Your browser doesn't support javascript.
loading
Effective intravitreal gene delivery to retinal pigment epithelium with hyaluronic acid nanospheres.
Crane, Ryan; Makia, Mustafa S; Zeibak, Stephanie; Tebbe, Lars; Ikele, Larissa; Woods, Christian Rutan; Conley, Shannon M; Acharya, Ghanashyam; Naash, Muna I; Al-Ubaidi, Muayyad R.
Afiliación
  • Crane R; Department of Biomedical Engineering, University of Houston, Houston, TX 77204, USA.
  • Makia MS; Department of Biomedical Engineering, University of Houston, Houston, TX 77204, USA.
  • Zeibak S; Department of Biomedical Engineering, University of Houston, Houston, TX 77204, USA.
  • Tebbe L; Department of Biomedical Engineering, University of Houston, Houston, TX 77204, USA.
  • Ikele L; Department of Biomedical Engineering, University of Houston, Houston, TX 77204, USA.
  • Woods CR; Department of Biomedical Engineering, University of Houston, Houston, TX 77204, USA.
  • Conley SM; Department of Cell Biology, University of Oklahoma Health Sciences Center, Oklahoma City, OK 73104, USA.
  • Acharya G; Department of Surgery, Baylor College of Medicine, Houston, TX 77030, USA.
  • Naash MI; Department of Biomedical Engineering, University of Houston, Houston, TX 77204, USA.
  • Al-Ubaidi MR; College of Optometry, University of Houston, Houston, TX 77204, USA.
Mol Ther Nucleic Acids ; 35(2): 102222, 2024 Jun 11.
Article en En | MEDLINE | ID: mdl-38868364
ABSTRACT
Inherited retinal degeneration (IRD) can cause a wide range of different forms of vision loss and blindness, and in spite of extensive advancements in gene therapy research, therapeutic approaches for targeting IRDs are still lacking. We have recently developed an approach for the intravitreal co-delivery of hyaluronic-acid nanospheres (HA-NSs) with sulfotyrosine (ST), effectively reaching the outer retina from the vitreal cavity. Here, our goal was to understand whether DNA-filled HA-NSs could generate gene expression in the outer retina. TxRed-labeled HA-NSs were compacted with plasmid DNA carrying a GFP reporter gene and intravitreally injected into the mouse retina. Follow-up at 4 weeks showed widespread gene expression in the outer retina and reduced, albeit present, expression at 8 weeks post-injection. Further analysis revealed this expression to be largely localized to the retinal pigment epithelium (RPE). These data show that intravitreal delivery of HA-NSs is a promising non-viral platform for the delivery of therapeutic genes and can generate pan-tissue, persistent gene expression in the RPE.
Palabras clave
Texto completo
Añadir a Mi BVS
Imprimir
XML
PubMed Links
Buscar en Google

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Idioma: En Revista: Mol Ther Nucleic Acids Año: 2024 Tipo del documento: Article País de afiliación: Estados Unidos Pais de publicación: Estados Unidos
Texto completo
Añadir a Mi BVS
Imprimir
XML
PubMed Links
Buscar en Google

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Idioma: En Revista: Mol Ther Nucleic Acids Año: 2024 Tipo del documento: Article País de afiliación: Estados Unidos Pais de publicación: Estados Unidos