Gene therapy articles from across Nature Portfolio

A lack of broadly applicable methods to precisely control therapeutic gene expression is a key challenge for gene therapies. We present a technology that offers a photoactivatable RNA base editor for tunable and reversible regulation of gene expression, with implications for improving the safety and efficacy of gene therapy.

A base editor designed using circular permutation reduces off-target effects while enhancing purity.

A lack of broadly applicable methods to precisely control therapeutic gene expression is a key challenge for gene therapies. We present a technology that offers a photoactivatable RNA base editor for tunable and reversible regulation of gene expression, with implications for improving the safety and efficacy of gene therapy.

Despite their immense potential, gene and cell therapies that target rare diseases are at risk of market withdrawal, owing to several challenges. The authors describe these hurdles and call for innovative measures to improve the economic sustainability of gene and cell therapies after regulatory approval.