Abstract
Deadline for Manuscript Submission: January 15, 2025
Several recombinant adeno-associated virus (AAV) gene therapies have been approved for clinical use in the United States, Europe, and Japan. In some cases, previously unknown acute and chronic toxicities have been observed, either in trials or in clinical use post-approval. Among these are liver toxicity, cardiotoxicity, immune myositis, and thrombocytopenia, with or without thrombotic microangiopathy. To facilitate reporting and analysis of these recent observations, Human Gene Therapy is planning a Special Issue devoted to Toxicity and Safety in Clinical AAV Gene Therapy in 2025. We are seeking original papers, review articles, and minireviews addressing in ocular gene therapy. Such papers may include, but are not limited to: Either comprehensive or brief, focused review articles on specific types of AAV-related toxicity Studies of the pathobiologic mechanisms of toxicity with AAV delivery Approaches to improve AAV gene therapy safety, either in vector design or recipient therapy Methods and protocols, including standardized protocols for immunologic assays that may be relevant to improving vector safety
The target deadline for submissions is January 15, 2025. Submissions after that date may not be reviewed in time for the special issue.
https://mc.manuscriptcentral.com/hum
Special Issue: Toxicity and Safety in Clinical AAV Gene Therapy