Adeno-associated virus (AAV)-based gene therapies (GT) have shown promise for the treatment of many diseases, including rare diseases with unmet medical needs. In...
PaVe-GT
Feb 27, 2023 · A rapidly maturing therapeutic platform — adeno-associated virus (AAV) gene therapy — holds great promise for many rare diseases. AAV gene therapies use a modified …
Next-generation strategies to improve safety and efficacy of …
Dec 1, 2024 · Three major directions for the global progress of adeno-associated virus (AAV) vectors for gene therapies (GT) are analyzed: 1) engineering vectors to increase transgene …
A Review of Analytical Methods for Adeno-Associated Virus
In this review, we summarize PK studies for the AAV-based GT products approved so far by U.S. Food and Drug Administration and European Medicines Agency. We then comparatively …
Recent advances in various adeno-associated viruses (AAVs) as …
Jan 12, 2024 · AAV vectors emerge as promising tools for HCC therapy due to their non-immunogenic nature, efficient cell entry, and prolonged gene expression. While AAV-mediated …
Immunogenicity assessment of AAV-based gene therapies: An IQ …
Sep 8, 2022 · A better understanding of the immunogenic properties of, and the immune pathways activated by, an AAV GT may help to optimize the immunosuppressive treatment …
经验分享：腺相关病毒载体（AAV）生物分布和脱落研究的分析方 …
Apr 29, 2024 · 基于腺相关病毒（Adeno-associated virus, AAV）的基因治疗（Gene therapy, GT）产品能够有效地递送目的基因并替代丢失或缺陷的基因，为患者的治疗带来希望。然 …
Hepatotoxicity in Adeno-Associated Viral Vector Gene Therapy
Dec 20, 2023 · Although mild in many patients, hepatotoxicity may affect efficacy of gene therapy (GT), lead to acute liver injury and death [2,3,4, 5•, 6•]. This has resulted in termination of …
Considerations for Preclinical Safety Assessment of Adeno …
Oct 7, 2018 · Herein, we discuss considerations in the design of a comprehensive preclinical safety program for AAV-based GT prior to administration in humans. Gene therapy (GT) is a …
AAV-mediated Gene Therapy and High-Dose, Peri-Gene Therapy …
Adeno-associated virus (AAV) mediated gene transfer (GT) is a rapidly emerging therapeutic approach for targeted gene delivery for the amelioration of inherited diseases, with 8 approved …