GP64-pseudotyped lentiviral vectors target liver endothelial cells and correct hemophilia A mice.

Lentiviral vectors (LV) are efficient vehicles for in vivo gene delivery to the liver. LV integration into the chromatin of target cells ensures their transmission upon proliferation, thus allowing potentially life-long gene therapy following a single administration, even to young individuals. The glycoprotein of the vesicular stomatitis virus (VSV.

Gene transfer and genome editing for familial hypercholesterolemia.

Familial hypercholesterolemia (FH) is an autosomal dominant inherited disease characterized by high circulating low-density lipoprotein (LDL) cholesterol. High circulating LDL cholesterol in FH is due to dysfunctional LDL receptors, and is mainly expressed by hepatocytes. Affected patients rapidly develop atherosclerosis, potentially leading to myocardial infarction and death within the third decade of life if left untreated.

Liver-directed lentiviral gene therapy corrects hemophilia A mice and achieves normal-range factor VIII activity in non-human primates.

Liver gene therapy with adeno-associated viral (AAV) vectors delivering clotting factor transgenes into hepatocytes has shown multiyear therapeutic benefit in adults with hemophilia. However, the mostly episomal nature of AAV vectors challenges their application to young pediatric patients. We developed lentiviral vectors, which integrate in the host cell genome, that achieve efficient liver gene transfer in mice, dogs and non-human primates, by intravenous delivery.

Review of the tribe Hyperaspidini Mulsant (Coleoptera: Coccinellidae) from Iran.

The Iranian species of the tribe Hyperaspidini Mulsant, 1846 (Coleoptera: Coccinellidae) are reviewed. The current list includes 12 species, all placed in a single genus Hyperaspis Chevrolat, 1836. Hyperapsis asiatica Lewis, 1896 and H.

An annotated checklist of Microweiseinae and Sticholotidini of Iran (Coleoptera, Coccinellidae).

An updated checklist of the Coccinellidae species of the former subfamily Sticholotidinae recorded from Iran is provided. Eleven species are reported: two species classified presently in the subfamily Microweiseinae (in the genera Paracoelopterus Normand, 1936 and Serangium Blackburn, 1889), and nine species classified in the tribe Sticholotidini of the subfamily Coccinellinae (in the genera Coelopterus Mulsant & Rey, 1852 and Pharoscymnus Bedel, 1906). Pharoscymnus smirnovi Dobzhansky, 1927 is removed from the list of the Coccinellidae of Iran.

Revision of the subgenus Orthoscymnus Canepari of Scymnus Kugelann (Coleoptera, Coccinellidae), with descriptions of four new species.

The subgenus Orthoscymnus Canepari, 1997 of Scymnus Kugelann, 1794 is herein revised. Seven species of the Orthoscymnus fauna are recognized, of which four species, Scymnus (Orthoscymnus) jilongicus sp. n.

Fauna Europaea: Coleoptera 2 (excl. series Elateriformia, Scarabaeiformia, Staphyliniformia and superfamily Curculionoidea).

Fauna Europaea provides a public web-service with an index of scientific names (including synonyms) of all living European land and freshwater animals, their geographical distribution at country level (up to the Urals, excluding the Caucasus region), and some additional information. The Fauna Europaea project covers about 230,000 taxonomic names, including 130,000 accepted species and 14,000 accepted subspecies, which is much more than the originally projected number of 100,000 species. This represents a huge effort by more than 400 contributing specialists throughout Europe and is a unique (standard) reference suitable for many users in science, government, industry, nature conservation and education.

Selective serotonin re-uptake inhibitors (SSRIs) for preventing migraine and tension-type headaches.

Background: Headache is a common medical problem. In view of recent discoveries about the role of serotonin in pain mechanisms, selective serotonin re-uptake inhibitors (SSRIs) have been evaluated for the prevention of migraine and tension-type headaches (TTH).

Objectives: To evaluate the efficacy and tolerability of SSRIs for preventing migraine and TTH.

[Neurofibromatosis (NF1) and neuroleprosy: immunoreaction against pathologic Schwann-cells. Physiopathogenetic observations].

Background: We reviewed the literature evaluating the immune reaction in neurofibromatosis (NF1) and neuroleprosy, so as to underline the immunopathegenetic parallelism and the possible therapeutic implications regarding the treatment of these two disorders. In particular we evaluated the systemic modifications and the local fibrotic events that lead to nerve damage in NF1 and complete neuronal destruction as in leprosy.

