Transduction of rat pancreatic islets with pseudotyped adeno-associated virus vectors (2009)
Craig, Anthony T, Gavrilova, Oksana, Dwyer, Nancy K, Jou, William, Pack, Stephanie, Liu, Eric, ...
Abstract Background Pancreatic islet transplantation is a promising treatment for type I diabetes mellitus, but current immunosuppressive strategies do not consistently provide long-term survival of...
Tran, Simon D., Sugito, Takayuki, Dipasquale, Giovanni, Cotrim, Ana P., Bandyopadhyay, Bidhan C., Riddle, Kathryn, ...
There is no satisfactory conventional treatment for patients who experience irreversible salivary gland damage after therapeutic radiation for head and neck cancer or because of Sjögren's syndrome....
Lotery, Andrew J., Yang, Grace S., Mullins, Robert F., Russell, Stephen R., Schmidt, Michael, Stone, Edwin M., ...
Gene transfer using adeno-associated viruses (AAVs) has been effective for treating inherited retinal diseases in animal models. Further evaluation in primates must be performed prior to clinical...
Davidson, Beverly L., Stein, Colleen S., Heth, Jason A., Martins, Inês, Kotin, Robert M., Derksen, Todd A., ...
Recombinant adeno-associated virus vectors based on serotype 2 (rAAV2) can direct transgene expression in the central nervous system (CNS), but it is not known how other rAAV serotypes perform as CNS...
Cloning and Characterization of Adeno-Associated Virus Type 5
Chiorini, John A., Kim, Frank, Yang, Linda, Kotin, Robert M.
Adeno-associated virus type 5 (AAV5) is distinct from other dependovirus serotypes based on DNA hybridization and serological data. To better understand the biology of AAV5, we have cloned and...
Chiorini, John A., Afione, Sandra, Kotin, Robert M.
Adeno-associated virus (AAV) replication depends on two viral components for replication: the AAV nonstructural proteins (Rep) in trans, and inverted terminal repeat (ITR) sequences in cis. AAV type...
Chiorini, John A., Zimmermann, Bastian, Yang, Linda, Smith, Richard H., Ahearn, Aaron, Herberg, Friedrich, ...
Adeno-associated virus encodes four nonstructural proteins, which are known as Rep78, Rep68, Rep52, and Rep40. Expression of these nonstructural proteins affects cell growth and gene expression...
Zabner, Joseph, Seiler, Michael, Walters, Robert, Kotin, Robert M., Fulgeras, Wendy, Davidson, Beverly L., ...
In the genetic disease cystic fibrosis, recombinant adeno-associated virus type 2 (AAV2) is being investigated as a vector to transfer CFTR cDNA to airway epithelia. However, earlier work has shown...
Kaludov, Nikola, Brown, Kevin E., Walters, Robert W., Zabner, Joseph, Chiorini, John A.
Adeno-associated virus serotype 4 (AAV4) and AAV5 have different tropisms compared to AAV2 and to each other. We recently reported that α2-3 sialic acid is required for AAV5 binding and...
Schmidt, Michael, Chiorini, John A., Afione, Sandra, Kotin, Robert
Hormones and neurotransmitters utilize cyclic AMP (cAMP) as a second messenger in signal transduction pathways to regulate cell growth and division, differentiation, gene expression, and metabolism....
PKA/PrKX activity is a modulator of AAV/adenovirus interaction
Di Pasquale, Giovanni, Chiorini, John A.
Interference between viruses occurs when infection by one virus results in the inhibition of replication of another virus. Adeno-associated virus (AAV2) is a human parvovirus with the unique...
Cloning of an Avian Adeno-Associated Virus (AAAV) and Generation of Recombinant AAAV Particles
Bossis, Ioannis, Chiorini, John A.
Recent studies have proposed that adeno-associated viruses (AAVs) are not evolutionarily linked to other mammalian autonomous parvoviruses but are more closely linked to the autonomous parvoviruses...
Drapkin, Paola T., O’Riordan, Catherine R., Yi, Su Min, Chiorini, John A., Cardella, Jonathan, Zabner, Joseph, ...
Developing gene therapy for cystic fibrosis has been hindered by limited binding and endocytosis of vectors by human airway epithelia. Here we show that the apical membrane of airway epithelia...
Reengineered salivary glands are stable endogenous bioreactors for systemic gene therapeutics
Voutetakis, Antonis, Kok, Marc R., Zheng, Changyu, Bossis, Ioannis, Wang, Jianghua, Cotrim, Ana P., ...
The use of critical-for-life organs (e.g., liver or lung) for systemic gene therapeutics can lead to serious safety concerns. To circumvent such issues, we have considered salivary glands (SGs) as an...
