RichardJude Samulski在Molecular Therapy上刚发表了一篇关于AAV载体的review,AAV Vectors The Rubik’s Cube of Human Gene Therapy。题目就很有意思,魔方本身有多个平面,每个平面上又有不同的颜色,各种角块、棱块的位置让魔方有海量的花色组合。AAV也是这样,不同的血清型,不同的衣壳改造方式,不同的表达框,也为...
Summary Adeno-associated virus (AAV) gene therapy is a promising approach for treating ocular monogenic or acquired diseases, though immunogenicity and safety remain critical considerations. We conducted a systematic review of 120 trials and 32 publications to assess immune responses across different ...
and AEs were medically manageable with appropriate monitoring and treatment20,21,22. Immune reactions stimulated by the AAV vector are thought to be the primary cause of AEs in systemic AAV gene therapy, and each vector serotype may have
Areas covered: In this review, we summarize the strategies of AAV gene therapy that are currently under preclinical and clinical evaluation for the treatment of degenerative neuromuscular disorders, with a focus on diseases such as DMD and SMA. In addition to gene replacement strategy, we provide ...
1.S.Ghosh,A.M.Brown,C.Jenkins,etal.,Viral Vector Systems for Gene Therapy:A Comprehensive Literature Review of Progress and Biosafety Challenges.Journal of ABSA International,2020,25(1):7-18. 3.J.Markarian,"Finding Success in AAV Manufacturing," BioPharm International 34(3)2021. ...
Phase 1 clinical trials using AAV gene therapy for pediatric disorders – spinal muscular atrophy (SMA) and giant axonal neuropathy (GAN) – are now underway.Areas covered: This review addresses the latest progress in the field of AAV gene delivery to the spinal cord, particularly focusing on ...
3. Keeler, A. M., & Flotte, T. R. (2019). Recombinant adeno-associated virus gene therapy in light of Luxturna (and Zolgensma and Glybera): Where are we, and how did we get here? *Annual Review of Virology*, 6, 601-621....
RichardJude Samulski在Molecular Therapy上刚发表了一篇关于AAV载体的review,AAV Vectors The Rubik’s Cube of Human Gene Therapy。题目就很有意思,魔方本身有多个平面,每个平面上又有不同的颜色,各种角块、棱块的位置让魔方有海量的花色组合。AAV也是这样,不同...
2. Allison M. Keeler and Terence R. Recombinant Adeno-Associated Virus Gene Therapy in Light of Luxturna (and Zolgensma and Glybera): Where Are We, and How Did We Get Here? Flotte Annual Review of Virology (2019) 6:601-621.3. Kuzmin DA, et al., The clinical landscape for AAV ...
Clinical applications of gene therapy for rare diseases: A review[J]. International Journal of Experimental Pathology, 2023. 3.https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/luxturna 4.https://www.fda.gov/vaccines-blood-biologics/zolgensma 5.https://www.fda.gov/...