Aledo-Serrano, Angel Gil-Nagel, Antonio Isla, Julian Mingorance, Ana Mendez-Hermida, Fernando Hernandez-Alcoceba, Ruben
Published in
Orphanet Journal of Rare Diseases
The COVID-19 pandemic is adding an unanticipated concern for those affected by genetic diseases. Most of the new treatment achievements for these patients are made possible as a result of advances in viral-based products. Among them, adenoviruses (AdV) and especially adeno-associated viruses (AAV) are important players. The concerns and the convers...
Srivastava, Arvind Mallela, Krishna M G Deorkar, Nandkumar Brophy, Ger
Published in
Journal of pharmaceutical sciences
Adeno-associated virus (AAV) has emerged as a leading platform for gene delivery for treating various diseases due to its excellent safety profile and efficient transduction to various target tissues. However, the large-scale production and long-term storage of viral vectors is not efficient resulting in lower yields, moderate purity, and shorter s...
Bower, Jacquelyn J Song, Liujiang Bastola, Prabhakar Hirsch, Matthew L
Published in
Viruses
Adeno-associated virus (AAV) was first characterized as small "defective" contaminant particles in a simian adenovirus preparation in 1965. Since then, a recombinant platform of AAV (rAAV) has become one of the leading candidates for gene therapy applications resulting in two FDA-approved treatments for rare monogenic diseases and many more current...
Meier, Anita Felicitas; Tobler, Kurt; Michaelsen, Kevin; Vogt, Bernd; Henckaerts, Els; 130609; Fraefel, Cornel;
Wild-type adeno-associated virus (AAV) can only replicate in the presence of helper factors, which can be provided by coinfecting helper viruses such as adenoviruses and herpesviruses. The AAV genome consists of a linear, single-stranded DNA (ssDNA), which is converted into different molecular structures within the host cell. Using high-throughput ...
Nagai, Jun Bellafard, Arash Qu, Zhe Yu, Xinzhu Ollivier, Matthias Gangwani, Mohitkumar R Diaz-Castro, Blanca Coppola, Giovanni Schumacher, Sarah M Golshani, Peyman
...
Published in
Neuron
Astrocytes respond to neurotransmitters and neuromodulators using G-protein-coupled receptors (GPCRs) to mediate physiological responses. Despite their importance, there has been no method to genetically, specifically, and effectively attenuate astrocyte Gq GPCR pathways to explore consequences of this prevalent signaling mechanism in vivo. We repo...
May, Francis J. Head, PamelaSara E. Venturoni, Leah E. Chandler, Randy J. Venditti, Charles P.
Published in
Molecular Therapy. Methods & Clinical Development
We describe an adeno-associated virus (AAV) gene therapy vector that targets the central nervous system (CNS) in a murine model of methylmalonic acidemia (MMA). The AAV is intravenously delivered and leads to methylmalonyl-CoA mutase expression in the CNS, with a concomitant reduction in circulating disease markers.
Prakoso, Darnel Tate, Mitchel Blasio, Miles J. De Ritchie, Rebecca H.
Published in
Clinical Science (London, England : 1979)
Diabetes increases the prevalence of heart failure by 6–8-fold, independent of other comorbidities such as hypertension and coronary artery disease, a phenomenon termed diabetic cardiomyopathy. Several key signalling pathways have been identified that drive the pathological changes associated with diabetes-induced heart failure. This has led to the...
Strings-Ufombah, Vanessa Malerba, Alberto Kao, Shih-Chu Harbaran, Sonal Roth, Fanny Cappellari, Ornella Lu-Nguyen, Ngoc Takahashi, Keiko Mukadam, Sophie Kilfoil, Georgina
...
Published in
Molecular Therapy. Nucleic Acids
Alanine expansions of PABPN1 cause oculopharyngeal muscular dystrophy. Administration of a single AAV9 vector expressing a corrected version of the PABPN1 gene and two siRNAs in miRNA backbones to silence endogenous PABPN1 can restore muscle size, weight, and force in an OPMD surrogate mouse model to wild-type levels.
Song, G G Lee, Y H
Published in
Zeitschrift fur Rheumatologie
Der Behandlungsansatz mit MMF erwies sich als Alternative zu CYC mit gleicher Wirksamkeit, und in Bezug auf die Sicherheit war MMF vergleichbar mit CYC bei Patienten mit aktiver AAV, was darauf hindeutet, dass MMF als Alternative zu CYC für die Induktion einer Remission bei AAV eingesetzt werden kann.
Zubair, Muhammad; 113963; Murris, Sjoerd R; 112308; Isa, Kaoru; Onoe, Hirotaka; Koshimizu, Yoshinori; Kobayashi, Kenta; Vanduffel, Wim; 10250; Isa, Tadashi;
To understand the connectome of the axonal arborizations of dopaminergic midbrain neurons, we investigated the anterograde spread of highly sensitive viral tracers injected into the ventral tegmental area (VTA) and adjacent areas in 3 macaques. In 2 monkeys, injections were centered on the lateral VTA with some spread into the substantia nigra, whi...
