Expressing transgenes that exceed the packaging capacity of adeno-associated virus capsids

K Chamberlain, JM Riyad, T Weber - Human gene therapy methods, 2016 - liebertpub.com
Recombinant adeno-associated virus vectors (rAAV) are being explored as gene delivery
vehicles for the treatment of various inherited and acquired disorders. rAAVs are attractive …

Custom adeno-associated virus capsids: the next generation of recombinant vectors with novel tropism

N Muzyczka, KH Warrington Jr - Human gene therapy, 2005 - liebertpub.com
Recombinant gene delivery vehicles based on adeno-associated virus (rAAV) have
emerged as promising vectors for the correction of genetic and acquired human disease …

Can adeno-associated viral vectors deliver effectively large genes?

P Tornabene, I Trapani - Human gene therapy, 2020 - liebertpub.com
Gene therapy with adeno-associated viral (AAV) vectors has reached the clinical stage for
many inherited and acquired diseases. However, due to a cargo capacity limited to< 5 kb …

Gene therapy progress and prospects: recombinant adeno-associated virus (rAAV) vectors

TR Flotte - Gene therapy, 2004 - nature.com
Recombinant adeno-associated virus (rAAV) vectors are unique among the vector classes
currently available for human gene therapy in that they are based upon a class of viruses …

The role of the adeno-associated virus capsid in gene transfer

KM Van Vliet, V Blouin, N Brument… - Drug Delivery …, 2008 - Springer
Adeno-associated virus (AAV) is one of the most promising viral gene transfer vectors that
has been shown to effect long-term gene expression and disease correction with low toxicity …

Adeno-associated virus vector as a platform for gene therapy delivery

D Wang, PWL Tai, G Gao - Nature reviews Drug discovery, 2019 - nature.com
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 …

Adeno-associated virus vectors in clinical trials

BJ Carter - Human gene therapy, 2005 - liebertpub.com
ADENO-ASSOCIATED VIRUS (AAV) VECTORS were first devel-oped 20 years ago (Carter,
2004) and an AAV vector was first administered to a human subject in November 1995 …

Adeno-associated virus vectorology, manufacturing, and clinical applications

JC Grieger, RJ Samulski - Methods in enzymology, 2012 - Elsevier
Adeno-associated virus (AAV) has emerged as an attractive vector for gene therapy. The
benefits of using AAV for gene therapy include long-term gene expression, the inability to …

Designer gene delivery vectors: molecular engineering and evolution of adeno-associated viral vectors for enhanced gene transfer

I Kwon, DV Schaffer - Pharmaceutical research, 2008 - Springer
Gene delivery vectors based on adeno-associated virus (AAV) are highly promising due to
several desirable features of this parent virus, including a lack of pathogenicity, efficient …

Transient transfection methods for clinical adeno-associated viral vector production

JF Wright - Human gene therapy, 2009 - liebertpub.com
Recombinant adeno-associated virus (AAV)-based vectors expressing therapeutic gene
products have shown great potential for human gene therapy. One major challenge for …