Structural and functional characterization of capsid binding by anti-AAV9 monoclonal antibodies from infants after SMA gene therapy
GJ Logan, M Mietzsch, N Khandekar, A D'Silva… - Molecular Therapy, 2023 - cell.com
Success in the treatment of infants with spinal muscular atrophy (SMA) underscores the
potential of vectors based on adeno-associated virus (AAV). However, a major obstacle to …
potential of vectors based on adeno-associated virus (AAV). However, a major obstacle to …
Characterization of the humanized FRG mouse model and development of an AAV-LK03 variant with improved liver lobular biodistribution
M Cabanes-Creus, RG Navarro, SHY Liao… - … Therapy Methods & …, 2023 - cell.com
Recent clinical successes have intensified interest in using adeno-associated virus (AAV)
vectors for therapeutic gene delivery. The liver is a key clinical target, given its critical …
vectors for therapeutic gene delivery. The liver is a key clinical target, given its critical …
Enhanced AAV transduction across preclinical CNS models: A comparative study in human brain organoids with cross-species evaluations
M Drouyer, J Merjane, T Nedelkoska… - … Therapy-Nucleic Acids, 2024 - cell.com
Viral vectors based on recombinant adeno-associated virus (rAAV) have become the most
widely used system for therapeutic gene delivery in the central nervous system (CNS) …
widely used system for therapeutic gene delivery in the central nervous system (CNS) …
Predictive power of deleterious single amino acid changes to infer on AAV2 and AAV2-13 capsids fitness
T La Bella, B Bertin, A Mihaljevic, J Nozi, P Vidal… - … Therapy Methods & …, 2024 - cell.com
Adeno-associated virus (AAV) is the most widely used vector for in vivo gene transfer. A
major limitation of capsid engineering is the incomplete understanding of the consequences …
major limitation of capsid engineering is the incomplete understanding of the consequences …
Novel AAV variants with improved tropism for human Schwann cells
Gene therapies and associated technologies are transforming biomedical research and
enabling novel therapeutic options for patients living with debilitating and incurable genetic …
enabling novel therapeutic options for patients living with debilitating and incurable genetic …
Effects of altering heparan sulfate proteoglycan binding and capsid hydrophilicity on retinal transduction by adeno-associated virus
SM Crosson, A Bennett, D Fajardo, JJ Peterson… - Journal of …, 2021 - Am Soc Microbiol
Adeno-associated viruses (AAVs) have recently emerged as the leading vector for retinal
gene therapy. However, AAV vectors that are capable of achieving clinically relevant levels …
gene therapy. However, AAV vectors that are capable of achieving clinically relevant levels …
Intravitreal injection of a rationally designed AAV capsid library in non-human primate identifies variants with enhanced retinal transduction and neutralizing antibody …
Adeno-associated virus (AAV) continues to be the gold standard vector for therapeutic gene
delivery and has proven especially useful for treating ocular disease. Intravitreal injection …
delivery and has proven especially useful for treating ocular disease. Intravitreal injection …
Development of CNS tropic AAV1-like variants with reduced liver-targeting following systemic administration in mice
M Drouyer, J Merjane, D Nazareth, M Knight, S Scott… - Molecular Therapy, 2024 - cell.com
Directed evolution of natural AAV9 using peptide display libraries have been widely used in
the search for an optimal recombinant AAV (rAAV) for transgene delivery across the blood …
the search for an optimal recombinant AAV (rAAV) for transgene delivery across the blood …