Results 11 to 20 of about 9,435,635 (153)

Terminal glucose as a receptor for adeno-associated virus 44.9 [PDF]

Journal of Virology
The isolation of novel adeno-associated virus (AAV) serotypes has led to significant advances in our understanding of parvovirus biology and vector development for gene therapy by identifying isolates with unique cell tropism and increased efficiency of ...
Giovanni Di Pasquale, Hemon Tehrani, Ida Shinder, Sandra Afione, John A. Chiorini   +4 more
doaj   +2 more sources

Fluorescence Microscopy in Adeno-Associated Virus Research

Viruses, 2023
Research on adeno-associated virus (AAV) and its recombinant vectors as well as on fluorescence microscopy imaging is rapidly progressing driven by clinical applications and new technologies, respectively.
Susanne K. Golm, Wolfgang Hübner, Kristian M. Müller   +2 more
doaj   +3 more sources

Adeno-associated virus serotype 9 antibodies in patients screened for treatment with onasemnogene abeparvovec

Molecular Therapy: Methods & Clinical Development, 2021
Spinal muscular atrophy is a progressive, recessively inherited monogenic neurologic disease, the genetic root cause of which is the absence of a functional survival motor neuron 1 gene. Onasemnogene abeparvovec (formerly AVXS-101) is an adeno-associated
John W. Day, Richard S. Finkel, Eugenio Mercuri, Kathryn J. Swoboda, Melissa Menier, Rudolf van Olden, Sitra Tauscher-Wisniewski, Jerry R. Mendell   +7 more
doaj   +2 more sources

Adeno-associated virus for cystic fibrosis gene therapy

Brazilian Journal of Medical and Biological Research, 2011
Gene therapy is an alternative treatment for genetic lung disease, especially monogenic disorders such as cystic fibrosis. Cystic fibrosis is a severe autosomal recessive disease affecting one in 2500 live births in the white population, caused by ...
S.V. Martini, P.R.M. Rocco, M.M. Morales
doaj   +4 more sources

A novel polyethyleneimine-coated adeno-associated virus-like particle formulation for efficient siRNA delivery in breast cancer therapy: preparation and in vitro analysis [PDF]

International Journal of Nanomedicine, 2012
Wei Shao1, Arghya Paul1, Sana Abbasi1, Parminder S Chahal2, Jimmy A Mena2, Johnny Montes2, Amine Kamen2, Satya Prakash11Biomedical Technology and Cell Therapy Research Laboratory, Department of Biomedical Engineering and Artificial Cells and Organs ...
Shao W, Paul A, Abbasi S, Chahal PS, Mena JA, Montes J, Kamen A, Prakash S   +7 more
doaj   +2 more sources

Development of Therapies for Spinal Muscular Atrophy Using Gene Therapy and Nanotechnology [PDF]

, 2013
Spinal muscular atrophy (SMA) is a genetic disease which is characterized by muscle weakness and atrophy. The disease arises from mutations in the survival motor neuron 1 (SMN1) gene causing degeneration of spinal cord motor neurons.
Little, Daniel
core   +5 more sources

Progress in the use of adeno-associated viral vectors for gene therapy [PDF]

, 2004
The development of safe and efficient gene transfer vectors is crucial for the success of gene therapy trials. A viral vector system promising to meet these requirements is based on the apathogenic adeno-associated virus (AAV-2), a member of the ...
Markus Braun-Falco, Hallek, M., Michael Hallek, Buning, H., Braun-Falco, M., Hildegard Büning, Büning, H;Braun-Falco, M;Hallek, M, Büning, Hildegard;Braun-Falco, Markus;Hallek, Michael   +7 more
core   +1 more source

Subretinal delivery of adeno-associated virus serotype 2 results in minimal immune responses that allow repeat vector administration in immunocompetent mice [PDF]

, 2009
Background: Adeno-associated virus serotype 2 (AAV2) vectors show considerable promise for ocular gene transfer. However, one potential barrier to efficacious long-term therapy is the development of immune responses against the vector or transgene ...
Ali, R.R., Barker, S.E., Robbie, S.J., Smith, A.J., Natkunarajah, M., Buch, P., Balaggan, K.S., Duran, Y., MacLaren, R.E., Bainbridge, J.W. B., Broderick, C.A.   +10 more
core   +1 more source

Efficient delivery of Cre-recombinase to neurons in vivo and stable transduction of neurons using adeno-associated and lentiviral vectors [PDF]

, 2004
BACKGROUND: Inactivating genes in vivo is an important technique for establishing their function in the adult nervous system. Unfortunately, conventional knockout mice may suffer from several limitations including embryonic or perinatal lethality and the
Hermens, Wim T J M C, Lieberman, A., Eggers, R., Verhaagen, J., Sablitzky, Fred, Chakravarthy, Sridhara, Zhang, Jing Ying, Zhang, J.Y., Hermens, Wim TJMC, Zhang, Jing, Verhaagen, J, Ahmed, Bushra, Anderson, P.N., Eggers, R, Eggers, Ruben, Levelt, Christiaan, Chakravarthy, S, Levelt, C., Ahmed, BY, Verhaagen, Joost, Lieberman, A.R., Hermens, WTJMC, Lieberman, AR, Anderson, Patrick N., Ahmed, Bushra Y, Hermens, W.T., Niclou, SP, Anderson, PN, NICLOU, Simone P., Lieberman, A R, Zhang, JY, Anderson, Patrick N, Sablitzky, F., Levelt, C, Sablitzky, F, Niclou, S.P., Chakravarthy, S., Ahmed, B.Y.   +37 more
core   +1 more source

Muscle regeneration affects Adeno Associated Virus 1 mediated transgene transcription

Scientific Reports, 2022
Duchenne muscular dystrophy is a severe neuromuscular disease causing a progressive muscle wasting due to mutations in the DMD gene that lead to the absence of dystrophin protein.
Amédée Mollard, Cécile Peccate, Anne Forand, Julie Chassagne, Laura Julien, Pierre Meunier, Zoheir Guesmia, Thibaut Marais, Marc Bitoun, France Piétri-Rouxel, Sofia Benkhelifa-Ziyyat, Stéphanie Lorain   +11 more
doaj   +1 more source