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Gene Therapy of Dominant CRX-Leber Congenital Amaurosis using Patient Stem Cell-Derived Retinal Organoids.
Kruczek, Kamil; Qu, Zepeng; Gentry, James; Fadl, Benjamin R; Gieser, Linn; Hiriyanna, Suja; Batz, Zachary; Samant, Mugdha; Samanta, Ananya; Chu, Colin J; Campello, Laura; Brooks, Brian P; Wu, Zhijian; Swaroop, Anand.
Afiliación
  • Kruczek K; Neurobiology, Neurodegeneration and Repair Laboratory, National Eye Institute, National Institutes of Health, MSC0610, 6 Center Drive, Bethesda, MD 20892, USA.
  • Qu Z; Neurobiology, Neurodegeneration and Repair Laboratory, National Eye Institute, National Institutes of Health, MSC0610, 6 Center Drive, Bethesda, MD 20892, USA.
  • Gentry J; Neurobiology, Neurodegeneration and Repair Laboratory, National Eye Institute, National Institutes of Health, MSC0610, 6 Center Drive, Bethesda, MD 20892, USA.
  • Fadl BR; Neurobiology, Neurodegeneration and Repair Laboratory, National Eye Institute, National Institutes of Health, MSC0610, 6 Center Drive, Bethesda, MD 20892, USA.
  • Gieser L; Neurobiology, Neurodegeneration and Repair Laboratory, National Eye Institute, National Institutes of Health, MSC0610, 6 Center Drive, Bethesda, MD 20892, USA.
  • Hiriyanna S; Ocular Gene Therapy Core, National Eye Institute, National Institutes of Health, Bethesda, MD, USA.
  • Batz Z; Neurobiology, Neurodegeneration and Repair Laboratory, National Eye Institute, National Institutes of Health, MSC0610, 6 Center Drive, Bethesda, MD 20892, USA.
  • Samant M; Neurobiology, Neurodegeneration and Repair Laboratory, National Eye Institute, National Institutes of Health, MSC0610, 6 Center Drive, Bethesda, MD 20892, USA.
  • Samanta A; Ocular Gene Therapy Core, National Eye Institute, National Institutes of Health, Bethesda, MD, USA.
  • Chu CJ; Laboratory of Immune System Biology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD, USA.
  • Campello L; Neurobiology, Neurodegeneration and Repair Laboratory, National Eye Institute, National Institutes of Health, MSC0610, 6 Center Drive, Bethesda, MD 20892, USA.
  • Brooks BP; Ophthalmic Genetics and Visual Function Branch, National Eye Institute, National Institutes of Health, Bethesda, MD, USA.
  • Wu Z; Ocular Gene Therapy Core, National Eye Institute, National Institutes of Health, Bethesda, MD, USA.
  • Swaroop A; Neurobiology, Neurodegeneration and Repair Laboratory, National Eye Institute, National Institutes of Health, MSC0610, 6 Center Drive, Bethesda, MD 20892, USA. Electronic address: swaroopa@nei.nih.gov.
Stem Cell Reports ; 16(2): 252-263, 2021 02 09.
Article en En | MEDLINE | ID: mdl-33513359
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Células Fotorreceptoras / Retina / Organoides / Transactivadores / Proteínas de Homeodominio / Amaurosis Congénita de Leber Tipo de estudio: Prognostic_studies Límite: Adult / Child / Child, preschool / Female / Humans Idioma: En Revista: Stem Cell Reports Año: 2021 Tipo del documento: Article País de afiliación: Estados Unidos
Texto completo
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XML
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Células Fotorreceptoras / Retina / Organoides / Transactivadores / Proteínas de Homeodominio / Amaurosis Congénita de Leber Tipo de estudio: Prognostic_studies Límite: Adult / Child / Child, preschool / Female / Humans Idioma: En Revista: Stem Cell Reports Año: 2021 Tipo del documento: Article País de afiliación: Estados Unidos