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November 28, 2025Gene TherapyOpen Access

Multi-targeting zinc finger nuclease vector unsilences paternal UBE3A in a mouse model of Angelman syndrome

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Authors

HBHannah O. BazickLJLucas M. JamesBTBonnie Taylor-Blake

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Overview

Gene therapy finds zinc finger nuclease delivery restores paternal UBE3A expression in neurons, indicating potential for therapeutics.

Key Points

  • Restoration of paternal UBE3A expression occurs following zinc finger nuclease treatment in neuron cultures.
  • Significant findings include UBE3A levels resembling wild-type in some neurons of the AS-model mouse.
  • Gene therapy facilitated by adeno-associated virus demonstrates specificity by targeting only designated sites.
  • Potential therapeutic implications suggest addressing neurodevelopmental disorders like Angelman syndrome may be possible.

Cite This Study

Bazick et al. (2025) studied this question.

synapsesocial.com/papers/6928f113a65b730b9ea79fcchttps://doi.org/10.1038/s41434-025-00582-1
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