Gene Therapy of Inherited Retinopathies: A Long and Successful Road from Viral Vectors to Patients

Por Colella, Pasqualina, Auricchio, Alberto
Publicado em 2012

Abnormal autophagy, ubiquitination, inflammation and apoptosis are dependent upon lysosomal storage and are useful biomarkers of mucopolysaccharidosis VI

Por Tessitore, Alessandra, Pirozzi, Marinella, Auricchio, Alberto
Publicado em 2009

AAV2/8 Vectors Purified from Culture Medium with a Simple and Rapid Protocol Transduce Murine Liver, Muscle, and Retina Efficiently

Por Doria, Monica, Ferrara, Antonella, Auricchio, Alberto
Publicado em 2013

Vector platforms for gene therapy of inherited retinopathies

Por Trapani, Ivana, Puppo, Agostina, Auricchio, Alberto
Publicado no Prog Retin Eye Res (2014)

Gene Therapy of ABCA4-Associated Diseases

Por Auricchio, Alberto, Trapani, Ivana, Allikmets, Rando
Publicado no Cold Spring Harb Perspect Med (2015)

In vivo detection of gene expression in liver by (31)P nuclear magnetic resonance spectroscopy employing creatine kinase as a marker gene

Por Auricchio, Alberto, Zhou, Rong, Wilson, James M., Glickson, Jerry D.
Publicado em 2001

Pharmacological read-through of nonsense ARSB mutations as a potential therapeutic approach for mucopolysaccharidosis VI

Por Bartolomeo, Rosa, Polishchuk, Elena V., Volpi, Nicola, Polishchuk, Roman S., Auricchio, Alberto
Publicado em 2012

Gene Therapy of Inherited Retinal Degenerations: Prospects and Challenges

Por Trapani, Ivana, Banfi, Sandro, Simonelli, Francesca, Surace, Enrico M., Auricchio, Alberto
Publicado no Hum Gene Ther (2015)

Hybrid Vectors Based on Adeno-Associated Virus Serotypes 2 and 5 for Muscle-Directed Gene Transfer

Por Hildinger, Markus, Auricchio, Alberto, Gao, Guangping, Wang, Lili, Chirmule, Narendra, Wilson, James M.
Publicado em 2001

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

Por Cotugno, Gabriella, Annunziata, Patrizia, Barone, Maria Vittoria, Karali, Marianthi, Banfi, Sandro, Auricchio, Alberto
Publicado em 2012

Effective AAV-mediated gene therapy in a mouse model of ethylmalonic encephalopathy

Por Di Meo, Ivano, Auricchio, Alberto, Lamperti, Costanza, Burlina, Alberto, Viscomi, Carlo, Zeviani, Massimo
Publicado em 2012

Similar Therapeutic Efficacy Between a Single Administration of Gene Therapy and Multiple Administrations of Recombinant Enzyme in a Mouse Model of Lysosomal Storage Disease

Por Ferla, Rita, Claudiani, Pamela, Cotugno, Gabriella, Saccone, Paola, De Leonibus, Elvira, Auricchio, Alberto
Publicado em 2014

Novel Adeno-associated Viruses Derived From Pig Tissues Transduce Most Major Organs in Mice

Por Bello, Alexander, Chand, Allan, Aviles, Jenna, Soule, Geoff, Auricchio, Alberto, Kobinger, Gary P.
Publicado em 2014

Low-dose Gene Therapy Reduces the Frequency of Enzyme Replacement Therapy in a Mouse Model of Lysosomal Storage Disease

Por Alliegro, Marialuisa, Ferla, Rita, Nusco, Edoardo, De Leonibus, Chiara, Settembre, Carmine, Auricchio, Alberto
Publicado no Mol Ther (2016)

Non-erythropoietic erythropoietin derivatives protect from light-induced and genetic photoreceptor degeneration

Por Colella, Pasqualina, Iodice, Carolina, Di Vicino, Umberto, Annunziata, Ida, Surace, Enrico M., Auricchio, Alberto
Publicado em 2011

The Microphthalmia Transcription Factor (Mitf) Controls Expression of the Ocular Albinism Type 1 Gene: Link between Melanin Synthesis and Melanosome Biogenesis

Por Vetrini, Francesco, Auricchio, Alberto, Du, Jinyan, Angeletti, Barbara, Fisher, David E., Ballabio, Andrea, Marigo, Valeria
Publicado em 2004

Sensory-motor behavioral characterization of an animal model of Maroteaux-Lamy syndrome (or Mucopolysaccharidosis VI)

Por Saccone, Paola, Cotugno, Gabriella, Russo, Fabio, Mastrogiacomo, Rosa, Tessitore, Alessandra, Auricchio, Alberto, De Leonibus, Elvira
Publicado em 2014

En Face Spectral-Domain Optical Coherence Tomography for the Monitoring of Lesion Area Progression in Stargardt Disease

Por Melillo, Paolo, Testa, Francesco, Rossi, Settimio, Di Iorio, Valentina, Orrico, Ada, Auricchio, Alberto, Simonelli, Francesca
Publicado no Invest Ophthalmol Vis Sci (2016)

Intramuscular viral delivery of paraplegin rescues peripheral axonopathy in a model of hereditary spastic paraplegia

Por Pirozzi, Marinella, Quattrini, Angelo, Andolfi, Gennaro, Dina, Giorgia, Malaguti, Maria Chiara, Auricchio, Alberto, Rugarli, Elena I.
Publicado em 2006

Intramuscular viral delivery of paraplegin rescues peripheral axonopathy in a model of hereditary spastic paraplegia

Por Pirozzi, Marinella, Quattrini, Angelo, Andolfi, Gennaro, Dina, Giorgia, Chiara Malaguti, Maria, Auricchio, Alberto, Rugarli, Elena I.
Publicado em 2014