[HTML][HTML] PET imaging of focused-ultrasound enhanced delivery of AAVs into the murine brain

J Ajenjo, JW Seo, J Foiret, B Wu, MN Raie, J Wang… - Theranostics, 2023 - ncbi.nlm.nih.gov
Rationale: Despite recent advances in the use of adeno-associated viruses (AAVs) as
potential vehicles for genetic intervention of central and peripheral nervous system …

[HTML][HTML] Novel tool to quantify with single-cell resolution the number of incoming AAV genomes co-expressed in the mouse nervous system

CJ Maturana, JL Verpeut, M Kooshkbaghi, EA Engel - Gene therapy, 2023 - nature.com
Adeno-associated viral (AAV) vectors are an established and safe gene delivery tool to
target the nervous system. However, the payload capacity of< 4.9 kb limits the transfer of …

[HTML][HTML] Adeno-associated virus vectors for retinal gene therapy in basic research and clinical studies

X Xia, X Guo - Frontiers in Medicine, 2023 - frontiersin.org
Retinal degenerative diseases, including glaucoma, age-related macular degeneration,
diabetic retinopathy, and a broad range of inherited retinal diseases, are leading causes of …

Tolerability and tropism of recombinant adeno-associated virus vectors in the African green monkey (Chlorocebus sabaeus) anterior chamber

KJ Chern, KZ Issac, ZD Gumbs, ME O'Connor… - Gene Therapy, 2023 - nature.com
While many studies have investigated the use of recombinant adeno-associated vectors
(rAAV) in the posterior chamber for treatment of inherited retinal diseases, fewer studies …

[HTML][HTML] Topoisomerase Inhibitors Increase Episomal DNA Expression by Inducing the Integration of Episomal DNA in Hepatic Cells

A Gómez-Moreno, E San Sebastian, J Moya… - Pharmaceutics, 2023 - mdpi.com
Gene therapy is a promising strategy to treat and cure most inherited metabolic liver
disorders. Viral vectors such as those based on adeno-associated viruses (AAVs) and …

[图书][B] Terapia Gênica com hPEDF via vetores AAVs e Nanopartículas na Asma Alérgica

DP Ferreira - 2023 - books.google.com
A asma é uma doença inflamatória crônica que acomete os pulmões, muitas vezes
associada a um processo de remodelamento com obstrução das vias aéreas e …

The Development of a Prostaglandin-Based Gene Therapy for the Treatment of Glaucoma

KJ Chern - 2023 - search.proquest.com
Glaucoma affects approximately 80 million people worldwide and is a leading cause of
blindness. Glaucoma is characterized by an imbalance between aqueous humor production …