Laure Blouin
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View article: Mobility Testing and Other Performance-Based Assessments of Functional Vision in Patients with Inherited Retinal Disease
Mobility Testing and Other Performance-Based Assessments of Functional Vision in Patients with Inherited Retinal Disease Open
In the field of clinical ophthalmology, many of the common visual function study end points do not effectively reflect the significant morbidity of inherited retinal diseases (IRDs) and its effect on the patient's quality of life. In the l…
View article: Gene Therapies for the Treatment of Leber Hereditary Optic Neuropathy
Gene Therapies for the Treatment of Leber Hereditary Optic Neuropathy Open
Introduction Leber hereditary optic neuropathy (LHON) is a rare, blinding, maternally inherited mitochondrial genetic disease in need of effective treatment. LHON is a nonsyndromic optic neuropathy affecting the retinal ganglion cells (RGC…
View article: Cross-Sectional Analysis of Baseline Visual Parameters in Subjects Recruited Into the RESCUE and REVERSE ND4-LHON Gene Therapy Studies
Cross-Sectional Analysis of Baseline Visual Parameters in Subjects Recruited Into the RESCUE and REVERSE ND4-LHON Gene Therapy Studies Open
Objective: This report presents a cross-sectional analysis of the baseline characteristics of subjects with Leber hereditary optic neuropathy enrolled in the gene therapy trials RESCUE and REVERSE, to illustrate the evolution of visual par…
View article: Bilateral visual improvement with unilateral gene therapy injection for Leber hereditary optic neuropathy
Bilateral visual improvement with unilateral gene therapy injection for Leber hereditary optic neuropathy Open
At 96 weeks after unilateral intravitreal injection of rAAV2/2- ND4 , vision improved in both eyes in 78% of subjects affected with LHON.
View article: Long-Term Follow-Up After Unilateral Intravitreal Gene Therapy for Leber Hereditary Optic Neuropathy: The RESTORE Study
Long-Term Follow-Up After Unilateral Intravitreal Gene Therapy for Leber Hereditary Optic Neuropathy: The RESTORE Study Open
Background: RESCUE and REVERSE were 2 Phase 3 clinical trials that assessed the efficacy and safety of intravitreal gene therapy with lenadogene nolparvovec (rAAV2/2- ND4 ) for the treatment of Leber hereditary optic neuropathy (LHON). RES…
View article: Immune Response and Intraocular Inflammation in Patients With Leber Hereditary Optic Neuropathy Treated With Intravitreal Injection of Recombinant Adeno-Associated Virus 2 Carrying the <i>ND4</i> Gene
Immune Response and Intraocular Inflammation in Patients With Leber Hereditary Optic Neuropathy Treated With Intravitreal Injection of Recombinant Adeno-Associated Virus 2 Carrying the <i>ND4</i> Gene Open
In this study, intravitreal administration of rAAV2/2-ND4 in patients with LHON was safe and well tolerated. Further investigations may shed light into the local immune response to rAAV2/2-ND4 as a potential explanation for the observed in…
View article: Safety of rAAV2/2-ND4 Gene Therapy for Leber Hereditary Optic Neuropathy
Safety of rAAV2/2-ND4 Gene Therapy for Leber Hereditary Optic Neuropathy Open
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