Annals of the New York Academy of Sciences
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Ann. N. Y. Acad. Sci. · Jan 2005
A phase I/II clinical trial of beta-globin gene therapy for beta-thalassemia.
Recent success in the long-term correction of mouse models of human beta-thalassemia and sickle cell anemia by lentiviral vectors and evidence of high gene transfer and expression in transduced human hematopoietic cells have led to a first clinical trial of gene therapy for the disease. A LentiGlobin vector containing a beta-globin gene (beta(A-T87Q)) that produces a hemoglobin (Hbbeta(A-T87Q)) that can be distinguished from normal hemoglobin will be used. The LentiGlobin vector is self-inactivating and contains large elements of the beta-globin locus control region as well as chromatin insulators and other features that should prevent untoward events. The study will be done in Paris with Eliane Gluckman as the principal investigator and Philippe Leboulch as scientific director.
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Ann. N. Y. Acad. Sci. · Jan 2005
Comparative StudyMethods for noninvasive measurement of tissue iron in Cooley's anemia.
To examine the relationship between myocardial storage iron and body iron burden, as assessed by hepatic storage iron measurements, we studied 22 patients with transfusion-dependent thalassemia syndromes, all being treated with subcutaneous deferoxamine, and 6 healthy subjects. Study participants were examined with a Philips 1.5-T Intera scanner using three multiecho spin echo sequences with electrocardiographic triggering and respiratory navigator gating. Myocardial and hepatic storage iron concentrations were determined using a new magnetic resonance method that estimates total tissue iron stores by separately measuring the two principal forms of storage iron, ferritin and hemosiderin. ⋯ By contrast, in the subset of 10 patients with beta-thalassemia major, the correlation between the 5-year average of hepatic iron concentration and the current myocardial storage iron was significant (R = .67, P = .03). In these patients, myocardial storage iron concentrations seem to reflect the control of body iron over a period of years. Magnetic resonance methods promise to provide more effective monitoring of iron deposition in vulnerable tissues, including the liver, heart, and endocrine organs, and could contribute to the development of iron-chelating regimens that more effectively prevent iron toxicity.
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Ann. N. Y. Acad. Sci. · Dec 2004
Randomized Controlled Trial Comparative Study Clinical TrialSubarachnoid analgesia in advanced labor: a comparison of subarachnoid analgesia and pudendal block in advanced labor: analgesic quality and obstetric outcome.
Pain control during labor is a primary objective of antalgic therapy. The use of the peridural as an elective procedure for labor analgesia is now corroborated by the international scientific community. Sometimes a combined spinal-peridural procedure is used together with the intrathecal administration of opioids to also cover the first stage of labor. ⋯ In Group P, however, 10 minutes after placement of the pudendal nerve block, 40 patients reported no improvement in pain symptomatology during contractions and only 16 reported less painful contractions (P < 0.0001). The duration of spinal analgesia (128 +/- 38 minutes) was enough in most cases for delivery to be completed. These results suggest that low-dose bupivacaine-fentanyl spinal analgesia represents an important option for pain relief in late labor, not the least because the procedure does not upset the dynamics of delivery or alter vital parameters and is welcomed by women in labor who are still able to collaborate actively in the birth of their baby.
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Ann. N. Y. Acad. Sci. · Dec 2004
ReviewCost-effective violence prevention through targeted family interventions.
Efforts in violence prevention can focus on individual youth, their families, their schools, or the communities in which they live. Among the small number of program models that have been proven effective in repeated replications, those that focus on improving family management and child care have been found to be the most cost-effective. ⋯ All of these programs involve detailed protocols, extensive staff training and supervision, and quality-assurance procedures. The factors limiting their wider adoption include staff resistance to their structured approach, cost-sharing issues between local and state levels of government, and the political power of existing programs.
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Ann. N. Y. Acad. Sci. · Dec 2004
Clinical TrialPsychosocial stress-induced activation of salivary alpha-amylase: an indicator of sympathetic activity?
Assessment of sympathoadrenal medullary system (SAM) activity is only possible to date via measurement of catecholamines in blood plasma or via electrophysiological methods. Both ways of measurement are restricted to endocrinological or psychophysiological laboratories, as both require either immediate freezing of blood samples or complex recording devices. Efforts have therefore been undertaken to find a method comparable to salivary cortisol measurements, in which noninvasive samples can be taken at any place and stored at room temperature for sufficient time before later analysis in the laboratory. ⋯ We further report that sAA exhibits a stable circadian pattern that mirrors that of salivary cortisol. In conclusion, the current data show that salivary alpha-amylase may serve as an easy-to-use index for SAM activity. However, some questions remain to be answered; for example, what impact does salivary flow rate exert on stress-induced sAA activity?