Gene Therapy

Latest Issues

Issues
Volume 53
  • Number 2, 2021
  • Number 2, 2021
Show more
Volume 53
  • Number 2, 2021
  • Number 2, 2021
Show more
Volume 53
  • Number 2, 2021
  • Number 2, 2021
Show more
Volume 53
  • Number 2, 2021
  • Number 2, 2021
Show more
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Latest articles

AAV3-miRNA vectors for growth suppression of human hepatocellular carcinoma cells in vitro and human liver tumors in a murine xenograft model in vivo
10.1038/s41434-020-0140-1
Ling Yin, Geoffrey D. Keeler, Yuanhui Zhang, Brad E. Hoffman, Chen Ling, Keyun Qing, Arun Srivastava
Rescue of retinal ganglion cells in optic nerve injury using cell-selective AAV mediated delivery of SIRT1
10.1038/s41434-021-00219-z
Ahmara G. Ross, Devin S. McDougald, Reas S. Khan, Thu T. Duong, Kimberly E. Dine, Puya Aravand, Jean Bennett, Venkata Ramana Murthy Chavali, Kenneth S. Shindler
Functional expression of complement factor I following AAV-mediated gene delivery in the retina of mice and human cells
10.1038/s41434-021-00239-9
Anna K. Dreismann, Michelle E. McClements, Alun R. Barnard, Elise Orhan, Jane P. Hughes, Peter J. Lachmann, Robert E. MacLaren
Nr2e3 is a genetic modifier that rescues retinal degeneration and promotes homeostasis in multiple models of retinitis pigmentosa
10.1038/s41434-020-0134-z
Sujun Li, Shyamtanu Datta, Emily Brabbit, Zoe Love, Victoria Woytowicz, Kyle Flattery, Jessica Capri, Katie Yao, Siqi Wu, Michael Imboden, Arun Upadhyay, Rasappa Arumugham, Wallace B. Thoreson, Margaret M. DeAngelis, Neena B. Haider
Therapeutic benefit after intracranial gene therapy delivered during the symptomatic stage in a feline model of Sandhoff disease
10.1038/s41434-020-00190-1
Victoria J. McCurdy, Aime K. Johnson, Heather L. Gray-Edwards, Ashley N. Randle, Allison M. Bradbury, Nancy E. Morrison, Misako Hwang, Henry J. Baker, Nancy R. Cox, Miguel Sena-Esteves, Douglas R. Martin
Liver targeting with rAAV7: balancing tropism with immune profiles
10.1038/s41434-021-00230-4
Terence R. Flotte

Most read

Nr2e3 is a genetic modifier that rescues retinal degeneration and promotes homeostasis in multiple models of retinitis pigmentosa
10.1038/s41434-020-0134-z
Sujun Li, Shyamtanu Datta, Emily Brabbit, Zoe Love, Victoria Woytowicz, Kyle Flattery, Jessica Capri, Katie Yao, Siqi Wu, Michael Imboden, Arun Upadhyay, Rasappa Arumugham, Wallace B. Thoreson, Margaret M. DeAngelis, Neena B. Haider
An adenoviral vector-based mucosal vaccine is effective in protection against botulism
10.1038/gt.2008.181
Q Xu, M E Pichichero, L L Simpson, Md Elias, L A Smith, M Zeng
Therapeutic benefit after intracranial gene therapy delivered during the symptomatic stage in a feline model of Sandhoff disease
10.1038/s41434-020-00190-1
Victoria J. McCurdy, Aime K. Johnson, Heather L. Gray-Edwards, Ashley N. Randle, Allison M. Bradbury, Nancy E. Morrison, Misako Hwang, Henry J. Baker, Nancy R. Cox, Miguel Sena-Esteves, Douglas R. Martin

Most cited

AAV3-miRNA vectors for growth suppression of human hepatocellular carcinoma cells in vitro and human liver tumors in a murine xenograft model in vivo
10.1038/s41434-020-0140-1
Ling Yin, Geoffrey D. Keeler, Yuanhui Zhang, Brad E. Hoffman, Chen Ling, Keyun Qing, Arun Srivastava
Rescue of retinal ganglion cells in optic nerve injury using cell-selective AAV mediated delivery of SIRT1
10.1038/s41434-021-00219-z
Ahmara G. Ross, Devin S. McDougald, Reas S. Khan, Thu T. Duong, Kimberly E. Dine, Puya Aravand, Jean Bennett, Venkata Ramana Murthy Chavali, Kenneth S. Shindler
Functional expression of complement factor I following AAV-mediated gene delivery in the retina of mice and human cells
10.1038/s41434-021-00239-9
Anna K. Dreismann, Michelle E. McClements, Alun R. Barnard, Elise Orhan, Jane P. Hughes, Peter J. Lachmann, Robert E. MacLaren
Nr2e3 is a genetic modifier that rescues retinal degeneration and promotes homeostasis in multiple models of retinitis pigmentosa
10.1038/s41434-020-0134-z
Sujun Li, Shyamtanu Datta, Emily Brabbit, Zoe Love, Victoria Woytowicz, Kyle Flattery, Jessica Capri, Katie Yao, Siqi Wu, Michael Imboden, Arun Upadhyay, Rasappa Arumugham, Wallace B. Thoreson, Margaret M. DeAngelis, Neena B. Haider
Therapeutic benefit after intracranial gene therapy delivered during the symptomatic stage in a feline model of Sandhoff disease
10.1038/s41434-020-00190-1
Victoria J. McCurdy, Aime K. Johnson, Heather L. Gray-Edwards, Ashley N. Randle, Allison M. Bradbury, Nancy E. Morrison, Misako Hwang, Henry J. Baker, Nancy R. Cox, Miguel Sena-Esteves, Douglas R. Martin
Liver targeting with rAAV7: balancing tropism with immune profiles
10.1038/s41434-021-00230-4
Terence R. Flotte

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