«The Concept of Gene Therapy. The Clinical Spectrum. Gene Delivery Strategies. THE DELIVERY VECTOR. RETROVIRAL VECTORS.. ADENOVIRAL VECTORS.. ...» Document abstract
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Language : english
medical studies
presentation
date published
20/07/2007
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SummaryGene therapy is an experimental form of treatment whereby sequences of nucleic acids (i.e., genes) are delivered to cells to change their biologic function. The concept initially arose as replacement therapy for monogenic inherited disorders. For these disorders, the aim is to replace a defective gene with the normal counterpart. The delivered genetic material undergoes transcription and translation using the host cell's machinery, leading to in situ production of the normal protein and thereby correction of the phenotypic defect.
Table of Contents- The Concept of Gene Therapy
- The Clinical Spectrum
- Gene Delivery Strategies
- THE DELIVERY VECTOR
- RETROVIRAL VECTORS.
- ADENOVIRAL VECTORS.
- ADENO-ASSOCIATED VIRUS
- Selected Disease Applications
- CYSTIC FIBROSIS
- HEMOPHILIA
- MYOCARDIAL AND PERIPHERAL ISCHEMIA
- HIV INFECTION AND AIDS
- REPLICATION-COMPETENT VIRUSES ("VIROTHERAPY")
- Recommendations
« society. Without such things as gene therapy which is part of DNA technology many medical feats would not have been accomplished. DNA ...» Document abstract
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2 word format
Language : english
biology
presentation
date published
02/10/2007
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SummaryDNA technology is vital to our technological progress as a society. Without such things as gene therapy which is part of DNA technology many medical feats would not have been accomplished. DNA manipulation is used in many aspects of life, ranging from farms to hospitals and the gene cloning we attempted in our lab was but a precursor to all these great advances.
Table of Contents- Introduction
- The DNA manipulation can lead to many advances and understanding how DNA works is important not only for the scientist but also for the general public
- Gel electrophoresis is a method used to separate DNA fragments for analysis and size comparison
- In the second week of the DNA lab (Part II), we resuspended the DNA pellet in 20µl TE buffer and vortexed the solution
- After everything was done, we mixed the restriction enzyme digests with the blue dye solution containing xylene cyanol and bromophenol blue
- Written Results
- The control gels in the experiment did not run out all the way making the result hard to interpret.
- The BglI cut at around 1700 and 1400 and the EcoRI and BglI cut at 1400 and 1600 base pairs, because the lightest ones go the farthest.
- Discussion
« In fact, gene therapy was recently first reported successful to "cure" severe combined immunodeficiency disease (Cavazzana-Calvo and colleagues, 2000). ...» Document abstract
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3 word format
Language : english
medical studies
school essay
date published
19/11/2007
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SummaryMedical writers have recently turned to the opening line of Charles Dickens's A Tale of Two Cities to describe these prevailing times in medicine and obstetrics as "the best of times. . . the worst of times. . ." (Grumbach, 1999; Morrison, 2000). Why are these times at once the best and worst of times for obstetrics? There are many reasons for this, and some are now considered.
Table of Contents- The chronicle of maternal and infant mortality during the 20th century described earlier should suggest that much is good in the health care of women and their infants'indeed, better than it has ever been.
- The year 2000 marks the 50th anniversary of the American College of Obstetricians and Gynecologists
- There are a number of other indicators from the National Center for Health Statistics and the Centers for Disease Control and Prevention that indicate African-American women are at a significant reproductive disadvantage.
- Other pressing problems include the erosion of health-care safety nets for the uninsured by for-profit plans that all too often quickly abandon the most vulnerable patients when profits are in jeopardy (Bodenheimer, 1997).
- The cost for medical malpractice'insurance premiums, attorney fees, and settlements and awards also continues to erode the health-care dollar.
- Attempts to mitigate some of these contentious issues have been only partially successful because of watered-down versions of legislations engineered by the plaintiffs bar.
