Your browser doesn't support javascript.
loading
Vision on gyrate atrophy: why treat the eye?
Bergen, Arthur A; Buijs, Mark Jn; Ten Asbroek, Anneloor Lma; Balfoort, Berith M; Boon, Camiel Jf; Brands, Marion M; Wanders, Ronald Ja; van Karnebeek, Clara Dm; Houtkooper, Riekelt H.
Afiliación
  • Bergen AA; Department of Human Genetics, Section Ophthalmogenetics, Amsterdam UMC, University of Amsterdam, 1105 AZ, Amsterdam, the Netherlands.
  • Buijs MJ; Department of Ophthalmology, Amsterdam UMC, University of Amsterdam, 1105 AZ, Amsterdam, the Netherlands.
  • Ten Asbroek AL; Emma Center for Personalized Medicine, Amsterdam UMC, Amsterdam, the Netherlands.
  • Balfoort BM; Department of Human Genetics, Section Ophthalmogenetics, Amsterdam UMC, University of Amsterdam, 1105 AZ, Amsterdam, the Netherlands.
  • Boon CJ; Department of Human Genetics, Section Ophthalmogenetics, Amsterdam UMC, University of Amsterdam, 1105 AZ, Amsterdam, the Netherlands.
  • Brands MM; Laboratory Genetic Metabolic Diseases, Amsterdam Gastroenterology, Endocrinology, and Metabolism, Amsterdam UMC, University of Amsterdam, 1105 AZ, Amsterdam, the Netherlands.
  • Wanders RJ; Department of Ophthalmology, Amsterdam UMC, University of Amsterdam, 1105 AZ, Amsterdam, the Netherlands.
  • van Karnebeek CD; Department of Ophthalmology, Leiden UMC, Leiden, the Netherlands.
EMBO Mol Med ; 16(1): 4-7, 2024 Jan.
Article en En | MEDLINE | ID: mdl-38177529
ABSTRACT
In the April issue of this Journal, Boffa and coworkers put forward a new therapeutic approach for Gyrate Atrophy of the Choroid and Retina (GACR; OMIM 258870) (Boffa et al, 2023). The authors propose to apply gene therapy to the liver for GACR, a metabolic disease primarily affecting eyesight due to retinal degeneration. Their vision is enthusiastically supported by a News and Views comment in the same issue (Seker Yilmaz and Gissen, 2023). However, based on disease pathology, patient's needs, ethical considerations, therapeutic developmental time lines, and current state of the art of gene therapy for liver and eye, we have a different view on this issue We argue below that local treatment of the eye is the preferred option for GACR.
Asunto(s)
Texto completo
Añadir a Mi BVS
Imprimir
XML
PubMed Links
Buscar en Google

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Degeneración Retiniana / Atrofia Girata Aspecto: Ethics Límite: Humans Idioma: En Revista: EMBO Mol Med Asunto de la revista: BIOLOGIA MOLECULAR Año: 2024 Tipo del documento: Article País de afiliación: Países Bajos Pais de publicación: Reino Unido
Texto completo
Añadir a Mi BVS
Imprimir
XML
PubMed Links
Buscar en Google

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Degeneración Retiniana / Atrofia Girata Aspecto: Ethics Límite: Humans Idioma: En Revista: EMBO Mol Med Asunto de la revista: BIOLOGIA MOLECULAR Año: 2024 Tipo del documento: Article País de afiliación: Países Bajos Pais de publicación: Reino Unido