Methods: With the above aim in mind we studied the histology, histochemistry and immunohistochemistry (Schwann cells and immunoglobulins) of four plexiform neurofibroma, one common neurofibroma and one case of borderline neuroleprosy (BT).

Tumour suppressor genes, immunology and local manifestations of neurofibromatosis phenotypes.

Research on Neurofibromatosis (NF) has been directed at understanding what determines disease quiescence, exacerbation, and the possible malignant evolution. Studies on NF have examined the role of genetic oncosuppression in the evolution of the defence against the non-self. Paraffin fixed specimens of benign and malignant neoplasia, occurring in patients with NF1 and NF2, were tested for the presence of p53: a reliable marker of genetic oncosupression.

[The clinico-immunological surveillance of neurofibromatosis].

Considering the more recent physiopathogenetic advancements in neurofibromatosis (NF), we propose to employ novel instrumental and laboratory procedures for the immunological and clinical surveillance of NF. In NF the evolution of the non-self can lead to disease expansion, at times transforming into malignancy. Contemporarily, the resulting immunological reactivity can either lead to the type of fibrosis that one sees in paraneoplastic connective tissue disease or be deficient.

Sneddon syndrome: cerebral perfusion studies by Tc99m HM-PAO and SPECT.

Sneddon syndrome is a non-inflammatory, non-atherosclerotic disease involving small and medium-sized arteries of the brain and of the skin. The arteriographic examination is often negative despite progressive impairment of the neurological status. In 3 patients with Sneddon syndrome cerebral perfusion was assessed with single photon emission computerized tomography (SPECT) and technetium99 m-hexamethylpropylenamineoxime (Tc99 HM PAO).

Prognostic relevance of hormonal and kinetic parameters in CNS neoplasms.

With the present study we investigated both the presence of cytoplasmic estrogen (E-Rc) and progesterone (P-Rc) receptor, nuclear E-R (E-Rn) and the percentage of cycling tumor cells with Ki-67 MAB in 32 CNS tumor specimens. The main purpose of our study was to evaluate a possible correlation between the hormonal and kinetic parameters and the clinical and neuroradiological follow-up.

A case of Vogt-Koyanagi-Harada syndrome in a patient affected by Hodgkin's disease.

The Vogt-Koyanagi-Harada (VKH) syndrome, a disease of unknown origin, characterized by inflammation of the uveal tract, retina and meninges, was observed in a patient with Hodgkin's disease (HD) who had been treated with chemotherapy and radiotherapy. Ten years later the patient developed a cluster of neurological symptoms and signs (fever, pain, bilateral hypoacousia, dizziness, uveitis and psychiatric disorder) which were initially interpreted as a relapse of HD. The diagnosis of VKH syndrome was thereafter suspected from the clinical findings, response to therapy and further evolution.

[Apropos of a case of cluster headache and a space-occupying lesion in the brain].

Reference is made to a patient with an approximately 20-yr history of cluster headache and crises refractory to medical prophylaxis for 2 years prior to admission. Over the last 30 days, the picture had been complicated by resistance to ergotamine and motor deficiency in the right leg. Neuroradiological investigation revealed a lesion occupying the left parieto-occipital space.

Brain-stem haematoma with complete recovery.

A case of CT-diagnosed brain-stem haematoma associated with signs of dorsal medullary involvement is reported. The patient recovered completely within 1 month.

Changes of biological rhythms in primary headache syndromes.

The circadian periodicity of some endocrine (PRL, cortisol, GH) and vegetative (oral temperature, blood pressure) functions has been studied in cluster headache, common migraine, atypical facial pain, and "mixed" headache. Changes in several biological rhythms have been found not only in cluster headache (CH) but also in other kinds of headache. Although a great individual variability of rhythometric changes has been observed, particularly in CH, the dysrhythmic condition seems to be more evident in chronic than in episodic CH.

Chronobiological aspects of headache syndromes due to sellar or pituitary pathology.

A chronobiological study was carried out in headache syndromes due to empty sella or to pituitary G.H.- and PRL-secreting adenomas.

Neuroendocrinological aspects of depression and symptomatological picture.

Cortisol and prolactin circadian rhythms were determined in a group of depressed hospitalized women who were submitted the next day to the dexamethasone suppression test. The depressed were classified as endogenous and non-endogenous according to symptomatological criteria. Some significant differences were found between the two groups when compared to control subjects in cortisol as well as prolactin biorhythms, and the cortisol suppression percentages were significantly lower in the endogenous than in the non-endogenous.