Search Results - "Cossette, Travis"

Enhancing face validity of mouse models of Alzheimer's disease with natural genetic variation by Onos, Kristen D, Uyar, Asli, Keezer, Kelly J, Jackson, Harriet M, Preuss, Christoph, Acklin, Casey J, O'Rourke, Rita, Buchanan, Rebecca, Cossette, Travis L, Sukoff Rizzo, Stacey J, Soto, Ileana, Carter, Gregory W, Howell, Gareth R

“…Classical laboratory strains show limited genetic diversity and do not harness natural genetic variation. Mouse models relevant to Alzheimer's disease (AD)…”
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Long-term Correction of Very Long-chain Acyl-CoA Dehydrogenase Deficiency in Mice Using AAV9 Gene Therapy by Keeler, Allison M, Conlon, Thomas, Walter, Glenn, Zeng, Huadong, Shaffer, Scott A, Dungtao, Fu, Erger, Kirsten, Cossette, Travis, Tang, Qiushi, Mueller, Christian, Flotte, Terence R

“…Very long-chain acyl-coA dehydrogenase (VLCAD) is the rate-limiting step in mitochondrial fatty acid oxidation. VLCAD-deficient mice and patients clinical…”
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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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Model‐EyeD: Harnessing the potential of the eye as a biomarker for Alzheimer’s disease and related dementias by Howell, Gareth R, Marola, Olivia J, Maclean, Michael, Cossette, Travis L, Hewes, Amanda, Morgan, James, Singer, Cliff, Risacher, Shannon L., Onos, Kristen D, Reagan, Alaina M

“…Background Disease‐modifying treatments for Alzheimer’s disease and related dementias (ADRD) will be most effective early in the disease process. Clinical use…”
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Stability and Safety of an AAV Vector for Treating RPGR-ORF15 X-Linked Retinitis Pigmentosa by Deng, Wen-Tao, Dyka, Frank M, Dinculescu, Astra, Li, Jie, Zhu, Ping, Chiodo, Vince A, Boye, Sanford L, Conlon, Thomas J, Erger, Kirsten, Cossette, Travis, Hauswirth, William W

“…Our collaborative successful gene replacement therapy using AAV vectors expressing a variant of human RPGR-ORF15 in two canine models provided therapeutic…”
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Long-term preservation of cone photoreceptors and restoration of cone function by gene therapy in the guanylate cyclase-1 knockout (GC1KO) mouse by Boye, Sanford L, Conlon, Thomas, Erger, Kirsten, Ryals, Renee, Neeley, Andy, Cossette, Travis, Pang, Jijing, Dyka, Frank M, Hauswirth, William W, Boye, Shannon E

“…The authors previously showed that subretinal delivery of AAV5 vectors containing murine guanylate cyclase-1 (GC1) cDNA driven by either photoreceptor-specific…”
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Dynamic bioinspired coculture model for probing ER + breast cancer dormancy in the bone marrow niche by Pradhan, Lina, Moore, DeVonte, Ovadia, Elisa M, Swedzinski, Samantha L, Cossette, Travis, Sikes, Robert A, van Golen, Kenneth, Kloxin, April M

“…Late recurrences of breast cancer are hypothesized to arise from disseminated tumor cells (DTCs) that reactivate after dormancy and occur most frequently with…”
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GENETICALLY DIVERSE MOUSE MODELS OF ALZHEIMER’S DISEASE EXHIBIT DIFFERENTIAL MYELOID CELL RESPONSE AND NEURODEGENERATION by Onos, Kristen D., Keezer, Kelly J., Williams, Harriet M., Preuss, Christoph, Acklin, Casey, O'Rourke, Rita, Buchanan, Rebecca, Cossette, Travis L., Carter, Gregory W., Sukoff Rizzo, Stacey J., Soto, Ileana, Howell, Gareth R.

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O1‐01‐03: GENETICALLY DIVERSE MOUSE MODELS OF ALZHEIMER'S DISEASE EXHIBIT DIFFERENTIAL MYELOID CELL RESPONSE AND NEURODEGENERATION by Onos, Kristen D., Keezer, Kelly J., Williams, Harriet M., Preuss, Christoph, Acklin, Casey, O'Rourke, Rita, Buchanan, Rebecca, Cossette, Travis L., Carter, Gregory W., Sukoff Rizzo, Stacey J., Soto, Ileana, Howell, Gareth R.

