Rescue of myocytes and locomotion through AAV2/9-2YF intracisternal gene therapy in a rat model of creatine transporter deficiency

Fernandes-Pires, Gabriella; Azevedo, Marcelo Duarte; Lanzillo, Marc; Roux-Petronelli, Clothilde; Binz, Pierre-Alain; Cudalbu, Cristina; Sandi, Carmen; Tenenbaum, Liliane; Braissant, Olivier

doi:10.1016/j.omtm.2024.101251

Molecular Therapy - Methods & Clinical Development

2024

DOI: 10.1016/j.omtm.2024.101251

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Rescue of myocytes and locomotion through AAV2/9-2YF intracisternal gene therapy in a rat model of creatine transporter deficiency

Gabriella Fernandes-Pires,

Marcelo Duarte Azevedo,

Marc Lanzillo

et al.

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Efficient AAV9 Purification Using a Single-Step AAV9 Magnetic Affinity Beads Isolation

Sia,

Fu,

Mohd Rodhi

et al. 2024

IJMS

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Adeno-associated viruses (AAVs) have emerged as promising tools for gene therapy due to their safety and efficacy in delivering therapeutic genes or gene editing sequences to various tissues and organs. AAV serotype 9 (AAV9), among AAV serotypes, stands out for its ability to efficiently target multiple tissues, thus holding significant potential for clinical applications. However, existing methods for purifying AAVs are cumbersome, expensive, and often yield inconsistent results. In this study, we explore a novel purification strategy utilizing Dynabeads™ CaptureSelect™ magnetic beads. The AAV9 magnetic beads capture AAV9 with high specificity and recovery between 70 and 90%, whereas the AAVX magnetic beads did not bind to the AAV9. Through continuous interaction with AAVs in solution, these beads offer enhanced clearance of genomic DNA and plasmids even in the absence of endonuclease. The beads could be regenerated at least eight times, and the used beads could be stored for up to six months and reused without a significant reduction in recovery. The potency of the AAV9-purified vectors in vivo was comparable to that of iodixanol purified vectors.

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Efficient AAV9 Purification Using a Single-Step AAV9 Magnetic Affinity Beads Isolation

Sia,

Fu,

Mohd Rodhi

et al. 2024

IJMS

View full text Add to dashboard Cite

show abstract

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Rescue of myocytes and locomotion through AAV2/9-2YF intracisternal gene therapy in a rat model of creatine transporter deficiency

Cited by 1 publication

References 59 publications

Efficient AAV9 Purification Using a Single-Step AAV9 Magnetic Affinity Beads Isolation

Efficient AAV9 Purification Using a Single-Step AAV9 Magnetic Affinity Beads Isolation

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