« are all advancing rapidly. Progress is also expected in enzyme replacement and gene replacement therapy. The next decade will see ...» Document abstract
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2 word format
Language : english
finance
school essay
date published
14/09/2007
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level : General public
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SummaryAs noted earlier in this report, Biogen Idec Inc. is an amalgamation of strengths. It has been the merger between IDEC Pharmaceuticals and Biogen. These two pharmaceutical giants have been as one corporation only as of November of 2003; therefore, there have been a very small number of developments within the company. The main focus will be on the merger itself.
The merger was completed on November the 12th establishing a global biotechnology leader under the symbol “BIIB” on the Nasdaq exchange. William H. Rastetter, Ph.D., the company's Executive Chairman, explains, "Based on [their] existing collaboration in oncology, [they] knew that this combination would be an excellent fit, both operationally and culturally.” The Board of Directors of Biogen Idec is consistent of twelve directors, six from each company.
The merger was completed on November the 12th establishing a global biotechnology leader under the symbol “BIIB” on the Nasdaq exchange. William H. Rastetter, Ph.D., the company's Executive Chairman, explains, "Based on [their] existing collaboration in oncology, [they] knew that this combination would be an excellent fit, both operationally and culturally.” The Board of Directors of Biogen Idec is consistent of twelve directors, six from each company.
Table of Contents- The merger was completed on November the 12th establishing a global biotechnology leader under the symbol 'BIIB' on the Nasdaq exchange
- Recent developments in the industry
- The new technology in development will help rid the human and animal world of a wide array of diseases by fabricating specific antibodies to ward off specific parasites
- Ethical issues facing industry
- Once the final stage of FDA approvals go through, drugs are to be marketed and sold to the public
- We feel that one of their internal successes is ideal staffing
- The above factors has proved to add to the endless potential for growth for Biogen
« JJ; Meyer, L.; Persoz, A.; Theodorou, I.; Rouzioux, C.. "Improved virological response to highly active antiretroviral therapy in HIV CCR5 receptor gene and HIV ...» Document abstract
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2 word format
Language : english
international relations
research papers
date published
19/12/2007
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level : General public
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SummaryGenetic mutations are not uncommon. They can happen for a variety of reasons and have a variety of effects. Those with negative effects are weeded out by natural selection, and those with positive effects prevail and are accumulated due to natural selection. These mutations can be caused by a variety of reasons such as copying errors during cell division, exposure to ultraviolet rays and viruses, or can happen deliberately (Wikipedia). Most mutations have no effect on a species or its individuals because DNA repair is quite successful in fixing most mistakes before they become permanent. But there are very few mutations which have beneficial consequences. One such mutation that results in benefits for a species/individuals that make it up, is the CCR5-Delta32 mutation. During this mutation the Delta32 base pair is deleted in human CCR5 which results in immunity to HIV Type 1 (if homozygous) or increased resistance to AIDS (if heterozygous) (Wikipedia). The very controversial current argument on this subject asks several questions: if the absence of the CCR5-Delta32 is ultimately beneficial to humans, or if its negative side effects outweigh the benefits; as well as, where the origins of this mutation are.
« on chromosome 3. The regulatory region in the SRIF gene is upstream from W: Nonpeptide antagonists of neuropeptide receptors: Tools for research and therapy. ...» Document abstract
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7 word format
Language : english
biology
research papers
date published
26/11/2007
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level : Advanced
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SummaryThe past several decades have witnessed a veritable explosion of knowledge about the central nervous system (CNS), and in no area has this been as impressive as in peptide neurobiology. Numerous peptide neurotransmitter candidates have been identified and characterized, their CNS distributions mapped, and their genes cloned. The tenet “one neuron-one transmitter” erroneously attributed to Dale has been convincingly refuted with numerous demonstrations of neurons containing multiple peptides or combinations of peptide and nonpeptide neurotransmitters. Additionally, since the early 1980s there has been an embarrassment of riches in the form of knowledge about neurotransmitter receptor diversity, diversity of receptor-effector coupling, and neurotransmitter transporters. These discoveries have not yet been fully integrated into what is known about normal or aberrant CNS function, although dysfunction at virtually any level could conceivably lead to neuropsychiatric deficits.
