel method of preventing immune reaction is immuno-isolation, in which the non-autologous tissues are physically isolated from the host tissues by placement in devices with perm-selective membranes. The membranes of these devices allow release of the therapeutic product required from the transplanted tissues, as well as diffusion of nutrients and waste necessary for survival of the non-autologous tissues. The membranes also prevent host immune mediators from contacting the non-autologous cells, thus preventing immune rejection. This technology has been tested for efficacy in large animal models, and is currently in the process of clinical trials in humans. This review will discuss the progress made in using immuno-isolation of non-autologous tissues in large animals. Immuno-isolation can be subdivided into two major areas of interest based on whether the non-autologous tissue used in the immuno-isolation device is genetically altered (gene therapy) or not. Studies using non-genetically altered non-autologous cells for immune-isolation have been dominated by the use of pancreatic islet cells for the treatment of diabetes. This work has been tested in large animal models of diabetes, including canine and primate model animals, and human clinical trials are underway. As well, there has also been work on treatment of neurological disorders such as Parkinson's disease or chronic pain using non-autologous immuno-isolated adrenal chromaffin cells or dopaminergic PC12 cells in large animals such as sheep and primates. This work will be reviewed in detail as to the types of disorders, immuno-isolation devices used and the type of large animals involved. Immune-isolation for gene therapy is a more recently developed field of research. In this case, the non-autologous cells used are first genetically altered to secrete a recombinant therapeutic product before placement in the immune-isolation devices. Genetic engineering of the non-autologous cells is beneficial, as it allows the use of a cell type that tolerates well the environment of the immune-isolation device, while still delivering the therapeutic product of interest. This form of gene therapy has been tested in our laboratory for delivery of marker products such as human growth hormone to canines. As several large animal models of human genetic disorders are available, such as canines affected with hemophilia or the lysosomal storage disease mucopolysaccharidosis, testing the efficacy of immuno-isolation for gene therapy in large animal models is an important prelude to human clinical trials. This review will discuss the topics outlined above, as well as some further considerations of the usefulness of large animal models in studying immune-isolation for non-autologous transplantation. Large animals may be more appropriate model organisms than rodents in which to study immune-isolation, as issues such as biocompatibility and immune response in a larger animal can be addressed. As well, large animal studies of immune isolation may provide data that are more relevant than rodent studies to the eventual application to human clinical trials.
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PMID:Non-autologous transplantation with immuno-isolation in large animals--a review. 961 31

In April and May 1997, a total of 100 general practitioners (GPs) in the Groot Gelre district of the Dutch General Practitioners Society were surveyed by phone for permission to send a questionnaire. 89 of 94 GPs who were sent a questionnaire replied, yielding a response rate of 89%. 78 of 89 GPs (88%) knew about the concept of pre-pregnancy health (PH) counseling and most of them already gave some kind of preconceptional advice (98% about folic acid, 93% about smoking, 88% about alcohol, 94% about various other substances, and 73% about the prevention of infections--toxoplasmosis, rubella). 87% of GPs questioned the patients about hereditary/congenital diseases that the child might inherit, 76% about hereditary diseases in the family, and 62% about diabetes. Less frequently occurring hereditary diseases were much less often asked about, such as Huntington's disease (14%), cystic fibrosis (19%), and hemophilia (14%). If the woman expressed the desire to have a child, 25% of GPs took an expanded case history. If a GP indicated a risk factor with regard to a potential pregnancy, 75% of them identified the possible consequences and informed the patient about them. 93% considered PH counseling part of their job responsibility and 91% were prepared to provide more PH care in the future. 53% of all GPs, however, indicated that they lacked sufficient knowledge to give adequate advice. The advantages of PH care were considered to outweigh possible disadvantages, such as medicalization of pregnancy, according to 74% of GPs. GPs appeared to lack time and appropriate knowledge, which indicates a need for postgraduate training.
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PMID:[Preconception counseling in family practice; a survey of 100 family physicians]. 962 54

The potential therapeutic applications of encapsulated cells are enormous. In the US alone, it has been estimated that nearly half-a-trillion dollars are spent each year to care for patients who suffer tissue loss or dysfunction. Over 6 million patients suffer from neurodegenerative disorders such as Alzheimer's disease and Parkinson's disease, over 14 million patients suffer from diabetes, and millions more from liver failure, hemophilia, and other diseases caused by the loss of specific vital cellular functions. It appears likely that by the end of the decade clinical trials of encapsulated cells to treat many of these diseases will become a reality. The Food and Drug Administration has already authorized studies to evaluate the safety and biological activity of several types of systems. A number of issues will have to be addressed, including the sourcing of raw materials, the design and building of manufacturing facilities, the scale-up and optimization process, storage and distribution of the product, and quality control.
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PMID:Encapsulated cell technology. 963 Oct 60

Genetic screening, gene therapy and other applications of genetic engineering are permissible in Judaism when used for the treatment, cure, or prevention of disease. Such genetic manipulation is not considered to be a violation of God's natural law, but a legitimate implementation of the biblical mandate to heal. If Tay-Sachs disease, diabetes, hemophilia, cystic fibrosis, Huntington's disease or other genetic diseases can be cured or prevented by "gene surgery," then it is certainly permitted in Jewish law. Genetic premarital screening is encouraged in Judaism for the purpose of discouraging at-risk marriages for a fatal illness such as Tay-Sachs disease. Neonatal screening for treatable conditions such as phenylketonuria is certainly desirable and perhaps required in Jewish law. Preimplantation screening and the implantation of only "healthy" zygotes into the mother's womb to prevent the birth of an affected child are probably sanctioned in Jewish law. Whether or not these assisted reproduction techniques may be used to choose the sex of one's offspring, to prevent the birth of a child with a sex-linked disease such as hemophilia, has not yet been ruled on by modern rabbinic decisions. Prenatal screening with the specific intent of aborting an affected fetus is not allowed according to most rabbinic authorities, although a minority view permits it "for great need." Not to have children if both parents are carriers of genetic diseases such as Tay-Sachs is not a Jewish option. Preimplantation screening is preferable. All screening test results must remain confidential. Judaism does not permit the alteration or manipulation of physical traits and characteristics such as height, eye and hair color, facial features and the like, when such change provides no useful benefit to mankind. On the other hand, it is permissible to clone organisms and microorganisms to facilitate the production of insulin, growth hormone, and other agents intended to benefit mankind and to cure and treat diseases.
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PMID:Judaism, genetic screening and genetic therapy. 984 72


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