Search Results - "Beattie, Stuart G."

Long-Term Effect of Gene Therapy on Leber’s Congenital Amaurosis by Bainbridge, James W.B, Mehat, Manjit S, Sundaram, Venki, Robbie, Scott J, Barker, Susie E, Ripamonti, Caterina, Georgiadis, Anastasios, Mowat, Freya M, Beattie, Stuart G, Gardner, Peter J, Feathers, Kecia L, Luong, Vy A, Yzer, Suzanne, Balaggan, Kamaljit, Viswanathan, Ananth, de Ravel, Thomy J.L, Casteels, Ingele, Holder, Graham E, Tyler, Nick, Fitzke, Fred W, Weleber, Richard G, Nardini, Marko, Moore, Anthony T, Thompson, Debra A, Petersen-Jones, Simon M, Michaelides, Michel, van den Born, L. Ingeborgh, Stockman, Andrew, Smith, Alexander J, Rubin, Gary, Ali, Robin R

“…Long-term follow-up of 12 persons with Leber's congenital amaurosis treated with gene therapy showed that about half of them had improvements in retinal…”
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A largely random AAV integration profile after LPLD gene therapy by Kaeppel, Christine, Beattie, Stuart G, Fronza, Raffaele, van Logtenstein, Richard, Salmon, Florence, Schmidt, Sabine, Wolf, Stephan, Nowrouzi, Ali, Glimm, Hanno, von Kalle, Christof, Petry, Harald, Gaudet, Daniel, Schmidt, Manfred

“…An adeno-associated virus (AAV) vector encoding a variant of human lipoprotein lipase was recently approved in Europe as the first gene therapy for the…”
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Reversal of RNA Missplicing and Myotonia after Muscleblind Overexpression in a Mouse Poly(CUG) Model for Myotonic Dystrophy by Kanadia, Rahul N., Shin, Jihae, Yuan, Yuan, Beattie, Stuart G., Wheeler, Thurman M., Thornton, Charles A., Swanson, Maurice S.

“…RNA-mediated pathogenesis is a recently developed disease model that proposes that certain types of mutant genes produce toxic transcripts that inhibit the…”
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Transient and intensive pharmacological immunosuppression fails to improve AAV-based liver gene transfer in non-human primates by Unzu, Carmen, Hervás-Stubbs, Sandra, Sampedro, Ana, Mauleón, Itsaso, Mancheño, Uxua, Alfaro, Carlos, de Salamanca, Rafael Enríquez, Benito, Alberto, Beattie, Stuart G, Petry, Harald, Prieto, Jesús, Melero, Ignacio, Fontanellas, Antonio

“…Adeno-associated vectors (rAAV) have been used to attain long-term liver gene expression. In humans, the cellular immune response poses a serious obstacle for…”
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Development of a Liver-specific Tet-On Inducible System for AAV Vectors and Its Application in the Treatment of Liver Cancer by Vanrell, Lucia, Di Scala, Marianna, Blanco, Laura, Otano, Itziar, Gil-Farina, Irene, Baldim, Victor, Paneda, Astrid, Berraondo, Pedro, Beattie, Stuart G, Chtarto, Abdelwahed, Tenenbaum, Lilianne, Prieto, Jesús, Gonzalez-Aseguinolaza, Gloria

“…Recombinant adeno-associated virus (rAAV) are effective gene delivery vehicles that can mediate long-lasting transgene expression. However, tight regulation…”
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Effect of adeno-associated virus serotype and genomic structure on liver transduction and biodistribution in mice of both genders by Pañeda, Astrid, Vanrell, Lucia, Mauleon, Itsaso, Crettaz, Julien S, Berraondo, Pedro, Timmermans, Eric J, Beattie, Stuart G, Twisk, Jaap, van Deventer, Sander, Prieto, Jesus, Fontanellas, Antonio, Rodriguez-Pena, Maria Sol, Gonzalez-Aseguinolaza, Gloria

“…Recombinant adeno-associated viral (AAV) vectors have unique properties, which make them suitable vectors for gene transfer. Here we assess the liver…”
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Sustained Enzymatic Correction by rAAV-Mediated Liver Gene Therapy Protects Against Induced Motor Neuropathy in Acute Porphyria Mice by Unzu, Carmen, Sampedro, Ana, Mauleón, Itsaso, Alegre, Manuel, Beattie, Stuart G, de Salamanca, Rafael Enríquez, Snapper, Jolanda, Twisk, Jaap, Petry, Harald, González-Aseguinolaza, Gloria, Artieda, Julio, Rodríguez-Pena, María Sol, Prieto, Jesús, Fontanellas, Antonio

“…Acute intermittent porphyria (AIP) is characterized by a hereditary deficiency of hepatic porphobilinogen deaminase (PBGD) activity. Clinical features are…”
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AAV gene therapy as a means to increase apolipoprotein (Apo) A-I and high-density lipoprotein-cholesterol levels: correction of murine ApoA-I deficiency by Vaessen, Stefan F. C., Veldman, Robert Jan, Comijn, Elisabeth M., Snapper, Jolanda, Sierts, Jeroen A., van den Oever, Karin, Beattie, Stuart G., Twisk, Jaap, Kuivenhoven, Jan Albert

“…Background Inherited apolipoprotein (Apo) A‐I deficiency is an orphan disorder characterized by high‐density lipoprotein (HDL)‐cholesterol deficiency and…”
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Phenotypic Correction of a Mouse Model for Primary Hyperoxaluria With Adeno-associated Virus Gene Transfer by Salido, Eduardo, Rodriguez-Pena, Marisol, Santana, Alfredo, Beattie, Stuart G., Petry, Harald, Torres, Armando