Table of Contents- By definition, a neuropeptide is a chain of two or more amino acids linked by peptide bonds, and differs from other proteins only in the length of the amino acid chain.
- Many of the known behavioral effects of neuropeptides are observed only after their direct injection into the CNS because most peptides do not penetrate the blood-brain barrier in amounts sufficient to produce effects before being inactivated by serum and tissue enzymes that degrade them.
- The tertiary structure for recognition is also used by the immune system for the production of specific antibodies, as well as by biological receptors.
- Neuropeptides are found throughout the CNS, as well as in various peripheral organs, such as the gastrointestinal tract, pancreas, and adrenal glands.
- In the cortex of rats SRIF is found in some of the large stellate-shaped neurons and in abundance among the fusiform-shaped, nonpyramidal neurons of layers II to V, and particularly in layer V of the sensory cortex.
- Through the use of retrograde tracing methods and dual staining techniques, several pathways for certain peptides have now been delineated.
- Some of the noradrenergic locus ceruleus neurons, in turn, project to the hypothalamic paraventricular nucleus where their input increases CRF synthesis and release.
- The neurotensin-neuromedin N gene was originally cloned from canine ileal mucosa, and complementary deoxyribonucleic acid (cDNA) probes constructed against this form were used to clone the rat gene.
« gene, the dopamine transporter gene, and the D2 dopamine receptor gene. smoking cessation counseling sessions, which does not constitute supportive therapy. ...» Document abstract
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5 word format
Language : english
biology
presentation
date published
19/02/2008
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level : Advanced
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SummaryThe goal of this paper is to evaluate the significance of the co-morbidity of nicotine abuse in adolescents afflicted with ADHD (Attention-Deficit Hyperactivity Disorder). Due to the nature of the disorder and its possible underlying pathophysiology of dopaminergic dysfunction, the use of nicotine can be implicated as a form of self-medication. I will provide evidence of the co-morbidity and early onset of nicotine dependence and prevalence of smoking habit in adolescents with ADHD. Furthermore, I will discuss prenatal exposure to nicotine and its behavioral consequences, followed by an analysis of the pharmacology of nicotine and proposed circuitry of ADHD. Nicotine use in ADHD adolescents can no longer be attributed to mainstream “peer pressure". There are reinforcing effects provided by nicotine that parallel stimulant medication used to treat the disorder.
« So far, the metabotropic receptor gene (mGluR) family has been shown to W: Nonpeptide antagonists of neuropeptide receptors: Tools for research and therapy. ...» Document abstract
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3 word format
Language : english
psychology
research papers
date published
26/11/2007
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level : Advanced
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SummaryGlutamate Receptors Glutamate receptors are found throughout the brain and are expressed on both neurons and glia, although not all glutamate receptor subtypes are found on both cell types. Glutamate receptors, sometimes referred to as excitatory amino acid receptors, were initially classified into N-methyl-D-aspartate (NMDA), quisqualate, and kainate receptors on the basis of their preferential activation by these exogenous agonists. More recently, five categories of glutamate receptors (NMDA, kainate, a-amino-3-hydroxy-5-methylisoxazole-4-propionic acid [AMPA], L-2-amino-4-phosphonobutyrate (L-AP4), and trans-1-aminocyclopentane-1,3-dicarboxylic acid [ACPD] receptors) have been established on the basis of pharmacological, electrophysiological, and molecular biological criteria. The L-AP4 receptor type is defined by its agonist and acts as an inhibitory autoreceptor, while the quisqualate receptors of the previous classification have been subdivided by means of more-specific agonists into AMPA and ACPD receptors. AMPA and Kainate receptors are sometimes collectively referred to as non-NMDA receptors. NMDA, kainate and AMPA receptors are ionotropic glutamate receptors; the L-AP4 and ACPD receptors are grouped as metabotropic receptors. Ionotropic receptors are ligand-gated cation-specific channels that are activated rapidly (milliseconds), whereas metabotropic receptors coupled to G proteins and second-messenger systems function more slowly on a scale of several hundred milliseconds to seconds.
