Titre : | Gene therapy’s comeback: how scientists are trying to make it safer |
Revue : | Nature |
Auteurs : | Ledford H |
Type de document : | Article |
Année de publication : | 31/05/2022 |
Langues: | Anglais |
Mots-clés : | article de type review ; CRISPR ; réponse immunitaire ; thérapie génique |
Résumé : |
Unwanted immune responses threaten to derail some gene therapies. But researchers are seeking ways to combat harmful inflammation.
After years of disappointment, gene-therapy research has undergone a renaissance, with several high-profile drug approvals and a string of promising clinical-trial results against devastating genetic diseases, including sickle-cell disease and some blood cancers. But as researchers attempt to develop treatments for new conditions, they are also trying to work out how to cope with worrying signs that immune responses to the therapies could hinder their efforts — and generate dangerous side effects. The concern was a popular topic at the American Society of Gene and Cell Therapy (ASGCT) annual meeting in Washington DC, held from 16 to 19 May, as presenters discussed the effects of inflammation on gene therapy and how to mitigate them. “Inflammation is par for the course, but often we don’t talk about it,” said Christine Kay, a surgeon at Vitreoretinal Associates in Gainesville, Florida, during a talk about gene therapies for eye disorders. “I’m glad that we’re beginning to.” Safety has long been a primary concern in gene-therapy research, particularly after the death of a gene-therapy trial participant and the discovery of gene-therapy-linked cancers around the turn of the century closed clinical trials and caused investors to pull away from the burgeoning field. But the field has resurged over the past decade, and several gene therapies have been approved by regulators around the world to treat conditions including cancer, blindness and a metabolic disorder. “We are on a roll with gene therapy,” Francis Collins, acting science adviser to US President Joe Biden, told the ASGCT meeting. “But we still have thousands of diseases that have not been approached.” |