Results 1 to 10 of about 32,863 (259)
Rationale and strategies for the development of safe and effective optimized AAV vectors for human gene therapy [PDF]
Molecular Therapy - Nucleic Acids, 2023 Recombinant adeno-associated virus (AAV) vectors have been, or are currently in use, in 332 phase I/II/III clinical trials in a number of human diseases, and in some cases, remarkable clinical efficacy has also been achieved.
Arun Srivastava
exaly +4 more sources
Redirecting AAV vectors to extrahepatic tissues. [PDF]
Mol Ther, 2023 Recombinant adeno-associated viral (AAV) vectors are the current benchmark for systemic delivery of gene therapies to multiple organs in vivo. Despite clinical successes, safe and effective gene delivery to extrahepatic tissues has proven challenging due to dose limiting toxicity arising from high liver uptake of AAV vectors.
Asokan A, Shen S.
europepmc +3 more sources
Innate Immune Responses to AAV Vectors [PDF]
Frontiers in Microbiology, 2011 Gene replacement therapy by in vivo delivery of adeno-associated virus (AAV) is attractive as a potential treatment for a variety of genetic disorders. However, while AAV has been used successfully in many models, other experiments in clinical trials and in animal models have been hampered by undesired responses from the immune system.
Giridhara R Jayandharan +2 more
exaly +4 more sources
T Cell-Mediated Immune Responses to AAV and AAV Vectors [PDF]
Frontiers in Immunology, 2021 Adeno-associated virus (AAV)-mediated gene transfer has benefited patients with inherited diseases, such as hemophilia B, by achieving long-term expression of the therapeutic transgene.
Hildegund C. J. Ertl
doaj +3 more sources
Biomedicines, 2014 Adeno-associated virus (AAV)-based vectors expressing therapeutic genes continue to demonstrate great promise for the treatment of a wide variety of diseases and together with other gene transfer vectors represent an emerging new therapeutic paradigm ...
J Fraser Wright, Wright J Fraser
exaly +3 more sources
A novel class of self-complementary AAV vectors with multiple advantages based on cceAAV lacking mutant ITR [PDF]
Molecular Therapy: Methods & Clinical DevelopmentSelf-complementary AAV vectors (scAAV) use a mutant inverted terminal repeat (mITR) for efficient packaging of complementary stranded DNA, enabling rapid transgene expression.
Junping Zhang +11 more
doaj +2 more sources
AAV vectors: The Rubik’s cube of human gene therapy [PDF]
Molecular Therapy, 2022 Richard Samulski, Achille François
exaly +2 more sources
Transcriptional Targeting Approaches in Cardiac Gene Transfer Using AAV Vectors [PDF]
Pathogens, 2023 Derk Frank +2 more
exaly +2 more sources
Co-transduction of dual-adeno-associated virus vectors in the neonatal and adult mouse utricles
Frontiers in Molecular Neuroscience, 2022 Adeno-associated virus (AAV)-mediated gene transfer is an efficient method of gene over-expression in the vestibular end organs. However, AAV has limited usefulness for delivering a large gene, or multiple genes, due to its small packaging capacity (<
Zhong-Rui Chen +19 more
doaj +1 more source
Hepatology Communications, 2023 Background and Aims:. Adeno-associated virus (AAV) vectors are widely used to deliver therapeutic transgenes to distinct tissues, including the liver.
Daniel Stone +20 more
doaj +1 more source