Cargando…
Absence of lenadogene nolparvovec DNA in a brain tumor biopsy from a patient in the REVERSE clinical study, a case report
BACKGROUND: Leber Hereditary Optic Neuropathy (LHON) is a rare, maternally-inherited mitochondrial disease that primarily affects retinal ganglion cells (RGCs) and their axons in the optic nerve, leading to irreversible, bilateral severe vision loss. Lenadogene nolparvovec gene therapy was developed...
Autores principales: | Newman, Nancy J., Schniederjan, Matthew, Mendoza, Pia R., Calkins, David J., Yu-Wai-Man, Patrick, Biousse, Valérie, Carelli, Valerio, Taiel, Magali, Rugiero, Francois, Singh, Pramila, Rogue, Alexandra, Sahel, José-Alain, Ancian, Philippe |
---|---|
Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
BioMed Central
2022
|
Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9277876/ https://www.ncbi.nlm.nih.gov/pubmed/35820885 http://dx.doi.org/10.1186/s12883-022-02787-y |
Ejemplares similares
-
Biodistribution of intravitreal (lenadogene) nolparvovec gene therapy in nonhuman primates
por: Calkins, David J., et al.
Publicado: (2021) -
Indirect Comparison of Lenadogene Nolparvovec Gene Therapy Versus Natural History in Patients with Leber Hereditary Optic Neuropathy Carrying the m.11778G>A MT-ND4 Mutation
por: Carelli, Valerio, et al.
Publicado: (2022) -
Gene Therapies for the Treatment of Leber Hereditary Optic Neuropathy
por: Sahel, José-Alain, et al.
Publicado: (2021) -
Long-Term Follow-Up After Unilateral Intravitreal Gene Therapy for Leber Hereditary Optic Neuropathy: The RESTORE Study
por: Biousse, Valérie, et al.
Publicado: (2021) -
Natural history of patients with Leber hereditary optic neuropathy—results from the REALITY study
por: Yu-Wai-Man, Patrick, et al.
Publicado: (2021)