Sort by:

• Highly Influential Citations
• Citations
• Acceleration
• Velocity
• Recency

Learn More

Biochemical, pathological, and skeletal improvement of mucopolysaccharidosis VI after gene transfer to liver but not to muscle.

• Alessandra Tessitore, Armida Faella, +6 authors Alberto Auricchio
• 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… (More)

Long-term amelioration of feline Mucopolysaccharidosis VI after AAV-mediated liver gene transfer.

• Gabriella Cotugno, Patrizia Annunziata, +15 authors Alberto Auricchio
• Molecular therapy : the journal of the American…
• 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,… (More)

AAV-mediated gene transfer for retinal diseases.

• Mariacarmela Allocca, Alessandra Tessitore, Gabriella Cotugno, Alberto Auricchio
• Expert opinion on biological therapy
• 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… (More)

Different serum enzyme levels are required to rescue the various systemic features of the mucopolysaccharidoses.

• Gabriella Cotugno, Alessandra Tessitore, +10 authors Alberto Auricchio
• Human gene therapy
• 2010

Mucopolysaccharidoses (MPSs) are lysosomal storage disorders characterized by progressive accumulation of glycosaminoglycans (GAGs) in various tissues. Enzyme replacement therapy (ERT) for several… (More)

Amelioration of both functional and morphological abnormalities in the retina of a mouse model of ocular albinism following AAV-mediated gene transfer.

• Enrico Maria Surace, Luciano Domenici, +8 authors Alberto Auricchio
• Molecular therapy : the journal of the American…
• 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)… (More)

Impact of Age at Administration, Lysosomal Storage, and Transgene Regulatory Elements on AAV2/8-Mediated Rat Liver Transduction

• Gabriella Cotugno, Patrizia Annunziata, Maria Vittoria Barone, Marianthi Karali, Sandro Banfi, Alberto Auricchio
• PloS one
• 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… (More)

Similar therapeutic efficacy between a single administration of gene therapy and multiple administrations of recombinant enzyme in a mouse model of lysosomal storage disease.

• Donatella Calcara, Pamela Claudiani, Gabriella Cotugno, Paola Saccone, Elvira De Leonibus, Alberto Auricchio
• Human gene therapy
• 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… (More)

Inhibition of ocular neovascularization by hedgehog blockade.

• Enrico Maria Surace, Kamaljit S. Balaggan, +6 authors Alberto Auricchio
• Molecular therapy : the journal of the American…
• 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.… (More)

Robust RNAi enhancement via human Argonaute-2 overexpression from plasmids, viral vectors and cell lines

• Kathleen Boerner, Dominik Niopek, +19 authors Dirk Grimm
• Nucleic acids research
• 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… (More)

Noninvasive repetitive imaging of somatostatin receptor 2 gene transfer with positron emission tomography.

• Gabriella Cotugno, M. Trabucco Aurilio, +7 authors Alberto Auricchio
• Human gene therapy
• 2011

Noninvasive in vivo imaging of gene expression is desirable to monitor gene transfer in both animal models and humans. Reporter transgenes with low endogenous expression levels are instrumental to… (More)

• ‹
• 1
• 2
• 3
• ›