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.