Results 11 to 20 of about 326,770 (247)

AAV gene therapy vectors in the TMJ

Clinical and Experimental Dental Research, 2022
AbstractObjectivesThe goal of this project was to evaluate the use of two adeno‐associated viral vector serotypes, adeno‐associated viral vectors (AAV)‐2 and AAV‐6, approved for and used for gene therapy in humans, for the delivery of therapeutic genes to the temporomandibular joint (TMJ) and the attendant sensory nerves.MethodsYoung adult wild‐type ...
Sabine M. Brouxhon, Michael Kerry O'Banion, Stephanos Kyrkanides   +2 more
openaire   +4 more sources

T Cell-Mediated Immune Responses to AAV and AAV Vectors [PDF]

Frontiers in Immunology, 2021
Adeno-associated virus (AAV)-mediated gene transfer has benefited patients with inherited diseases, such as hemophilia B, by achieving long-term expression of the therapeutic transgene.
Hildegund C. J. Ertl
doaj   +3 more sources

Dual AAV Vectors for Stargardt Disease

, 2017
Stargardt disease (STGD1), due to mutations in the large ABCA4 gene, is the most common inherited macular degeneration in humans. Attempts at developing gene therapy approaches for treatment of STGD1 are currently ongoing. Among all the vectors available for gene therapy of inherited retinal diseases, those based on adeno-associated viruses (AAV) are ...
Trapani I., Ivana Trapani
openaire   +4 more sources

Capsid Modifications for Targeting and Improving the Efficacy of AAV Vectors

Molecular Therapy: Methods & Clinical Development, 2019
In the past decade, recombinant vectors based on a non-pathogenic parvovirus, the adeno-associated virus (AAV), have taken center stage as a gene delivery vehicle for the potential gene therapy for a number of human diseases.
Hildegard Büning, Arun Srivastava
doaj   +2 more sources

Common AAV gene therapy vectors show nonselective transduction of ex vivo human brain tissue

Molecular Therapy: Methods & Clinical Development
The ability to deliver a therapeutic sequence to a specific cell type in the human brain would make possible innumerable therapeutic options for some of our most challenging diseases; however, studies on adeno-associated virus (AAV) vector tropism have ...
JP McGinnis, Joshua Ortiz-Guzman, Maria Camila Guevara, Sai Mallannagari, Benjamin D.W. Belfort, Suyang Bao, Snigdha Srivastava, Maria Morkas, Emily Ji, Angela Addison, Evelyne K. Tantry, Sarah Chen, Ying Wang, Zihong Chen, Kalman A. Katlowitz, Jeffrey J. Lange, Melissa M. Blessing, Carrie A. Mohila, M. Cecilia Ljungberg, Guillermo Aldave, Ali Jalali, Akash Patel, Sameer A. Sheth, Howard L. Weiner, Shankar Gopinath, Ganesh Rao, Akdes Serin Harmanci, Daniel J. Curry, Benjamin R. Arenkiel   +28 more
doaj   +2 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

AAV Vectors for the Nucleolus [PDF]

Molecular Therapy, 2012
During the replication of wild-type adeno-associated virus (AAV), viral capsids are assembled in the nucleolus,1 and the capsid proteins interact with the nucleolar proteins nucleolin (NCL) and nucleophosmin (NPM1), possibly promoting their transport from the cytoplasm into the nucleolus.2,3 In the case of replication-incompetent AAV vectors, the ...
openaire   +2 more sources

Chemically Defined, High-Density Insect Cell-Based Expression System for Scalable AAV Vector Production

Molecular Therapy: Methods & Clinical Development, 2020
The recombinant adeno-associated virus (AAV) vector is one of the most utilized viral vectors in gene therapy due to its robust, long-term in vivo transgene expression and low toxicity.
James H. Kurasawa, Andrew Park, Carrie R. Sowers, Rebecca A. Halpin, Andrey Tovchigrechko, Claire L. Dobson, Albert E. Schmelzer, Changshou Gao, Susan D. Wilson, Yasuhiro Ikeda   +9 more
doaj   +1 more source

A Small Virus to Deliver Small Antibodies: New Targeted Therapies Based on AAV Delivery of Nanobodies

Microorganisms, 2021
Nanobodies are camelid-derived single-domain antibodies that present some advantages versus conventional antibodies, such as a smaller size, and higher tissue penetrability, stability, and hydrophilicity. Although nanobodies can be delivered as proteins,
Noelia Silva-Pilipich, Cristian Smerdou, Lucía Vanrell   +2 more
doaj   +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