Gene/Protein Disease Symptom Drug Enzyme Compound
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Query: UMLS:C0026850 (muscular dystrophy)
 5,870 document(s) hit in 31,850,051 MEDLINE articles (0.00 seconds)

Technical development has produced a curious phenomenon, the ebbing of the moral imperative of reproduction. After 2000 years of fight against diseases and death, the respect for life has declined threatening the destruction of Western civilization and family by destroying unborn babies via abortion. Artificial in vitro experimentation can potentially lead to elimination of very young, old, or sick people. Dr. Bernard Nathanson in his book "Aborting America" called abortion infanticide, one of the most abominable crimes. The beginning of life start at conception as shown by recent extra-corporal, in vitro fertilization resulting in a viable fetus, as in the case of Luisa Brown who was conceived in a tube by Drs. Edwards' and Steptoe's technique. The creation of human embryo banks and experimentation on human embryos amount to biological pronography. Respect for the human species and reproduction should manifest itself in the fight against sterility and genetic diseases, such as muscular dystrophy, hemophilia, Down's syndrome, and Huntington's chorea. The fight against AIDS and the elimination of the risk of contracting it by contaminated blood is also a medical priority. In the end, the question still remains: can science itself save the world without moral imperatives, is not the dilemma of Faust and the vileness of Mephistopheles conjured with the nuclear experience and human experimentation.
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PMID:[The human meaning of life]. 250 45

Gene therapy has generated enormous scientific, medical and public interest over the last decade. Clinical trials involving approximately 2000 patients worldwide have targeted simple genetic diseases such as cystic fibrosis, muscular dystrophy, adenosine deaminase deficiency, Gaucher's disease and familial hypercholesterolemia, as well as complex acquired diseases such as cancer and AIDS. The central nervous system is a new and particularly exciting target for gene therapy because its unique properties prevent the successful treatment of many neurological disorders by conventional means. This review discusses the potential applications of in vivo gene therapy to neurological disorders that have the greatest potential for genetic treatments.
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PMID:Towards gene therapy for the central nervous system. 985 68

A major technological revolution is on the horizon that promises cures for inherited diseases such as cystic fibrosis, sickle cell anemia, muscular dystrophy, hemophilia, leukocyte-adhesion deficiency, and Gaucher's disease. This revolutionary technology also promises effective treatments for acquired diseases such as cancer and AIDS. The technique is gene therapy and it has already been used in humans. Some applications raise ethical considerations that not only affect the individual who is treated but have implications for future generations as well.
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PMID:The current clinical revolution. Applications of gene therapy in treatment of disease. 1013 65

Briefly stated my point is that the well-being of each person in a community conceived abstractly may be all too easily sacrificed for the sake of the abstraction. Physicians may offer critically ill patients places in programs of experimental treatment, but there is commonly a catch to the offer. To take part in a program of clinical experiment a patient must not only risk a possible failure of a fresh drug and the chance of destructive side effects from the drug, but the patients must risk only getting the traditional treatment along with a placebo rather than the experimental drug. Placebo control, double blind critical protocols for testing effects of fresh drugs on critically ill patients are a commonplace. I question the scientific objectivity of the protocols and the underlying ethic, and suggest use of alternate protocols. Experimental tests in the treatment of gram-negative bacteria blood infections, muscular dystrophy, and AIDS and AIDS-related diseases are examples.
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PMID:We have a prejudice against ourselves -- sentiment, ethics, and reason. 1164 7

Nuclear factor-kappaB (NF-kappaB) is a major transcription factor that plays an essential role in several aspects of human health including the development of innate and adaptive immunity. The dysregulation of NF-kappaB is associated with many disease states such as AIDS, atherosclerosis, asthma, arthritis, cancer, diabetes, inflammatory bowel disease, muscular dystrophy, stroke, and viral infections. Recent evidence also suggests that the dysfunction of NF-kappaB is a major mediator of some human genetic disorders. Appropriate regulation and control of NF-kappaB activity, which can be achieved by gene modification or pharmacological strategies, would provide a potential approach for the management of NF-kappaB related human diseases. This review summarizes the current knowledge of the physiological and pathophysiological functions of NF-kappaB and its possible role as a target of therapeutic intervention
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PMID:Nuclear factor-kappaB: its role in health and disease. 1517 63

Skeletal muscle atrophy/wasting is a serious complication of a wide range of diseases and conditions such as aging, disuse, AIDS, chronic obstructive pulmonary disease, space travel, muscular dystrophy, chronic heart failure, sepsis, and cancer. Emerging evidence suggests that nuclear factor-kappa B (NF-kappaB) is one of the most important signaling pathways linked to the loss of skeletal muscle mass in various physiological and pathophysiological conditions. Activation of NF-kappaB in skeletal muscle leads to degradation of specific muscle proteins, induces inflammation and fibrosis, and blocks the regeneration of myofibers after injury/atrophy. Recent studies employing genetic mouse models have provided strong evidence that NF-kappaB can serve as an important molecular target for the prevention of skeletal muscle loss. In this article, we have outlined the current understanding regarding the role of NF-kappaB in skeletal muscle with particular reference to different models of muscle wasting and the development of novel therapy.
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PMID:Nuclear factor-kappa B signaling in skeletal muscle atrophy. 1857 72

