Articles with "retinal gene" as a keyword



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Retinal gene delivery enhancement by lycopene incorporation into cationic niosomes based on DOTMA and polysorbate 60

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Published in 2017 at "Journal of Controlled Release"

DOI: 10.1016/j.jconrel.2017.03.386

Abstract: &NA; The present study aimed to evaluate the incorporation of the natural lipid lycopene into niosome formulations based on cationic lipid DOTMA and polysorbate 60 non‐ionic surfactant to analyze the potential application of this novel… read more here.

Keywords: delivery enhancement; incorporation; gene delivery; dotma polysorbate ... See more keywords
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Seeing the Light after 25 Years of Retinal Gene Therapy.

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Published in 2018 at "Trends in molecular medicine"

DOI: 10.1016/j.molmed.2018.06.006

Abstract: The retina has been at the forefront of translational gene therapy. Proof-of-concept that gene therapy could restore vision in a large animal led to the initiation of the first successful clinical trials and, in turn,… read more here.

Keywords: light years; gene therapy; retinal gene; seeing light ... See more keywords
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Immune responses to retinal gene therapy using adeno-associated viral vectors – Implications for treatment success and safety

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Published in 2021 at "Progress in Retinal and Eye Research"

DOI: 10.1016/j.preteyeres.2020.100915

Abstract: Recombinant adeno-associated virus (AAV) is the leading vector for gene therapy in the retina. As non-pathogenic, non-integrating, replication deficient vector, the recombinant virus efficiently transduces all key retinal cell populations. Successful testing of AAV vectors… read more here.

Keywords: immune responses; gene therapy; adeno associated; retinal gene ... See more keywords
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Retinal gene therapy: an eye-opener of the 21st century

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Published in 2020 at "Gene Therapy"

DOI: 10.1038/s41434-020-0168-2

Abstract: Combined efforts in the field of gene therapy have generated exceptional advances recently by delivering opportunities to treat diseases unreachable by established therapies [1–3]. This has only been possible by conjoining the strength of several… read more here.

Keywords: field; treatment; gene therapy; retinal gene ... See more keywords
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Every nano-step counts: a critical reflection on do’s and don’ts in researching nanomedicines for retinal gene therapy

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Published in 2023 at "Expert Opinion on Drug Delivery"

DOI: 10.1080/17425247.2023.2167979

Abstract: ABSTRACT Introduction Retinal disease affects millions of people worldwide, generating a massive social and economic burden. Current clinical trials for retinal diseases are dominated by gene augmentation therapies delivered with recombinant viruses as key players.… read more here.

Keywords: gene therapy; nano step; every nano; gene ... See more keywords
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Overview of Retinal Gene Therapy: Current Status and Future Challenges.

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Published in 2022 at "Cold Spring Harbor perspectives in medicine"

DOI: 10.1101/cshperspect.a041278

Abstract: The success of the first Food and Drug Administration (FDA)- and European Medicines Agency (EMA)-approved gene therapy for genetic disease, voretigene neparovovec-rzyl, (Luxturna) has helped pave the way for development of retinal gene therapies to… read more here.

Keywords: therapy current; overview retinal; gene therapy; gene ... See more keywords