Fantastic AAV gene therapy vectors and how to find them—random diversification, rational design and machine learning
Parvoviruses are a diverse family of small, non-enveloped DNA viruses that infect a wide
variety of species, tissues and cell types. For over half a century, their intriguing biology and …
variety of species, tissues and cell types. For over half a century, their intriguing biology and …
Valoctocogene roxaparvovec gene therapy for hemophilia A
MC Ozelo, J Mahlangu, KJ Pasi… - … England Journal of …, 2022 - Mass Medical Soc
Abstract Background Valoctocogene roxaparvovec (AAV5-hFVIII-SQ) is an adeno-
associated virus 5 (AAV5)–based gene-therapy vector containing a coagulation factor VIII …
associated virus 5 (AAV5)–based gene-therapy vector containing a coagulation factor VIII …
Progress in bioengineering of myotropic adeno-associated viral gene therapy vectors
J Liu, TW Koay, O Maiakovska, M Zayas… - Human Gene …, 2023 - liebertpub.com
The ability to specifically, safely, and efficiently transfer therapeutic payloads to the striated
musculature via a minimally invasive delivery route remains one of the most important but …
musculature via a minimally invasive delivery route remains one of the most important but …
[HTML][HTML] Novel human liver-tropic AAV variants define transferable domains that markedly enhance the human tropism of AAV7 and AAV8
M Cabanes-Creus, RG Navarro, E Zhu… - … Therapy Methods & …, 2022 - 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 …
Assessment of pre-clinical liver models based on their ability to predict the liver-tropism of adeno-associated virus vectors
A Westhaus, M Cabanes-Creus, KL Dilworth… - Human Gene …, 2023 - liebertpub.com
The liver is a prime target for in vivo gene therapies using recombinant adeno-associated
viral vectors. Multiple clinical trials have been undertaken for this target in the past 15 years; …
viral vectors. Multiple clinical trials have been undertaken for this target in the past 15 years; …
AAV-p40 bioengineering platform for variant selection based on transgene expression
A Westhaus, M Cabanes-Creus, T Jonker… - Human Gene …, 2022 - liebertpub.com
The power of adeno-associated viral (AAV)-directed evolution for identifying novel vector
variants with improved properties is well established, as evidenced by numerous …
variants with improved properties is well established, as evidenced by numerous …
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 …
Ancestral library identifies conserved reprogrammable liver motif on AAV capsid
Gene therapy is emerging as a modality in 21st-century medicine. Adeno-associated viral
(AAV) gene transfer is a leading technology to achieve efficient and durable expression of a …
(AAV) gene transfer is a leading technology to achieve efficient and durable expression of a …
Correlation of antigen expression with epigenetic modifications after rAAV delivery of a human factor IX variant in mice and rhesus macaques
K Pekrun, CJ Stephens, A Gonzalez-Sandoval… - Molecular Therapy, 2024 - cell.com
We investigated long-term human coagulation factor IX (huFIX) expression of a novel variant
when delivered into mice and rhesus macaques and compared transduction efficiencies …
when delivered into mice and rhesus macaques and compared transduction efficiencies …
Natural Adeno-Associated Virus Serotypes and Engineered Adeno-Associated Virus Capsid Variants: Tropism Differences and Mechanistic Insights
E Lopez-Gordo, K Chamberlain, JM Riyad… - Viruses, 2024 - mdpi.com
Today, adeno-associated virus (AAV)-based vectors are arguably the most promising in vivo
gene delivery vehicles for durable therapeutic gene expression. Advances in molecular …
gene delivery vehicles for durable therapeutic gene expression. Advances in molecular …