Table of Contents- AMPA Receptors Recent cloning efforts have clearly demonstrated that AMPA and kainate receptors are distinct receptor complexes, although they can be activated by the same agonists.
- Kainate Receptors Although kainate is an effective agonist of AMPA receptors, it also activates its own distinct class of ionotropic receptors, the kainate-preferring receptors.
- NMDA receptors have a number of distinct recognition sites for endogenous and exogenous ligands, each with discrete binding domains. At present there are at least seven pharmacologically distinct sites through which compounds can alter the activity of this receptor.
- Metabotropic Receptors Not as much is known about the last group of glutamate receptors, the metabotropic receptors.
« age- and sex-matched controls found no differences in the gene sequence in W: Nonpeptide antagonists of neuropeptide receptors: Tools for research and therapy. ...» Document abstract
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11 word format
Language : english
biology
research papers
date published
26/11/2007
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level : Advanced
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SummaryNeuropeptide receptors have undergone the same process of discovery and characterization that receptors for other neurotransmitters have enjoyed. The process begins with the pharmacological characterization of the receptor's physicochemical binding properties by assessing the affinity of various metabolically derived and synthetic peptide fragments, and the native molecule, for the receptor binding site found in membrane preparations. Peptide receptor locations are mapped with radioactive or fluorescent tags that are inserted into peptide molecules, which often contain substituted amino acids at the most vulnerable peptidase cleavage sites. Previously, once the peptide receptor was characterized pharmacologically, it was usually purified from some relatively enriched biological tissue source or brain region by affinity column chromatography. After it had been purified, binding parameters and activity were recharacterized for the reconstituted purified receptor protein and structural information obtained by X-ray crystallography. This process was closely followed in the purification of the neurotensin-neuromedin N receptor.
Table of Contents- The neurotensin receptor was first characterized by photoaffinity labeling and cross-linking of radioiodinated ligands, which resulted in two labeled subunits of about 49 Kd and 51 Kd from rat brain synaptosomes.
- The much more powerful tools of molecular biology have been utilized more recently.
- Neuropeptide receptors have been associated with just about every type of second messenger signal transduction system that has been identified.
- Peptides are degraded to smaller fragments, and eventually to single amino acids, by specific enzymes termed peptidases.
- The metabolism of TRH has been investigated fairly completely, principally because of the limited number of fragments that can be generated from a tripeptide.
- The peptides involved in neuroendocrine regulation have cell bodies residing in the hypothalamus that receive feedback from all levels of the endocrine axes.
- Regional differences in CRF receptor regulation by corticosterone have also been reported, which have been shown to partly result from differential glycosylation of the CRF receptor.
- Alzheimer's Disease Dementia of the Alzheimer's Type represents up to two thirds of the demented population encountered in clinical practice, and over half of the nursing home beds in the United States are currently occupied by such patients.
- The CRF-containing interneurons of the cortex are also consistently depleted in Alzheimer's disease. As with SRIF, subcortical areas containing CRF neurons may be spared, but unlike SRIF, CRF receptors are increased in number (up-regulated) with no change in affinity.
- Corticotropin-Releasing Factor After a search spanning nearly three decades, CRF was isolated and characterized in 1981 as a 41-amino acid peptide.
- A series of studies have demonstrated significant elevations of CRF concentrations in the CSF of drug-free patients with major depression or following suicide.
- Like many other neuropeptide transmitters, central administration of SRIF produces a variety of behavioral and physiological effects.
- Decreased neurotensin concentrations in CSF have been reported in several populations of patients with schizophrenia when compared to controls or patients with other psychiatric disorders.
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