mRNA Gene Therapy Research Targeting Alpha-Galactosidase A...

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2025-12-16 10:03:36 - Translate

mRNA Gene Therapy Research Targeting Alpha-Galactosidase A Deficiency 2027

The Promise of a Curative Single Dose Gene Therapy Research represents the most profound potential shift in the management of this inherited condition, offering the possibility of a one-time, potentially curative treatment. Current investigations primarily utilize Adeno-Associated Virus (AAV) vectors to deliver a functional copy of the deficient GLA gene to the patient’s cells, often...

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