Based on their pre-clinical data, Bennett's team pursued
clinical trials in children with a defective form of the
RPE65 gene. Their initial trials showed a stark improvement in
light sensitivity and visual function in these children.[9][10][11][12] Based on this, the therapy, marketed as
LUXTURNA®, was approved by the FDA for use in humans. Currently, her laboratory is investigating gene therapy approaches for other retinal diseases.[13][14][15]
^Bennett, J.; Anand, V.; Acland, G. M.; Maguire, A. M. (2000). "Cross-species comparison of in vivo reporter gene expression after recombinant adeno-associated virus-mediated retinal transduction". Vertebrate Phototransduction and the Visual Cycle, Part B. Methods in Enzymology. Vol. 316. pp. 777–789.
doi:
10.1016/s0076-6879(00)16762-x.
ISBN978-0-12-182217-0.
ISSN0076-6879.
PMID10800714.
^Acland, G. M.; Aguirre, G. D.; Ray, J.; Zhang, Q.; Aleman, T. S.; Cideciyan, A. V.; Pearce-Kelling, S. E.; Anand, V.; Zeng, Y.; Maguire, A. M.; Jacobson, S. G. (May 2001). "Gene therapy restores vision in a canine model of childhood blindness". Nature Genetics. 28 (1): 92–95.
doi:
10.1038/ng0501-92.
ISSN1061-4036.
PMID11326284.
S2CID13105734.
^Bainbridge, James W. B.; Smith, Alexander J.; Barker, Susie S.; Robbie, Scott; Henderson, Robert; Balaggan, Kamaljit; Viswanathan, Ananth; Holder, Graham E.; Stockman, Andrew; Tyler, Nick; Petersen-Jones, Simon (2008-05-22). "Effect of gene therapy on visual function in Leber's congenital amaurosis". The New England Journal of Medicine. 358 (21): 2231–2239.
doi:
10.1056/NEJMoa0802268.
hdl:10261/271174.
ISSN1533-4406.
PMID18441371.
^US 2003003582, Wakefield, John & Bennett, Jean, "Trans-viral vector mediated gene transfer to the retina", issued 2003-01-02, assigned to Tranzyme Inc.
^US 10987433, Bennett, Jean; Bennicelli, Jeannette & Dooley, Scott J. et al., "Compositions and methods for Correction of Heritable Ocular Disease", published 2021-04-27, assigned to
The Trustees of the University of Pennsylvania and Lloyd G. Mitchell
Based on their pre-clinical data, Bennett's team pursued
clinical trials in children with a defective form of the
RPE65 gene. Their initial trials showed a stark improvement in
light sensitivity and visual function in these children.[9][10][11][12] Based on this, the therapy, marketed as
LUXTURNA®, was approved by the FDA for use in humans. Currently, her laboratory is investigating gene therapy approaches for other retinal diseases.[13][14][15]
^Bennett, J.; Anand, V.; Acland, G. M.; Maguire, A. M. (2000). "Cross-species comparison of in vivo reporter gene expression after recombinant adeno-associated virus-mediated retinal transduction". Vertebrate Phototransduction and the Visual Cycle, Part B. Methods in Enzymology. Vol. 316. pp. 777–789.
doi:
10.1016/s0076-6879(00)16762-x.
ISBN978-0-12-182217-0.
ISSN0076-6879.
PMID10800714.
^Acland, G. M.; Aguirre, G. D.; Ray, J.; Zhang, Q.; Aleman, T. S.; Cideciyan, A. V.; Pearce-Kelling, S. E.; Anand, V.; Zeng, Y.; Maguire, A. M.; Jacobson, S. G. (May 2001). "Gene therapy restores vision in a canine model of childhood blindness". Nature Genetics. 28 (1): 92–95.
doi:
10.1038/ng0501-92.
ISSN1061-4036.
PMID11326284.
S2CID13105734.
^Bainbridge, James W. B.; Smith, Alexander J.; Barker, Susie S.; Robbie, Scott; Henderson, Robert; Balaggan, Kamaljit; Viswanathan, Ananth; Holder, Graham E.; Stockman, Andrew; Tyler, Nick; Petersen-Jones, Simon (2008-05-22). "Effect of gene therapy on visual function in Leber's congenital amaurosis". The New England Journal of Medicine. 358 (21): 2231–2239.
doi:
10.1056/NEJMoa0802268.
hdl:10261/271174.
ISSN1533-4406.
PMID18441371.
^US 2003003582, Wakefield, John & Bennett, Jean, "Trans-viral vector mediated gene transfer to the retina", issued 2003-01-02, assigned to Tranzyme Inc.
^US 10987433, Bennett, Jean; Bennicelli, Jeannette & Dooley, Scott J. et al., "Compositions and methods for Correction of Heritable Ocular Disease", published 2021-04-27, assigned to
The Trustees of the University of Pennsylvania and Lloyd G. Mitchell