نتایج جستجو برای: adeno associated virus

تعداد نتایج: 1852842  

Journal: :Brain : a journal of neurology 2012
Serge Herson Faycal Hentati Aude Rigolet Anthony Behin Norma B Romero France Leturcq Pascal Laforêt Thierry Maisonobe Rim Amouri Hafedh Haddad Muriel Audit Marie Montus Carole Masurier Bernard Gjata Christophe Georger Mustapha Cheraï Pierre Carlier Jean-Yves Hogrel Ariane Herson Yves Allenbach François M Lemoine David Klatzmann H Lee Sweeney Richard C Mulligan Bruno Eymard Didier Caizergues Thomas Voït Olivier Benveniste

γ-Sarcoglycanopathy or limb girdle muscular dystrophy type 2C is an untreatable disease caused by autosomal recessively inherited mutations of the γ-sarcoglycan gene. Nine non-ambulatory patients (two males, seven females, mean age 27 years; range 16-38 years) with del525T homozygous mutation of the γ-sarcoglycan gene and no γ-sarcoglycan immunostaining on muscle biopsy were divided into three ...

2004
Marcio O. Lasaro Hildegund C.J. Ertl

Abbreviations: Adeno-associated virus, (AAV); bacilli Calmette-Guerin, (BCG); base pairs, (bp); cottontail rabbit papilloma virus, (CRPV); E6-associated protein, (E6-AP); epidermal growth factor receptor, (EGFR); histone deacetylases, (HDAC); human papillomavirus, (HPV); inverted terminal repeats, (ITRs); listerolysin O, (LLO); of lysosome-associated membrane protein, (LAMP); open reading frame...

Journal: :Circulation research 2014
Serena Zacchigna Lorena Zentilin Mauro Giacca

The use of vectors based on the small parvovirus adeno-associated virus has gained significant momentum during the past decade. Their high efficiency of transduction of postmitotic tissues in vivo, such as heart, brain, and retina, renders these vectors extremely attractive for several gene therapy applications affecting these organs. Besides functional correction of different monogenic disease...

Journal: :Cell & gene therapy insights 2022

Adenoviruses (Ad) have a long history as DNA-transfer vehicles in various medical applications including vivo gene therapy [1]. In this context, the favorable safety profile of Ad has been considered major benefit, since lack integration into host cell genome eliminates potential risks associated with insertional mutagenesis. This is combined comparatively high packaging capacity for foreign DN...

Journal: :Journal of virology 1984
J E Janik M M Huston J A Rose

The three primary capsid proteins (A, B, and C) of adeno-associated viruses have been shown previously to contain overlapping amino acid sequences (R. McPherson and J. Rose, J. Virol. 46:523-529, 1983). In the present study we demonstrate definitively that these proteins are encoded in the right half of the adeno-associated virus 2 genome, and one or both of the smallest adeno-associated RNA sp...

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