Extracellular vesicles: nature's nanoparticles for improving gene transfer with adeno-associated virus vectors.
Sign Up to like & getrecommendations! Published in 2018 at "Wiley interdisciplinary reviews. Nanomedicine and nanobiotechnology"
DOI: 10.1002/wnan.1488
Abstract: Gene therapy, the ability to treat a disease at the level of nucleic acid, has journeyed from science fiction, to hard lessons learned from early clinical trials, to improved technologies with efficacy in patients for… read more here.
Keywords: associated virus; aav vectors; adeno associated; gene transfer ... See more keywords
CRISPR-Mediated Integration of Large Gene Cassettes Using AAV Donor Vectors.
Sign Up to like & getrecommendations! Published in 2017 at "Cell reports"
DOI: 10.1016/j.celrep.2017.06.064
Abstract: The CRISPR/Cas9 system has recently been shown to facilitate high levels of precise genome editing using adeno-associated viral (AAV) vectors to serve as donor template DNA during homologous recombination (HR). However, the maximum AAV packaging capacity… read more here.
Keywords: genome editing; integration; crispr mediated; donor ... See more keywords
Synthetically Engineered Adeno-Associated Virus for Efficient, Safe, and Versatile Gene Therapy Applications.
Sign Up to like & getrecommendations! Published in 2020 at "ACS nano"
DOI: 10.1021/acsnano.0c03850
Abstract: Gene therapy directly targets mutations causing disease, allowing for a specific treatment at a molecular level. Adeno-associated virus (AAV) has been of increasing interest as a gene delivery vehicle, as AAV vectors are safe, effective,… read more here.
Keywords: gene therapy; associated virus; aav vectors; adeno associated ... See more keywords
Self-inactivating, all-in-one AAV vectors for precision Cas9 genome editing via homology-directed repair in vivo
Sign Up to like & getrecommendations! Published in 2021 at "Nature Communications"
DOI: 10.1038/s41467-021-26518-y
Abstract: Adeno-associated virus (AAV) vectors are important delivery platforms for therapeutic genome editing but are severely constrained by cargo limits. Simultaneous delivery of multiple vectors can limit dose and efficacy and increase safety risks. Here, we… read more here.
Keywords: genome editing; directed repair; homology directed; aav vectors ... See more keywords
Protocol for efficient generation and characterization of adeno-associated viral (AAV) vectors.
Sign Up to like & getrecommendations! Published in 2017 at "Human gene therapy methods"
DOI: 10.1089/hum.2017.192
Abstract: AAV vectors are a powerful tool for gene transfer approaches. We have established a simple and fast plasmid-based production system for achieving high AAV titers within 6 working days. The same procedure can be used… read more here.
Keywords: aav; protocol efficient; efficient generation; characterization adeno ... See more keywords
AAV vectors: Are they safe?
Sign Up to like & getrecommendations! Published in 2020 at "Human gene therapy"
DOI: 10.1089/hum.2020.187
Abstract: letter to editors do not require abstracts. read more here.
Keywords: vectors safe; aav vectors;
Developing New Vectors for Retinal Gene Therapy.
Sign Up to like & getrecommendations! Published in 2023 at "Cold Spring Harbor perspectives in medicine"
DOI: 10.1101/cshperspect.a041291
Abstract: Since their discovery over 55 years ago, adeno-associated virus (AAV) vectors have become powerful tools for experimental and therapeutic in vivo gene delivery, particularly in the retina. Increasing knowledge of AAV structure and biology has… read more here.
Keywords: developing new; new vectors; aav vectors; gene delivery ... See more keywords
Semirational bioengineering of AAV vectors with increased potency and specificity for systemic gene therapy of muscle disorders
Sign Up to like & getrecommendations! Published in 2022 at "Science Advances"
DOI: 10.1126/sciadv.abn4704
Abstract: Bioengineering of viral vectors for therapeutic gene delivery is a pivotal strategy to reduce doses, facilitate manufacturing, and improve efficacy and patient safety. Here, we engineered myotropic adeno-associated viral (AAV) vectors via a semirational, combinatorial… read more here.
Keywords: muscle; gene therapy; aav vectors; specificity ... See more keywords
Overcoming the Challenges Imposed by Humoral Immunity to AAV Vectors to Achieve Safe and Efficient Gene Transfer in Seropositive Patients
Sign Up to like & getrecommendations! Published in 2022 at "Frontiers in Immunology"
DOI: 10.3389/fimmu.2022.857276
Abstract: One of the major goals of in vivo gene transfer is to achieve long-term expression of therapeutic transgenes in terminally differentiated cells. The extensive clinical experience and the recent approval of Luxturna® (Spark Therapeutics, now… read more here.
Keywords: overcoming challenges; aav; gene transfer; aav vectors ... 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
A strategy for high antibody expression with low anti-drug antibodies using AAV9 vectors
Sign Up to like & getrecommendations! Published in 2023 at "Frontiers in Immunology"
DOI: 10.3389/fimmu.2023.1105617
Abstract: Introduction Use of adeno-associated virus (AAV) vectors is complicated by host immune responses that can limit transgene expression. Recent clinical trials using AAV vectors to deliver HIV broadly neutralizing antibodies (bNAbs) by intramuscular administration resulted… read more here.
Keywords: drug antibodies; anti drug; expression; aav vectors ... See more keywords