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Preclinical potency and safety studies of an AAV2-mediated gene therapy vector for the treatment of MERTK associated retinitis pigmentosa by Conlon, Thomas J, Deng, Wen-Tao, Erger, Kirsten, Cossette, Travis, Pang, Ji-jing, Ryals, Renee, Clément, Nathalie, Cleaver, Brian, McDoom, Issam, Boye, Shannon E, Peden, Marc C, Sherwood, Mark B, Abernathy, Corinne R, Alkuraya, Fowzan S, Boye, Sanford L, Hauswirth, William W

“…Abstract Proof of concept for MERTK gene replacement therapy has been demonstrated using different viral vectors in the Royal College of Surgeon (RCS) rat, a…”
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Recombinant AAV Serotype and Capsid Mutant Comparison for Pulmonary Gene Transfer of α-1-Antitrypsin Using Invasive and Noninvasive Delivery by Liqun Wang, Rejean, McLaughlin, Thomas, Cossette, Travis, Tang, Qiushi, Foust, Kevin, Campbell-Thompson, Martha, Martino, Ashley, Cruz, Pedro, Loiler, Scott, Mueller, Christian, Flotte, Terence R

“…Recombinant adeno-associated viral (rAAV) vectors have been widely used in pulmonary gene therapy research. In this study, we evaluated the transduction and…”
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Biodistribution and toxicology studies of AAV2/rh8-CBA- beta -hexosaminidase after intracranial delivery in C57/BI6 mice by Erger, Kirsten, Mandel, Ronald, Castleman, William, Campbell-Thompson, Martha, Abernathy, Corinne, Getz, Matthew, Cossette, Travis, Schneider, Lynda, Brown, Ryan, Sena-Esteves, Miguel, Conlon, Thomas

“…Tay-Sachs disease (TSD) is an autosomal recessive neurodegenerative disease resulting from a lack of functional lysosomal enzyme [Beta]-N-acetylhexosaminidase…”
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Efficient Hepatic Delivery and Expression from a Recombinant Adeno-associated Virus 8 Pseudotyped α1-Antitrypsin Vector by Conlon, Thomas J., Cossette, Travis, Erger, Kirsten, Choi, Young-Kook, Clarke, Tracy, Scott-Jorgensen, Marda, Song, Sihong, Campbell-Thompson, Martha, Crawford, James, Flotte, Terence R.

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Long-term Correction of Very Long-chain Acyl-CoA Dehydrogenase Deficiency in Mice Using AAV9 Gene Therapy

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Preclinical toxicology and biodistribution studies of recombinant adeno-associated virus 1 human acid α-glucosidase by Conlon, Thomas J, Erger, Kirsten, Porvasnik, Stacy, Cossette, Travis, Roberts, Cheryl, Combee, Lynn, Islam, Saleem, Kelley, Jeffry, Cloutier, Denise, Clément, Nathalie, Abernathy, Corinne R, Byrne, Barry J

“…A biodistribution and toxicology study was performed to test the acute toxicities of intradiaphragmatic injection of a recombinant adeno-associated virus…”
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Glycogen Storage Disease Type Ia in Canines : A Model for Human Metabolic and Genetic Liver Disease by Erger, Kirsten, Fiske, Laurie, Specht, Andrew J., Cossette, Travis, Verstegen, John, Campbell-Thompson, Martha, Struck, Maggie B., Lee, Young Mok, Chou, Janice Y., Byrne, Barry J., Correia, Catherine E., Mah, Cathryn S., Weinstein, David A., Conlon, Thomas J.

“…A canine model of Glycogen storage disease type Ia (GSDIa) is described. Affected dogs are homozygous for a previously described M121I mutation resulting in a…”
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Recombinant AAV Serotype and Capsid Mutant Comparison for Pulmonary Gene Transfer of [alpha]-1-Antitrypsin Using Invasive and Noninvasive Delivery by Liqun Wang, Rejean, Mclaughlin, Thomas, Cossette, Travis, Tang, Qiushi, Foust, Kevin, Campbell-thompson, Martha, Martino, Ashley, Cruz, Pedro, Loiler, Scott, Mueller, Christian, Flotte, Terence R

“…Recombinant adeno-associated viral (rAAV) vectors have been widely used in pulmonary gene therapy research. In this study, we evaluated the transduction and…”
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Systemic correction of a fatty acid oxidation defect by intramuscular injection of a recombinant adeno-associated virus vector by Conlon, Thomas J, Walter, Glenn, Owen, Renius, Cossette, Travis, Erger, Kirsten, Gutierrez, Greg, Goetzman, Eric, Matern, Dietrich, Vockley, Jerry, Flotte, Terence R

“…Mitochondrial beta-oxidation of fatty acids is required to meet physiologic energy requirements during illness and periods of fasting or physiologic stress,…”
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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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Efficient hepatic delivery and expression from a recombinant adeno-associated virus 8 pseudotyped alpha1-antitrypsin vector by Conlon, Thomas J, Cossette, Travis, Erger, Kirsten, Choi, Young-Kook, Clarke, Tracy, Scott-Jorgensen, Marda, Song, Sihong, Campbell-Thompson, Martha, Crawford, James, Flotte, Terence R

“…alpha1-Antitrypsin (AAT) deficiency is a single-gene disorder in which a mutation in the AAT (approved symbol SERPINA1) gene (PI*Z) leads to misfolding of the…”
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