The optimization of upstream and downstream processes for production of recombinant
adeno-associated virus (rAAV) with consistent quality depends on the ability to rapidly …

In vivo gene replacement for the treatment of inherited disease is one of the most compelling
concepts in modern medicine. Adeno-associated virus (AAV) vectors have been extensively …

Gene therapy for hemophilia B aims to ameliorate bleeding risk and provide endogenous
factor IX (FIX) activity/synthesis through a single treatment, eliminating the requirement for …

Genome editing via homologous recombination (HR)(gene targeting) in human
hematopoietic stem cells (HSCs) has the power to reveal gene–function relationships and …

Here we investigated the transduction characteristics of adeno-associated viral vector (AAV)
serotypes 1, 2, 5, 8 and 9 in the marmoset cerebral cortex. Using three constructs that each …

Gene therapy finds its niche | Nature Biotechnology Skip to main content Thank you for visiting
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Etranacogene dezaparvovec (AMT-061) is a recombinant AAV5 vector including a gene
cassette containing the factor IX (FIX) Padua variant under the control of a liver-specific …

Adeno-associated virus (AAV) vectors are clinically proven gene delivery vehicles that are
attracting an increasing amount of attention. Non-genome-containing empty AAV capsids …

One of the main challenges in the gene therapy viral vector development is to establish an
optimized process for its large scale production. This requires optimization for upstream and …

Accurate titration of adeno-associated viral (AAV) vector genome copies is critical for
ensuring correct and reproducible dosing in both preclinical and clinical settings …