Basic fibroblast growth factor (bFGF, FGF-2) has an inhibitory effect on the expression of the myostatin gene in murine C2C12 myoblasts, as shown in our recent investigation. To further verify the regulatory effects of bFGF on the myostatin gene and to better understand its mechanism in skeletal muscle, and to promote clinical applications of bFGF to treat skeletal muscle diseases correlated to muscular dystrophy or AIDS and so on, recombinant human bFGF (rh-bFGF) was added into media and stimulated murine C2C12 myoblasts to investigate the dose-dependent effect of bFGF on suppression of myostatin gene expression and the role of extracellular signal-regulated kinase 1/2 (ERK1/2) in the regulatory mechanism. Simultaneously, complete coding sequence of ovine?8 kDa-bFGF gene was inserted into eukaryotic vector pCMV-neo (originated from pEGFP-N1 vector, from which the EGFP gene has been removed), the recombinant plasmid pCMV-neo-bFGF was harvested and injected into the mouse skeletal muscle of posterior limb. Expression levels of bFGF, myostatin, and ERK1/2 genes in murine C2C12 myoblasts and the skeletal muscle were analyzed by real-time reverse transcription-polymerase chain reaction and Western blotting analysis, respectively. The results showed that bFGF impaired the expression of myostatin gene in a dose-dependent manner in C2C12 cells, with increasing concentration of rh-bFGF, myostatin mRNA declined gradually. In addition, results in skeletal muscle indicated that bFGF also suppressed the expression of the myostatin gene in vivo. Furthermore, we found ERK1/2 participated in the regulatory mechanism of bFGF on the expression of the myostatin gene.
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PMID:Roles of extracellular signal-regulated kinase 1/2 on the suppression of myostatin gene expression induced by basic fibroblast growth factor. 1898 75

Growth hormone (GH) exercises its growth effects by stimulating insulin-like growth factor I (IGF-I) synthesis in the liver (endocrine IGF-I) and by inducing chondrocyte differentiation/replication and local production of IGF-I (paracrine/autocrine IGF-I). Injectable recombinant human (rh)IGF-I (mecasermin) has been available for nearly 20 years for treatment of the rare instances of GH insensitivity caused by GH receptor defects or GH-inhibiting antibodies. Full restoration of normal growth, as occurs with rhGH replacement of GH deficiency, is not seen, presumably because only the endocrine deficiency is addressed. RhIGF-I has also been effective as an insulin-sensitizing agent in severe insulin-resistant conditions. Although the insulin-sensitizing effect may benefit both type 1 and type 2 diabetes, there are no ongoing clinical trials because of concern about risk of retinopathy and other complications. Promotion of rhIGF-I for treatment of idiopathic short stature has been intensive, with neither data nor rationale suggesting that there might be a better response than has been documented with rhGH. Other applications that have either been considered or are undergoing clinical trial are based on the ubiquitous tissue-building properties of IGF-I and include chronic liver disease, cystic fibrosis, wound healing, AIDS muscle wasting, burns, osteoporosis, Crohn's disease, anorexia nervosa, Werner syndrome, X-linked severe combined immunodeficiency, Alzheimer's disease, muscular dystrophy, amyotrophic lateral sclerosis, hearing loss prevention, spinal cord injury, cardiovascular protection, and prevention of retinopathy of prematurity. The most frequent side effect is hypoglycemia, which is readily controlled by administration with meals. Other common adverse effects involve hyperplasia of lymphoid tissue, which may require tonsillectomy/adenoidectomy, accumulation of body fat, and coarsening of facies. The anti-apoptotic properties of IGF-I are implicated in cancer pathogenesis-a concern for long-term therapy. It is unlikely that mecasermin will be useful beyond the orphan indications of severe insulin resistance and GH insensitivity.
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PMID:Mecasermin (recombinant human insulin-like growth factor I). 1919 69

Protease inhibitors (PIs) are regulatory proteins found in numerous animal tissues and fluids, plants, and microorganisms that reduce and inhibit the exacerbated and uncontrolled activity of the target proteases. Specific PIs are also effective tools for inactivating proteases involved in human diseases like arthritis, pancreatitis, hepatitis, cancer, AIDS, thrombosis, emphysema, hypertension, and muscular dystrophy among others. Plant PIs-small peptides with a high content of cystine residues in disulfide bridges-possess a remarkable resistance to heat treatment and a high stability against shifts in pH, denaturing agents, ionic strength, and proteolysis. In recent years, novel biologic activities have been reported for plant PIs, including antimicrobial, anticoagulant, antioxidant action plus inhibition of tumor-cell growth; thus pointing to possible applications in medicine, agriculture, and biotechnology. In this review, we provide a comparative overview of plant-PIs classifying them in four groups according of their thermal and pH stability (high stability and hyperstable -to temperature and to pHs-, respectively), then emphasizing the relevance of the physicochemical characteristics of these proteins for potential biotechnological and industrial applications. Finally, we analyze the biologic activities of the stable protease inhibitors previously characterized that are the most relevant to potential applications in biomedicine, the food industry, and agriculture.
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PMID:Biotechnological, biomedical, and agronomical applications of plant protease inhibitors with high stability: A systematic review. 3200