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Expert Opinion on Biological Therapy Volume 11, 2011 - Issue 8

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Recombinant AAV-directed gene therapy for type I glycogen storage diseases

Janice Y ChouEunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Section on Cellular Differentiation, Program on Developmental Endocrinology and Genetics, Building 10, Room 9D42, Bethesda, MD 20892 1830, USA+1 301 496 1094; +1 301 402 6035; [email protected]

Brian C MansfieldEunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Section on Cellular Differentiation, Program on Developmental Endocrinology and Genetics, Building 10, Room 9D42, Bethesda, MD 20892 1830, USA+1 301 496 1094; +1 301 402 6035; [email protected];Correlogic Systems, Inc., Germantown, MD 20876, USA

Pages 1011-1024 | Published online: 20 Apr 2011

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https://doi.org/10.1517/14712598.2011.578067

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Ersin Gümüş & Hasan Özen. (2023) Glycogen storage diseases: An update. World Journal of Gastroenterology 29:25, pages 3932-3963.
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Abishek Wadhwa, Anas Aljabbari, Abhijeet Lokras, Camilla Foged & Aneesh Thakur. (2020) Opportunities and Challenges in the Delivery of mRNA-Based Vaccines. Pharmaceutics 12:2, pages 102.
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Lisa Zhang, Jun‐Ho Cho, Irina Arnaoutova, Brian C. Mansfield & Janice Y. Chou. (2019) An evolutionary approach to optimizing glucose‐6‐phosphatase‐α enzymatic activity for gene therapy of glycogen storage disease type Ia. Journal of Inherited Metabolic Disease 42:3, pages 470-479.
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Zeljka Trepotec, Eva Lichtenegger, Christian Plank, Manish K. Aneja & Carsten Rudolph. (2019) Delivery of mRNA Therapeutics for the Treatment of Hepatic Diseases. Molecular Therapy 27:4, pages 794-802.
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Jonathan Spoor, Hamid Farajifard & Nima Rezaei. (2019) Congenital neutropenia and primary immunodeficiency diseases. Critical Reviews in Oncology/Hematology 133, pages 149-162.
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Young Mok Lee, Thomas J. Conlon, Andrew Specht, Kirsten E. Coleman, Laurie M. Brown, Ana M. Estrella, Monika Dambska, Kathryn R. Dahlberg & David A. Weinstein. (2018) Long-term safety and efficacy of AAV gene therapy in the canine model of glycogen storage disease type Ia. Journal of Inherited Metabolic Disease 41:6, pages 977-984.
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Anna Rita Cappello, Rosita Curcio, Rosamaria Lappano, Marcello Maggiolini & Vincenza Dolce. (2018) The Physiopathological Role of the Exchangers Belonging to the SLC37 Family. Frontiers in Chemistry 6.
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Janice Y. Chou, Goo-Young Kim & Jun-Ho Cho. (2017) Recent development and gene therapy for glycogen storage disease type Ia. Liver Research 1:3, pages 174-180.
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Samuel T. Crowley & Kevin G. Rice. (2015) “Evolving nanoparticle gene delivery vectors for the liver: What has been learned in 30 years”. Journal of Controlled Release 219, pages 457-470.
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Miguel ManoRudy IppodrinoLorena ZentilinSerena Zacchigna & Mauro Giacca. (2015) Genome-wide RNAi screening identifies host restriction factors critical for in vivo AAV transduction. Proceedings of the National Academy of Sciences 112:36, pages 11276-11281.
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Janice Y. Chou, Hyun Sik Jun & Brian C. Mansfield. (2014) Type I glycogen storage diseases: disorders of the glucose‐6‐phosphatase/glucose‐6‐phosphate transporter complexes. Journal of Inherited Metabolic Disease 38:3, pages 511-519.
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J. Clar, E. Mutel, B. Gri, A. Creneguy, A. Stefanutti, S. Gaillard, N. Ferry, O. Beuf, G. Mithieux, T. H. Nguyen & F. Rajas. (2015) Hepatic lentiviral gene transfer prevents the long-term onset of hepatic tumours of glycogen storage disease type 1a in mice. Human Molecular Genetics 24:8, pages 2287-2296.
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Daisuke Satoh, Tohru Maeda, Tetsuya Ito, Yoko Nakajima, Mariko Ohte, Akane Ukai, Katsunori Nakamura, Shin Enosawa, Masashi Toyota, Yoshitaka Miyagawa, Hajime Okita, Nobutaka Kiyokawa, Hidenori Akutsu, Akihiro Umezawa & Tamihide Matsunaga. (2013) Establishment and directed differentiation of induced pluripotent stem cells from glycogen storage disease type Ib patient. Genes to Cells 18:12, pages 1053-1069.
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Young Mok Lee, Chi-Jiunn Pan, Dwight D. Koeberl, Brian C. Mansfield & Janice Y. Chou. (2013) The upstream enhancer elements of the G6PC promoter are critical for optimal G6PC expression in murine glycogen storage disease type Ia. Molecular Genetics and Metabolism 110:3, pages 275-280.
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. (2012) Current World Literature. Current Opinion in Pediatrics 24:6, pages 770-779.
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Young Mok Lee, Hyun Sik Jun, Chi-Jiunn Pan, Su Ru Lin, Lane H. Wilson, Brian C. Mansfield & Janice Y. Chou. (2012) Prevention of hepatocellular adenoma and correction of metabolic abnormalities in murine glycogen storage disease type Ia by gene therapy. Hepatology 56:5, pages 1719-1729.
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Recombinant AAV-directed gene therapy for type I glycogen storage diseases

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