List of works - Pasqualina Colella

AAV-mediated gene supply for treatment of degenerative and neovascular retinal diseases.

scientific article published on October 2010

AP20187-mediated activation of a chimeric insulin receptor results in insulin-like actions in skeletal muscle and liver of diabetic mice.

scientific article

Author Correction: Engineering monocyte/macrophage-specific glucocerebrosidase expression in human hematopoietic stem cells using genome editing

scientific article published on 20 August 2020

Effective delivery of large genes to the retina by dual AAV vectors

scientific article published on 15 December 2013

Efficient gene delivery to the cone-enriched pig retina by dual AAV vectors

scientific article published on 27 February 2014

Emerging Issues in AAV-Mediated In Vivo Gene Therapy

scientific article published in December 2017

Engineering monocyte/macrophage-specific glucocerebrosidase expression in human hematopoietic stem cells using genome editing

scientific article published on 03 July 2020

Improved dual AAV vectors with reduced expression of truncated proteins are safe and effective in the retina of a mouse model of Stargardt disease

scientific article

Long-term exposure to Myozyme results in a decrease of anti-drug antibodies in late-onset Pompe disease patients

scientific article

Myosin7a deficiency results in reduced retinal activity which is improved by gene therapy

scientific article

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

scientific article published on 19 March 2011

Photoreceptor degeneration in mice: adeno-associated viral vector-mediated delivery of erythropoietin.

scientific article published in January 2013

Rescue of GSDIII Phenotype with Gene Transfer Requires Liver- and Muscle-Targeted GDE Expression.

scientific article published on 28 December 2017

Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase.

scientific article published in November 2017

Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice.

scientific article published on May 2008

Transposon-mediated Generation of Cellular and Mouse Models of Splicing Mutations to Assess the Efficacy of snRNA-based Therapeutics

scientific article published on 29 November 2016

List of works by Pasqualina Colella

AAV-mediated gene supply for treatment of degenerative and neovascular retinal diseases.

AP20187-mediated activation of a chimeric insulin receptor results in insulin-like actions in skeletal muscle and liver of diabetic mice.

Author Correction: Engineering monocyte/macrophage-specific glucocerebrosidase expression in human hematopoietic stem cells using genome editing

Effective delivery of large genes to the retina by dual AAV vectors

Efficient gene delivery to the cone-enriched pig retina by dual AAV vectors

Emerging Issues in AAV-Mediated In Vivo Gene Therapy

Engineering monocyte/macrophage-specific glucocerebrosidase expression in human hematopoietic stem cells using genome editing

Improved dual AAV vectors with reduced expression of truncated proteins are safe and effective in the retina of a mouse model of Stargardt disease

Long-term exposure to Myozyme results in a decrease of anti-drug antibodies in late-onset Pompe disease patients

Myosin7a deficiency results in reduced retinal activity which is improved by gene therapy

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

Photoreceptor degeneration in mice: adeno-associated viral vector-mediated delivery of erythropoietin.

Rescue of GSDIII Phenotype with Gene Transfer Requires Liver- and Muscle-Targeted GDE Expression.

Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase.

Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice.

Transposon-mediated Generation of Cellular and Mouse Models of Splicing Mutations to Assess the Efficacy of snRNA-based Therapeutics