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Expert Opinion on Orphan Drugs Volume 3, 2015 - Issue 6
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Review

Recombinant adeno-associated virus vectors in the treatment of rare diseases

Eric HastieGene Therapy Center, University of North Carolina, 7119 Thurston Bowles Building (104 Manning Drive), Campus Box 7352, Chapel Hill, NC 27599-7352, USA
(Postdoctoral Researcher) &
R Jude SamulskiDepartment of Pharmacology, University of North Carolina, 7119 Thurston Bowles Building (104 Manning Drive), Campus Box 7352, Chapel Hill, NC 27599-7352, USA+1 919 962 3285; +1 919 966 0907; [email protected]
(Director)
Pages 675-689 | Published online: 15 May 2015

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Esther Rodríguez-Márquez, Nadja Meumann & Hildegard Büning. (2021) Adeno-associated virus (AAV) capsid engineering in liver-directed gene therapy. Expert Opinion on Biological Therapy 21:6, pages 749-766.
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Articles from other publishers (17)

Emilia A. Zin, Bilge E. Ozturk, Deniz Dalkara & Leah C. Byrne. (2023) Developing New Vectors for Retinal Gene Therapy. Cold Spring Harbor Perspectives in Medicine 13:12, pages a041291.
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Sehar Riaz, Saumil Sethna, Todd Duncan, Muhammad A. Naeem, T. Michael Redmond, Sheikh Riazuddin, Saima Riazuddin, Livia S. Carvalho & Zubair M. Ahmed. (2023) Dual AAV-based PCDH15 gene therapy achieves sustained rescue of visual function in a mouse model of Usher syndrome 1F. Molecular Therapy 31:12, pages 3490-3501.
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Odelya E. Pagovich, Katie M. Stiles, Anna E. Camilleri, Anthony R. Russo, Saparja Nag & Ronald G. Crystal. (2021) Gene therapy in a murine model of chronic eosinophilic leukemia-not otherwise specified (CEL-NOS). Leukemia 36:2, pages 525-531.
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Anna E. Camilleri, Saparja Nag, Anthony R. Russo, Katie M. Stiles, Ronald G. Crystal & Odelya E. Pagovich. (2021) Gene therapy for a murine model of eosinophilic esophagitis. Allergy 76:9, pages 2740-2752.
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Sheila Maestro, Nicholas D. Weber, Nerea Zabaleta, Rafael Aldabe & Gloria Gonzalez-Aseguinolaza. (2021) Novel vectors and approaches for gene therapy in liver diseases. JHEP Reports 3:4, pages 100300.
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Zheng Wei, Xiaomei Liu, Taiming Li, Xiaofang Li, Qungang Zhou, Jianxiang Wu & Chun Zhang. (2021) Transduction of mouse retina by insect cell packaged recombinant adeno-associated viruses and their mutants via intravitreal injection. Future Virology 16:3, pages 181-190.
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Marcus Davidsson, Matilde Negrini, Swantje Hauser, Alexander Svanbergsson, Marcus Lockowandt, Giuseppe Tomasello, Fredric P. Manfredsson & Andreas Heuer. (2020) A comparison of AAV-vector production methods for gene therapy and preclinical assessment. Scientific Reports 10:1.
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Sehrish Javed, Tharushan Selliah, Yu-Ju Lee & Wei-Hsiang Huang. (2020) Dosage-sensitive genes in autism spectrum disorders: From neurobiology to therapy. Neuroscience & Biobehavioral Reviews 118, pages 538-567.
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Vicky Ping ChenYang GaoLiyi GengMike SteeleNathan JenksKah-Whye PengStephen Brimijoin. (2020) Systemic Safety of a Recombinant AAV8 Vector for Human Cocaine Hydrolase Gene Therapy: A Good Laboratory Practice Preclinical Study in Mice. Human Gene Therapy 31:1-2, pages 70-79.
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Suhail Khoja, Matthew Nitzahn, Brian Truong, Jenna Lambert, Brandon Willis, Gabriella Allegri, Véronique Rüfenacht, Johannes Häberle & Gerald S. Lipshutz. (2019) A constitutive knockout of murine carbamoyl phosphate synthetase 1 results in death with marked hyperglutaminemia and hyperammonemia. Journal of Inherited Metabolic Disease 42:6, pages 1044-1053.
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David Hampson, Alexander Hooper & Yosuke Niibori. (2019) The Application of Adeno-Associated Viral Vector Gene Therapy to the Treatment of Fragile X Syndrome. Brain Sciences 9:2, pages 32.
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Andrès F. Muro, Lorenzo D’Antiga & Federico Mingozzi. 2019. Pediatric Hepatology and Liver Transplantation. Pediatric Hepatology and Liver Transplantation 799 829 .
Jean-Baptiste Dupont, Michael W. Lawlor & Martin K. Childers. 2019. Muscle Gene Therapy. Muscle Gene Therapy 565 577 .
Nily Dan. 2018. Lipid Nanocarriers for Drug Targeting. Lipid Nanocarriers for Drug Targeting 529 562 .
Françoise Piguet, Sandro Alves & Nathalie Cartier. (2017) Clinical Gene Therapy for Neurodegenerative Diseases: Past, Present, and Future. Human Gene Therapy 28:11, pages 988-1003.
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Julien Baruteau, Simon N. Waddington, Ian E. Alexander & Paul Gissen. (2017) Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects. Journal of Inherited Metabolic Disease 40:4, pages 497-517.
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Nily Dan. 2017. Nanostructures for Novel Therapy. Nanostructures for Novel Therapy 517 538 .

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