Genetic background modifies phenotypic severity and longevity in a mouse model of Niemann-Pick disease type C1
Rodriguez-Gil, Jorge L, Watkins-Chow, Dawn E, Baxter, Laura L, Elliot, Gene, Harper, Ursula L, Wincovitch, Stephen M, Wedel, Julia C, Incao, Arturo A, Huebecker, Mylene, Boehm, Frederick J, Garver, William S, Porter, Forbes D, Broman, Karl W, Platt, Frances M, Pavan, William J
Published in Disease models & mechanisms (13.03.2020)
Published in Disease models & mechanisms (13.03.2020)
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Systemic AAV9 gene therapy improves the lifespan of mice with Niemann-Pick disease, type C1
Chandler, Randy J, Williams, Ian M, Gibson, Alana L, Davidson, Cristin D, Incao, Arturo A, Hubbard, Brandon T, Porter, Forbes D, Pavan, William J, Venditti, Charles P
Published in Human molecular genetics (01.01.2017)
Published in Human molecular genetics (01.01.2017)
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Transcriptome of HPβCD-treated Niemann-Pick disease type C1 cells highlights GPNMB as a biomarker for therapeutics
Rodriguez-Gil, Jorge L, Baxter, Laura L, Watkins-Chow, Dawn E, Johnson, Nicholas L, Davidson, Cristin D, Carlson, Steven R, Incao, Arturo A, Wallom, Kerri L, Farhat, Nicole Y, Platt, Frances M, Dale, Ryan K, Porter, Forbes D, Pavan, William J
Published in Human molecular genetics (30.11.2021)
Published in Human molecular genetics (30.11.2021)
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Improved systemic AAV gene therapy with a neurotrophic capsid in Niemann-Pick disease type C1 mice
Davidson, Cristin D, Gibson, Alana L, Gu, Tansy, Baxter, Laura L, Deverman, Benjamin E, Beadle, Keith, Incao, Arturo A, Rodriguez-Gil, Jorge L, Fujiwara, Hideji, Jiang, Xuntian, Chandler, Randy J, Ory, Daniel S, Gradinaru, Viviana, Venditti, Charles P, Pavan, William J
Published in Life science alliance (01.10.2021)
Published in Life science alliance (01.10.2021)
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198. Adeno-Associated Viral Gene Therapy To Treat Niemann-Pick Disease, Type C1
Chandler, Randy J., Williams, Ian M., Incao, Arturo A., Porter, Forbes D., Pavan, William J., Venditti, Charles P.
Published in Molecular therapy (01.05.2015)
Published in Molecular therapy (01.05.2015)
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613. A Comparison of CNS Transduction After Systemic versus Cranial Delivery of an AAV2/9 CamKII Promoter-eGFP Vector in Mice
Gibson, Alana L., Chandler, Randy J., Williams, Ian M., Incao, Arturo A., Porter, Forbes D., Pavan, William J., Venditti, Charles P.
Published in Molecular therapy (01.05.2016)
Published in Molecular therapy (01.05.2016)
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Journal Article
Genetic background modifies phenotypic severity and longevity in a mouse model of Niemann-Pick disease type C1
Rodriguez-Gil, Jorge L, Watkins-Chow, Dawn E, Baxter, Laura L, Elliot, Gene, Harper, Ursula L, Wincovitch, Stephen M, Wedel, Julia C, Incao, Arturo A, Huebecker, Mylene, Boehm, Frederick J, Garver, William S, Porter, Forbes D, Broman, Karl W, Platt, Frances M, Pavan, William J
Published in Disease models & mechanisms (01.01.2020)
Get full text
Published in Disease models & mechanisms (01.01.2020)
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