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- Publications
- Influence
AAV-mediated gene transfer for retinal diseases
- M. Allocca, A. Tessitore, Gabriella Cotugno, A. Auricchio
- Biology, Medicine
- Expert opinion on biological therapy
- 22 November 2006
Vectors based on the adeno-associated virus (rAAV) are able to transduce the retina of animal models, including non-human primates, for a long-term period, safely and at sustained levels. The ability… Expand
Long-term amelioration of feline Mucopolysaccharidosis VI after AAV-mediated liver gene transfer.
- Gabriella Cotugno, P. Annunziata, +15 authors A. Auricchio
- Biology, Medicine
- Molecular therapy : the journal of the American…
- 1 March 2011
Mucopolysaccharidosis VI (MPS VI) is caused by deficient arylsulfatase B (ARSB) activity resulting in lysosomal storage of glycosaminoglycans (GAGs). MPS VI is characterized by dysostosis multiplex,… Expand
Gene therapy for mucopolysaccharidosis type VI is effective in cats without pre-existing immunity to AAV8.
- R. Ferla, T. O'malley, +7 authors A. Auricchio
- Biology, Medicine
- Human gene therapy
- 30 January 2013
Liver gene transfer with adeno-associated viral (AAV) 2/8 vectors is being considered for therapy of systemic diseases like mucopolysaccharidosis type VI (MPS VI), a lysosomal storage disease due to… Expand
Impact of Age at Administration, Lysosomal Storage, and Transgene Regulatory Elements on AAV2/8-Mediated Rat Liver Transduction
- Gabriella Cotugno, P. Annunziata, M. Barone, M. Karali, S. Banfi, A. Auricchio
- Biology, Medicine
- PloS one
- 13 March 2012
Liver-directed gene transfer is being investigated for the treatment of systemic or liver-specific diseases. Recombinant vectors based on adeno-associated virus serotype 8 (AAV2/8) efficiently… Expand
Similar therapeutic efficacy between a single administration of gene therapy and multiple administrations of recombinant enzyme in a mouse model of lysosomal storage disease.
- R. Ferla, Pamela Claudiani, Gabriella Cotugno, P. Saccone, E. De Leonibus, A. Auricchio
- Medicine
- Human gene therapy
- 4 March 2014
Enzyme replacement therapy (ERT) has become the standard of care for several lysosomal storage disorders (LSDs). Despite ERT's undisputed efficacy, the requirement for multiple and costly… Expand
Biochemical, pathological, and skeletal improvement of mucopolysaccharidosis VI after gene transfer to liver but not to muscle.
- A. Tessitore, A. Faella, +6 authors A. Auricchio
- Biology, Medicine
- Molecular therapy : the journal of the American…
- 2008
Mucopolysaccharidosis VI (MPS VI) is caused by deficient activity of arylsulfatase B (ARSB), resulting in intralysosomal storage of dermatan sulfate (DS) and multisystem disease without central… Expand
Inhibition of ocular neovascularization by hedgehog blockade.
- E. Surace, K. Balaggan, +6 authors A. Auricchio
- Biology, Medicine
- Molecular therapy : the journal of the American…
- 1 March 2006
Ocular neovascularization associated with proliferative diabetic retinopathy and age-related macular degeneration is the leading cause of severe visual loss in adults in developed countries.… Expand
Robust RNAi enhancement via human Argonaute-2 overexpression from plasmids, viral vectors and cell lines
- K. Börner, Dominik Niopek, +19 authors D. Grimm
- Biology, Medicine
- Nucleic acids research
- 17 September 2013
As the only mammalian Argonaute protein capable of directly cleaving mRNAs in a small RNA-guided manner, Argonaute-2 (Ago2) is a keyplayer in RNA interference (RNAi) silencing via small interfering… Expand
Amelioration of both functional and morphological abnormalities in the retina of a mouse model of ocular albinism following AAV-mediated gene transfer.
- E. Surace, L. Domenici, +8 authors A. Auricchio
- Biology, Medicine
- Molecular therapy : the journal of the American…
- 1 October 2005
X-linked recessive ocular albinism type I (OA1) is due to mutations in the OA1 gene (approved gene symbol GPR143), which is expressed in the retinal pigment epithelium (RPE). The Oa1 (Gpr143)… Expand
Different serum enzyme levels are required to rescue the various systemic features of the mucopolysaccharidoses.
- Gabriella Cotugno, A. Tessitore, +10 authors A. Auricchio
- Biology, Medicine
- Human gene therapy
- 26 March 2010
Mucopolysaccharidoses (MPSs) are lysosomal storage disorders characterized by progressive accumulation of glycosaminoglycans (GAGs) in various tissues. Enzyme replacement therapy (ERT) for several… Expand