AAV gene therapy vectors in the TMJ
Sign Up to like & getrecommendations! Published in 2022 at "Clinical and Experimental Dental Research"
DOI: 10.1002/cre2.636
Abstract: The goal of this project was to evaluate the use of two adeno‐associated viral vector serotypes, adeno‐associated viral vectors (AAV)‐2 and AAV‐6, approved for and used for gene therapy in humans, for the delivery of… read more here.
Keywords: aav gene; therapy vectors; gene therapy; vectors tmj ... See more keywords
Is subretinal AAV gene replacement still the only viable treatment option for choroideremia?
Sign Up to like & getrecommendations! Published in 2021 at "Expert opinion on orphan drugs"
DOI: 10.1080/21678707.2021.1882300
Abstract: ABSTRACT Introduction: Choroideremia is an X-linked inherited retinal degeneration resulting from mutations in the CHM gene, encoding Rab escort protein-1 (REP1), a protein regulating intracellular vesicular transport. Loss-of-function mutations in CHM lead to progressive loss… read more here.
Keywords: choroideremia; subretinal aav; option choroideremia; gene ... See more keywords
Creating an arsenal of Adeno-associated virus (AAV) gene delivery stealth vehicles
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DOI: 10.1371/journal.ppat.1006929
Abstract: The Adeno-associated virus (AAV) gene delivery system is ushering in a new and exciting era in the United States; following the first approved gene therapy (Glybera) in Europe, the FDA has approved a second therapy,… read more here.
Keywords: gene; aav gene; gene delivery; adeno associated ... See more keywords
Organoids and microphysiological systems: Promising models for accelerating AAV gene therapy studies
Sign Up to like & getrecommendations! Published in 2022 at "Frontiers in Immunology"
DOI: 10.3389/fimmu.2022.1011143
Abstract: The FDA has predicted that at least 10-20 gene therapy products will be approved by 2025. The surge in the development of such therapies can be attributed to the advent of safe and effective gene… read more here.
Keywords: aav gene; organoids microphysiological; gene therapy; microphysiological systems ... See more keywords
Immunogenicity and toxicity of AAV gene therapy
Sign Up to like & getrecommendations! Published in 2022 at "Frontiers in Immunology"
DOI: 10.3389/fimmu.2022.975803
Abstract: Gene transfer using adeno-associated viral (AAV) vectors has made tremendous progress in the last decade and has achieved cures of debilitating diseases such as hemophilia A and B. Nevertheless, progress is still being hampered by… read more here.
Keywords: toxicity aav; aav gene; aav vectors; gene ... See more keywords