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

Prompt coronary catheterization and revascularization have markedly improved the
outcomes of myocardial infarction, but have also resulted in a growing number of surviving …

Neurotropic adeno-associated virus (AAV) serotypes such as AAV9 have been
demonstrated to transduce spinal alpha motor neurons when administered intravenously (iv) …

Purpose of review In this review, we discuss the clinical and genetic features of 5q spinal
muscular atrophy and highlight approved and upcoming therapies. Recent findings We …

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 …

Adeno-associated viral vectors (AAVs) have become popular for gene therapy, given their
many advantages, including their reduced inflammatory profile compared with that of other …

In the past two decades, adeno-associated virus (AAV) vector manufacturing has made
remarkable advancements to meet large-scale production demands for preclinical and …

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 …

Recombinant adeno-associated virus (rAAV) has emerged as an important gene therapy
vector with many clinical trials currently in progress. Analytical characterization and …

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 …