Structure of Adeno-Associated Virus Serotype 5
Walters, Robert W., Agbandje-McKenna, Mavis, Bowman, Valorie D., Moninger, Thomas O., Olson, Norman H., Seiler, Michael, ...
Adeno-associated virus serotype 5 (AAV5) requires sialic acid on host cells to bind and infect. Other parvoviruses, including Aleutian mink disease parvovirus (ADV), canine parvovirus (CPV), minute...
Cloning and Characterization of a Bovine Adeno-Associated Virus
Schmidt, Michael, Katano, Hisako, Bossis, Ioannis, Chiorini, John A.
To better understand the relationship between primate adeno-associated viruses (AAVs) and those of other mammals, we have cloned and sequenced the genome of an AAV found as a contaminant in two...
Structure of Adeno-Associated Virus Type 4
Padron, Eric, Bowman, Valorie, Kaludov, Nikola, Govindasamy, Lakshmanan, Levy, Hazel, Nick, Phillip, ...
Adeno-associated virus (AAV) is a member of the Parvoviridae, belonging to the Dependovirus genus. Currently, several distinct isolates of AAV are in development for use in human gene therapy...
Identification and Characterization of Novel Adeno-Associated Virus Isolates in ATCC Virus Stocks
Schmidt, Michael, Grot, Emmanuelle, Cervenka, Peter, Wainer, Sandra, Buck, Charles, Chiorini, John A.
Adeno-associated viruses (AAVs) depend on a helper virus for efficient replication. To identify novel AAV isolates, we screened a diverse set of virus isolates for the presence of AAV DNA. AAVs found...
Gangliosides Are Essential for Bovine Adeno-Associated Virus Entry
Schmidt, Michael, Chiorini, John A.
Recombinant adeno-associated viruses (AAV) are promising gene therapy vectors. We have recently identified a bovine adeno-associated virus (BAAV) that demonstrates unique tropism and transduction...
Davidson, Beverly L., Stein, Colleen S., Heth, Jason A., Martins, Inês, Kotin, Robert M., Derksen, Todd A., ...
Recombinant adeno-associated virus vectors based on serotype 2 (rAAV2) can direct transgene expression in the central nervous system (CNS), but it is not known how other rAAV serotypes perform as CNS...
Cloning and Characterization of Adeno-Associated Virus Type 5
Chiorini, John A., Kim, Frank, Yang, Linda, Kotin, Robert M.
Adeno-associated virus type 5 (AAV5) is distinct from other dependovirus serotypes based on DNA hybridization and serological data. To better understand the biology of AAV5, we have cloned and...
Chiorini, John A., Afione, Sandra, Kotin, Robert M.
Adeno-associated virus (AAV) replication depends on two viral components for replication: the AAV nonstructural proteins (Rep) in trans, and inverted terminal repeat (ITR) sequences in cis. AAV type...
Chiorini, John A., Zimmermann, Bastian, Yang, Linda, Smith, Richard H., Ahearn, Aaron, Herberg, Friedrich, ...
Adeno-associated virus encodes four nonstructural proteins, which are known as Rep78, Rep68, Rep52, and Rep40. Expression of these nonstructural proteins affects cell growth and gene expression...
Zabner, Joseph, Seiler, Michael, Walters, Robert, Kotin, Robert M., Fulgeras, Wendy, Davidson, Beverly L., ...
In the genetic disease cystic fibrosis, recombinant adeno-associated virus type 2 (AAV2) is being investigated as a vector to transfer CFTR cDNA to airway epithelia. However, earlier work has shown...
Kaludov, Nikola, Brown, Kevin E., Walters, Robert W., Zabner, Joseph, Chiorini, John A.
Adeno-associated virus serotype 4 (AAV4) and AAV5 have different tropisms compared to AAV2 and to each other. We recently reported that α2-3 sialic acid is required for AAV5 binding and...
Schmidt, Michael, Chiorini, John A., Afione, Sandra, Kotin, Robert
Hormones and neurotransmitters utilize cyclic AMP (cAMP) as a second messenger in signal transduction pathways to regulate cell growth and division, differentiation, gene expression, and metabolism....
PKA/PrKX activity is a modulator of AAV/adenovirus interaction
Di Pasquale, Giovanni, Chiorini, John A.
Interference between viruses occurs when infection by one virus results in the inhibition of replication of another virus. Adeno-associated virus (AAV2) is a human parvovirus with the unique...
Cloning of an Avian Adeno-Associated Virus (AAAV) and Generation of Recombinant AAAV Particles
Bossis, Ioannis, Chiorini, John A.
Recent studies have proposed that adeno-associated viruses (AAVs) are not evolutionarily linked to other mammalian autonomous parvoviruses but are more closely linked to the autonomous parvoviruses...
