Neuraminidase could improve AAV9 gene therapy for Batten disease

admin_vjr_user — Thu, 12 Aug 2021 10:07:41 +0000

Kathrin Meyer, PhD, Nationwide Children’s Hospital, Columbus, OH, discusses the administration of neuraminidase prior to or in combination with AAV9 gene therapy in Batten disease, a neurological condition that can lead to macular degeneration. In a mouse model, intravitreal administration of neuraminidase resulted in enhanced delivery of AVV9 into the eye by removing certain sugar residues on entry receptors, improving penetration of the virus into deeper structures of the eye. While additional research is required to assess safety in larger models, neuraminidase pretreatment holds potential in a range of neurological conditions affecting the eye, particularly when targeting the inner nuclear layer. This interview took place during the American Society for Cell & Gene Therapy 24th Annual Meeting 2021.

Batten Disease – VJRegenMed

Neuraminidase could improve AAV9 gene therapy for Batten disease