Adeno-associated virus as a delivery vector for gene therapy of …
2024年4月3日 · Adeno-associated virus (AAV) has emerged as a pivotal delivery tool in clinical gene therapy owing to its minimal pathogenicity and ability to establish long-term gene expression in...
Delivering AAV to the central nervous and sensory systems
These new vectors – predominately recombinant adeno-associated virus (AAV) variants – target nervous tissues with differing efficiencies. This review gives an overview of current gene therapies in in the brain, ear, and eye, and describes the optimal …
Designing and optimizing AAV-mediated gene therapy for ...
2024年9月27日 · Recombinant adeno-associated viruses (rAAVs) have emerged as an attractive tool for gene delivery, and demonstrated tremendous promise in gene therapy and gene editing—therapeutic modalities with potential “one-and-done” treatment benefits compared to conventional drugs.
AAV gene transfer therapy mechanism of action Following gene …
This review discusses the contribution of the innate and adaptive immune response against AAVs, highlighting the challenges and potential strategies to mitigate these responses, thereby enhancing...
Gene Therapy Using Adeno-Associated Virus Vectors - PMC
In the absence of helper virus, AAV (serotype 2) can set up latency by integrating into chromosome 19q13.4, establishing itself as the only mammalian DNA virus known to be capable of site-specific integration. The AAV-2 genome is a …
Adeno-associated virus vector as a platform for gene therapy ... - PubMed
Adeno-associated virus (AAV) vectors are the leading platform for gene delivery for the treatment of a variety of human diseases. Recent advances in developing clinically desirable AAV capsids, optimizing genome designs and harnessing revolutionary biotechnologies have contributed substantially to t …
Adeno-associated viruses for gene therapy - ScienceDirect
2024年2月1日 · In this review, we focus on AAV vector-based gene therapy, shedding light on this promising technique and its remarkable success in haemophilia, with a special focus on hepatic complications and their management in daily clinical practice.
A new vision for AAV-delivered gene therapies - Wyss Institute
2021年2月10日 · (BOSTON) — In recent years, adeno-associated virus (AAV) has been recognized as the leading vehicle (vector) for in vivo delivery of therapeutic genes because it is non-pathogenic and efficiently targets many different cell and tissue types. The recent Federal Drug Administration (FDA) approvals of AAV-based gene-replacement therapies to ...
Recombinant adeno-associated virus 8 vector in gene therapy ...
2024年1月1日 · Adeno-associated virus (AAV) is one of the most promising gene therapy vectors and a powerful tool for delivering the gene of interest. Among the AAV vectors, AAV serotype 8 (AAV8) has attracted much attention for its efficient and …
AAV-based in vivo gene therapy for neurological disorders
2023年9月1日 · A vector, such as an adeno-associated virus (AAV), can deliver a gene replacement therapy to provide a healthy copy of the mutated gene, or a gene supplementation therapy to provide a...