Drapkin, Paola T., O’Riordan, Catherine R., Yi, Su Min, Chiorini, John A., Cardella, Jonathan, Zabner, Joseph, ...
Developing gene therapy for cystic fibrosis has been hindered by limited binding and endocytosis of vectors by human airway epithelia. Here we show that the apical membrane of airway epithelia...
Reengineered salivary glands are stable endogenous bioreactors for systemic gene therapeutics
Voutetakis, Antonis, Kok, Marc R., Zheng, Changyu, Bossis, Ioannis, Wang, Jianghua, Cotrim, Ana P., ...
The use of critical-for-life organs (e.g., liver or lung) for systemic gene therapeutics can lead to serious safety concerns. To circumvent such issues, we have considered salivary glands (SGs) as an...
Structure of Adeno-Associated Virus Serotype 5
Walters, Robert W., Agbandje-McKenna, Mavis, Bowman, Valorie D., Moninger, Thomas O., Olson, Norman H., Seiler, Michael, ...
Adeno-associated virus serotype 5 (AAV5) requires sialic acid on host cells to bind and infect. Other parvoviruses, including Aleutian mink disease parvovirus (ADV), canine parvovirus (CPV), minute...
Cloning and Characterization of a Bovine Adeno-Associated Virus
Schmidt, Michael, Katano, Hisako, Bossis, Ioannis, Chiorini, John A.
To better understand the relationship between primate adeno-associated viruses (AAVs) and those of other mammals, we have cloned and sequenced the genome of an AAV found as a contaminant in two...
Structure of Adeno-Associated Virus Type 4
Padron, Eric, Bowman, Valorie, Kaludov, Nikola, Govindasamy, Lakshmanan, Levy, Hazel, Nick, Phillip, ...
Adeno-associated virus (AAV) is a member of the Parvoviridae, belonging to the Dependovirus genus. Currently, several distinct isolates of AAV are in development for use in human gene therapy...
Identification and Characterization of Novel Adeno-Associated Virus Isolates in ATCC Virus Stocks
Schmidt, Michael, Grot, Emmanuelle, Cervenka, Peter, Wainer, Sandra, Buck, Charles, Chiorini, John A.
Adeno-associated viruses (AAVs) depend on a helper virus for efficient replication. To identify novel AAV isolates, we screened a diverse set of virus isolates for the presence of AAV DNA. AAVs found...
Gangliosides Are Essential for Bovine Adeno-Associated Virus Entry
Schmidt, Michael, Chiorini, John A.
Recombinant adeno-associated viruses (AAV) are promising gene therapy vectors. We have recently identified a bovine adeno-associated virus (BAAV) that demonstrates unique tropism and transduction...
Structurally Mapping the Diverse Phenotype of Adeno-Associated Virus Serotype 4▿
Govindasamy, Lakshmanan, Padron, Eric, McKenna, Robert, Muzyczka, Nicholas, Kaludov, Nikola, Chiorini, John A., ...
The adeno-associated viruses (AAVs) can package and deliver foreign DNA into cells for corrective gene delivery applications. The AAV serotypes have distinct cell binding, transduction, and antigenic...
DiMattia, Michael, Govindasamy, Lakshmanan, Levy, Hazel C., Gurda-Whitaker, Brittney, Kalina, Amy, Kohlbrenner, Erik, ...
The production, purification, crystallization and preliminary crystallographic analysis of empty adeno-associated virus serotype 5 capsids are reported.
Schmidt, Michael, Voutetakis, Antonis, Afione, Sandra, Zheng, Changyu, Mandikian, Danielle, Chiorini, John A.
Recombinant adeno-associated virus (rAAV) is a promising vector for gene therapy. Recent isolations of novel AAV serotypes have led to significant advances by broadening the tropism and increasing...
Schmidt, Michael, Govindasamy, Lakshmanan, Afione, Sandra, Kaludov, Nick, Agbandje-McKenna, Mavis, Chiorini, John A.
A new adeno-associated virus (AAV), referred to as AAV(VR-942), has been isolated as a contaminant of adenovirus strain simian virus 17. The sequence of the rep gene places it in the AAV serotype 2...
Transduction of rat pancreatic islets with pseudotyped adeno-associated virus vectors
Craig, Anthony T, Gavrilova, Oksana, Dwyer, Nancy K, Jou, William, Pack, Stephanie, Liu, Eric, ...
Ortolano, Saida, Di Pasquale, Giovanni, Crispino, Giulia, Anselmi, Fabio, Mammano, Fabio, Chiorini, John A.
Connexin 26 (Cx26) and connexin 30 (Cx30) are encoded by two genes (GJB2 and GJB6, respectively) that are found within 50 kb in the same complex deafness locus, DFNB1. Immunocytochemistry and...