Research into gene and cell therapy has accelerated over the last two decades as we gradually overcame hurdles such as appropriate vector selection, insertional mutagenesis, and construct design. As a result, gene therapies for diseases such as spinal muscular atrophy (SMA) and Leber’s congenital amaurosis (LCA) have been approved. However, several questions still need to be addressed to bring more of these therapies to patients. These issues include off-target transduction of vectors such as adeno-associated virus (AAV), inefficient production of vectors (for example, the proportion of full to empty AAV capsids), and immune responses to vectors and expressed transgenes that can reduce efficacy and cause severe unwanted side effects in some cases.

This Methods Collection will focus on the new technologies used to drive forward cell and gene therapy. These could include novel vector production methods (e.g., improved methods for AAV capsid design for targeting different tissues, methods for GMP vector production), novel methods for producing gene-modified cells, technologies for the characterization of vectors (e.g., titration methods, empty-to-full AAV capsid ratio), and methods for the screening of pre-existing immunity to gene therapy vectors.

The goal of this collection is to bring together some of the most recent advances in cell and gene therapy to act as a resource for researchers and clinicians as they work to deliver more life-changing gene therapies to patients.