Mariangela Lupo

Pozzuoli, Italy

Last Updated: 09/26/2026

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Found 10 publications

Retinal gene therapy for Stargardt disease with dual AAV intein vectors is both safe and effective in large animal models.

Retinal gene therapy for Stargardt disease with dual AAV intein vectors is both safe and effective in large animal models.

Journal: Science advances
Published: March 26, 2025

A DNA base-specific sequence interposed between CRX and NRL contributes to RHODOPSIN expression.

A DNA base-specific sequence interposed between CRX and NRL contributes to RHODOPSIN expression.

Journal: Scientific reports
Published: July 05, 2024

CoCas9 is a compact nuclease from the human microbiome for efficient and precise genome editing.

CoCas9 is a compact nuclease from the human microbiome for efficient and precise genome editing.

Journal: Nature communications
Published: January 05, 2024

Medical Device Development for Children and Young People-Reviewing the Challenges and Opportunities.

Medical Device Development for Children and Young People-Reviewing the Challenges and Opportunities.

Journal: Pharmaceutics
Published: October 27, 2021

Inclusion of a degron reduces levelsof undesired inteins after AAV-mediated proteintrans-splicing in the retina.

Inclusion of a degron reduces levelsof undesired inteins after AAV-mediated proteintrans-splicing in the retina.

Journal: Molecular therapy. Methods & clinical development
Published: August 13, 2021

Children's Preferences for Oral Dosage Forms and Their Involvement in Formulation Research via EPTRI (European Paediatric Translational Research Infrastructure).

Children's Preferences for Oral Dosage Forms and Their Involvement in Formulation Research via EPTRI (European Paediatric Translational Research Infrastructure).

Journal: Pharmaceutics
Published: March 30, 2021

Paediatric clinical research in Europe: an insight on experts' needs and perspectives.

Paediatric clinical research in Europe: an insight on experts' needs and perspectives.

Journal: Contemporary clinical trials communications
Published: March 13, 2020

Intein-mediated protein trans-splicing expands adeno-associated virus transfer capacity in the retina.

Intein-mediated protein trans-splicing expands adeno-associated virus transfer capacity in the retina.

Journal: Science translational medicine
Published: September 17, 2018

Investigating the roles and training of paediatric research nurses working across Europe: a questionnaire-based survey.

Investigating the roles and training of paediatric research nurses working across Europe: a questionnaire-based survey.

Journal: BMJ paediatrics open
Published: June 27, 2017

Rhodopsin targeted transcriptional silencing by DNA-binding.

Rhodopsin targeted transcriptional silencing by DNA-binding.

Journal: eLife
Published: October 10, 2015
Showing 1-10 of 10

Last Updated: 09/26/2026