Exosome-associated SUMOylation mutant AAV demonstrates improved ocular gene transfer efficiency in vivo
Sign Up to like & getrecommendations! Published in 2020 at "Virus research"
DOI: 10.1016/j.virusres.2020.197966
Abstract: Exosome associated Adeno-associated virus (AAV) vectors have emerged as a promising tool in gene therapy. Recently, we elucidated the role of SUMOylation post-translational modification in AAV2 capsid and demonstrated that capsid modifications at SUMOylation sites,… read more here.
Keywords: sumoylation; exosome associated; ocular gene; gene transfer ... See more keywords
Improved ocular gene transfer with a Neddylation-site modified AAV-RPE65 vector in rd12 mice
Sign Up to like & getrecommendations! Published in 2020 at "Eye"
DOI: 10.1038/s41433-020-0838-8
Abstract: Leber congenital amaurosis type 2 (LCA2) is an inherited disease that affects the integrity of the retina and results in severe visual impairment early in life [1]. This disease is caused due to mutations in… read more here.
Keywords: vector; aav2 vectors; ocular gene; mice ... See more keywords
Advancements in ocular gene therapy delivery: vectors and subretinal, intravitreal, and suprachoroidal techniques
Sign Up to like & getrecommendations! Published in 2022 at "Expert Opinion on Biological Therapy"
DOI: 10.1080/14712598.2022.2121646
Abstract: ABSTRACT Introduction Ocular gene therapy represents fertile ground for rapid innovation, with ever-expanding therapeutic strategies, molecular targets, and indications. Areas covered Potential indications for ocular gene therapy have classically focused on inherited retinal disease (IRD)… read more here.
Keywords: advancements ocular; therapy; gene therapy; ocular gene ... See more keywords
Lentiviral Vectors for Ocular Gene Therapy
Sign Up to like & getrecommendations! Published in 2022 at "Pharmaceutics"
DOI: 10.3390/pharmaceutics14081605
Abstract: This review offers the basics of lentiviral vector technologies, their advantages and pitfalls, and an overview of their use in the field of ophthalmology. First, the description of the global challenges encountered to develop safe… read more here.
Keywords: therapy; gene therapy; lentiviral vectors; ocular gene ... See more keywords