Rita L. Ferla

Naples, Italy

Last Updated: 09/26/2026

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Found 13 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

Safe and effective liver-directed AAV-mediated homology-independent targeted integration in mouse models of inherited diseases.

Safe and effective liver-directed AAV-mediated homology-independent targeted integration in mouse models of inherited diseases.

Journal: Cell reports. Medicine

Published: October 04, 2023

Efficacy, pharmacokinetics, and safety in the mouse and primate retina of dual AAV vectors for Usher syndrome type 1B.

Efficacy, pharmacokinetics, and safety in the mouse and primate retina of dual AAV vectors for Usher syndrome type 1B.

Journal: Molecular therapy. Methods & clinical development

Published: November 09, 2022

Liver-Directed Adeno-Associated Virus-Mediated Gene Therapy for Mucopolysaccharidosis Type VI.

Liver-Directed Adeno-Associated Virus-Mediated Gene Therapy for Mucopolysaccharidosis Type VI.

Journal: NEJM evidence

Published: July 01, 2022

Therapeutic homology-independent targeted integration in retina and liver.

Therapeutic homology-independent targeted integration in retina and liver.

Journal: Nature communications

Published: January 04, 2021

Low incidence of hepatocellular carcinoma in mice and cats treated with systemic adeno-associated viral vectors.

Low incidence of hepatocellular carcinoma in mice and cats treated with systemic adeno-associated viral vectors.

Journal: Molecular therapy. Methods & clinical development

Published: September 02, 2020

Current Status on Clinical Development of Adeno-Associated Virus-Mediated Liver-Directed Gene Therapy for Inborn Errors of Metabolism.

Current Status on Clinical Development of Adeno-Associated Virus-Mediated Liver-Directed Gene Therapy for Inborn Errors of Metabolism.

Journal: Human gene therapy

Published: September 14, 2019

Non-clinical Safety and Efficacy of an AAV2/8 Vector Administered Intravenously for Treatment of Mucopolysaccharidosis Type VI.

Non-clinical Safety and Efficacy of an AAV2/8 Vector Administered Intravenously for Treatment of Mucopolysaccharidosis Type VI.

Journal: Molecular therapy. Methods & clinical development

Published: May 04, 2017

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

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

Journal: Molecular therapy : the journal of the American Society of Gene Therapy

Published: June 01, 2016

Long-Term Effects of Safinamide on Dyskinesia in Mid- to Late-Stage Parkinson's Disease: A Post-Hoc Analysis.

Long-Term Effects of Safinamide on Dyskinesia in Mid- to Late-Stage Parkinson's Disease: A Post-Hoc Analysis.

Journal: Journal of Parkinson's disease

Published: September 26, 2015

Prevalence of anti-adeno-associated virus serotype 8 neutralizing antibodies and arylsulfatase B cross-reactive immunologic material in mucopolysaccharidosis VI patient candidates for a gene therapy trial.

Prevalence of anti-adeno-associated virus serotype 8 neutralizing antibodies and arylsulfatase B cross-reactive immunologic material in mucopolysaccharidosis VI patient candidates for a gene therapy trial.

Journal: Human gene therapy

Published: February 06, 2015

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

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

Journal: Human gene therapy

Published: April 15, 2014

Showing 1-12 of 13

Last Updated: 09/26/2026