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 different...

Adeno-associated virus (AAV) is a versatile viral vector technology that can be engineered for very specific functionality in gene therapy applications. To date, AAV has been shown to be safe and effective in preclinical and clinical settings.

Adeno-associated viruses (AAVs) are the safest and most effective gene delivery vehicles to drive long-term transgene expression in gene therapy.

4 天之前 · Advancements in our understanding of genetic disease and adeno-associated virus has prompted great excitement into the field of AAV-mediated gene therapy, particularly for genetic diseases of the ...

Adeno-associated virus (AAV) vectors are currently among the most frequently used viral vectors for gene therapy. At recent meetings of the American Society for Gene Therapy, nearly half of the presentations involved the use of AAV.

2023年3月1日 · In this article, we present a review of AAV discovery, properties, different serotypes, and tropism, and a following detailed explanation of their uses in gene therapy for disease of different organs and systems.

2019年2月1日 · Preclinical and clinical successes in AAV-mediated gene replacement, gene silencing and gene editing have helped AAV gain popularity as the ideal therapeutic vector, with two AAV-based...

AAV is driving today’s therapeutic discoveries and is used in several US and EU approved gene therapies currently available. How does AAV work? Simply put, AAV can be transformed from a naturally occurring virus into a delivery mechanism for gene therapy.

In the present review, we address the different approaches to improve AAV vectors for gene therapy with a focus on AAV capsid selection and engineering, strategies to overcome anti-AAV immune response, and vector genome design, ending with a glimpse at vector production methods and the current state of recombinant AAV (rAAV) at the clinical level.

2024年12月4日 · With their safety profile, low immunogenicity, and flexibility for targeting specific tissues, AAVs have become leading tools in gene therapy and research, driving advancements across genetic and acquired disease treatments.