“…Primary hyperoxaluria type I (PH1) is an inborn error of metabolism caused by deficiency of the hepatic enzyme alanine-glyoxylate aminotransferase (AGXT or…”
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Safety and liver transduction efficacy of rAAV5-cohPBGD in nonhuman primates: a potential therapy for acute intermittent porphyria by Pañeda, Astrid, Lopez-Franco, Esperanza, Kaeppel, Christine, Unzu, Carmen, Gil-Royo, Ana Gloria, D'Avola, Delia, Beattie, Stuart G, Olagüe, Cristina, Ferrero, Roberto, Sampedro, Ana, Mauleon, Itsaso, Hermening, Stephan, Salmon, Florence, Benito, Alberto, Gavira, Juan Jose, Cornet, María Eugenia, del Mar Municio, María, von Kalle, Christof, Petry, Harald, Prieto, Jesus, Schmidt, Manfred, Fontanellas, Antonio, González-Aseguinolaza, Gloria

“…Acute intermittent porphyria (AIP) results from haplo-insufficient activity of porphobilinogen deaminase (PBGD) and is characterized clinically by…”
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Reply to: NGS library preparation may generate artifactual integration sites of AAV vectors by Kaeppel, Christine, Beattie, Stuart G, Fronza, Raffaele, van Logtenstein, Richard, Salmon, Florence, Schmidt, Sabine, Wolf, Stephan, Nowrouzi, Ali, Glimm, Hanno, von Kalle, Christof, Petry, Harald, Gaudet, Daniel, Schmidt, Manfred

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In vivo post-transcriptional gene silencing of α-1 antitrypsin by adeno-associated virus vectors expressing siRNA by Cruz, Pedro E, Mueller, Christian, Cossette, Travis L, Golant, Alexandra, Tang, Qiushi, Beattie, Stuart G, Brantly, Mark, Campbell-Thompson, Martha, Blomenkamp, Keith S, Teckman, Jeffrey H, Flotte, Terence R

“…α-1 Antitrypsin (AAT) deficiency is one of the most common genetic diseases in North America, with a carrier frequency of approximately 4% in the US…”
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Adeno-associated virus liver transduction efficiency measured by in vivo [18F]FHBG positron emission tomography imaging in rodents and nonhuman primates by Pañeda, Astrid, Collantes, Maria, Beattie, Stuart G, Otano, Itzia, Snapper, Jolanda, Timmermans, Eric, Guembe, Laura, Petry, Harald, Lanciego, Jose Luis, Benito, Alberto, Prieto, Jesus, Rodriguez-Pena, Maria Sol, Peñuelas, Iván, Gonzalez-Aseguinolaza, Gloria

“…Recombinant adeno-associated virus 5 (rAAV5) represents a candidate vector with unique advantages for the treatment of hepatic disorders because of its narrow…”
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Recombinant adeno-associated virus-mediated gene delivery of long chain acyl coenzyme A dehydrogenase (LCAD) into LCAD-deficient mice by Beattie, Stuart G., Goetzman, Eric, Tang, Qiuishi, Conlon, Thomas, Campbell-Thompson, Martha, Matern, Dietrich, Vockley, Jerry, Flotte, Terence R.

“…Background Very long chain acyl coenzyme A (CoA) dehydrogenase (VLCAD) deficiency is a relatively common mitochondrial β‐oxidation disorder. The most severe…”
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Biochemical correction of short-chain acyl-coenzyme A dehydrogenase deficiency after portal vein injection of rAAV8-SCAD by Beattie, Stuart G, Goetzman, Eric, Conlon, Thomas, Germain, Sean, Walter, Glenn, Campbell-Thompson, Martha, Matern, Dietrich, Vockley, Jerry, Flotte, Terence R

“…Recombinant adeno-associated viral vectors pseudotyped with serotype 5 and 8 capsids (AAV5 and AAV8) have been shown to be efficient gene transfer reagents for…”
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Novel tools for production and purification of recombinant adeno-associated viral vectors by Harris, Julian D, Beattie, Stuart G, Dickson, J George

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Safe and efficacious delivery of the human clotting factor IX gene to non human primates using a recombinant AAV vector produced in a fully-scalable GMP-compliant production system by Nijmeijer, Bart A., Petry, Harald, Sonnemans, Marc A., Beattie, Stuart G., Salmon, Lisa Spronck Florence, Preusting, Hans, Van Oorschot, Arie, Twisk, Jaap

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In vivo post-transcriptional gene silencing of [alpha]-1 antitrypsin by adeno-associated virus vectors expressing siRNA by Cruz, Pedro E, Mueller, Christian, Cossette, Travis L, Golant, Alexandra, Tang, Qiushi, Beattie, Stuart G, Brantly, Mark, Campbell-Thompson, Martha, Blomenkamp, Keith S, Teckman, Jeffrey H, Flotte, Terence R

“…alpha-1 Antitrypsin (AAT) deficiency is one of the most common genetic diseases in North America, with a carrier frequency of approximately 4% in the US…”
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875. Ribozyme Approaches towards Down-Regulation of PiZ Mutant Human A-1 Anti-Trypsin by Conlon, Thomas J, Beattie, Stuart G, Snyder, Chris W, Lewin, Alfred S, Flotte, Terry R

“…Alpha 1-antitrypsin (AAT), a 52kDa serine protease inhibitor, is normally secreted from hepatocytes and circulates in the plasma, protecting lung elastin from…”
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